BridgeBio Pharma (BBIO) has upcoming FDA regulatory milestones for BBO-11818. The table below outlines estimated target dates and event types for these pending regulatory actions.
This section highlights FDA-related milestones and regulatory updates for drugs developed by BridgeBio Pharma (BBIO).
Over the past two years, BridgeBio Pharma has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
BEYONTTRA, Encaleret, BBO-11818, Acoramidis, LGMD2I/R9, BBP-418, and Infigratinib. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
Select a button below to view the list of FDA events for that drug.
BEYONTTRA FDA Regulatory Events
BEYONTTRA is a drug developed by BridgeBio Pharma for the following indication: in individuals with transthyretin amyloid cardiomyopathy.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- BEYONTTRA
- Announced Date:
- May 6, 2026
- Indication:
- in individuals with transthyretin amyloid cardiomyopathy
Announcement
BridgeBio Pharma, Inc. announced the Brazilian Health Regulatory Agency (ANVISA) has granted marketing authorization for acoramidis, under the brand name BEYONTTRA, for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
AI Summary
BridgeBio Pharma announced that Brazil’s Health Regulatory Agency (ANVISA) has granted marketing authorization for acoramidis, branded as BEYONTTRA, to treat adult patients with wild-type or variant transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The approval is based on positive results from the Phase 3 ATTRibute-CM study.
In that study, acoramidis showed one of the fastest benefits seen in ATTR-CM trials: by Month 1 there were numerically fewer cumulative cardiovascular events (including cardiovascular mortality or recurrent heart-related hospitalizations) versus placebo; by Month 30 there was a 42% reduction in a composite of all-cause mortality and recurrent cardiovascular hospitalizations, and a 50% reduction in cumulative cardiovascular hospitalizations. BEYONTTRA is an oral near-complete (≥90%) stabilizer of transthyretin (TTR).
BridgeBio will partner with Biopas, a Swixx BioPharma company, to commercialize BEYONTTRA in Brazil, with rollout expected in the second half of 2026. Full prescribing information will be available through ANVISA; common side effects reported include mild gastrointestinal symptoms.Read Announcement
- Drug:
- BEYONTTRA
- Announced Date:
- May 4, 2026
- Indication:
- in individuals with transthyretin amyloid cardiomyopathy
Announcement
BridgeBio Pharma, Inc. announced today upcoming presentations, including one late-breaking oral presentation, of new data from the Phase 3 ATTRibute-CM study in individuals with transthyretin amyloid cardiomyopathy (ATTR-CM) at Heart Failure 2026, organized by the Heart Failure Association of the European Society of Cardiology (ESC-HF), taking place in Barcelona, Spain on May 9-12, 2026.
AI Summary
BridgeBio announced it will present new Phase 3 ATTRibute-CM data at Heart Failure 2026 in Barcelona (May 9–12, 2026). The program includes a late-breaking oral presentation from Bayer on acoramidis’ effect on the outcome of outpatient worsening heart failure, highlighting important clinical findings for people with transthyretin amyloid cardiomyopathy (ATTR-CM).
The late-breaking oral is titled “Effect of Acoramidis on Temporal Variability of Serum Transthyretin and its Influence on Outcomes: Insights from the ATTRibute-CM Trial,” presented by Senthil Selvaraj, M.D., on Monday, May 11 at 3:30 pm CEST. A moderated ePoster comparing acoramidis with tafamidis for hospitalizations, mortality, and safety will be presented by Emer Joyce, M.D., Ph.D., on Sunday, May 10 at 3:30 pm CEST.
Other presentations include data showing acoramidis attenuates NT‑proBNP rise (Marianna Fontana, May 10 at 3:30 pm CEST) and multiple posters on consistent quality‑of‑life benefits measured by KCCQ‑OS (May 9). These sessions will examine biomarkers, outcomes, and patient health status in ATTR‑CM.
Read Announcement
Encaleret FDA Regulatory Timeline and Events
Encaleret is a drug developed by BridgeBio Pharma for the following indication: Autosomal Dominant Hypocalcemia Type 1 (ADH1).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Encaleret
- Announced Date:
- May 5, 2026
- Indication:
- Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Announcement
BridgeBio Pharma, Inc. announced today that additional data in individuals with autosomal dominant hypocalcemia type 1 (ADH1) from CALIBRATE, its Phase 3 study of encaleret, will be shared in an oral presentation at the 2026 European Congress of Endocrinology (ECE) taking place in Prague, Czech Republic on May 9-12, 2026.
AI Summary
BridgeBio Pharma announced it will share additional data from CALIBRATE, its Phase 3 study of encaleret for autosomal dominant hypocalcemia type 1 (ADH1), in an oral presentation at the 2026 European Congress of Endocrinology in Prague (May 9–12). The CALIBRATE primary results are titled "Encaleret Restores Mineral Homeostasis in ADH1" and will be presented by Filomena Cetani, M.D., Ph.D., on Tuesday, May 12 at 11:50 am CEST.
BridgeBio staff will also present related findings: Arun Mathew from BridgeBio Endocrinology will give an oral talk on genetic testing in non-surgical hypoparathyroidism (over 400 individuals) at 12:20 pm CEST on May 12, and will present an ePoster on ADH1 epidemiology and clinical management. Additional disease-monitoring poster data (CLARIFY) will be shown by other investigators during the congress.
Read Announcement- Drug:
- Encaleret
- Announced Date:
- October 29, 2025
- Indication:
- Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Announcement
BridgeBio Pharma, Inc. announced positive topline results from CALIBRATE, its global Phase 3 study of encaleret in autosomal dominant hypocalcemia type 1 (ADH1).
AI Summary
BridgeBio Pharma announced positive topline results from CALIBRATE, a global Phase 3 study of encaleret in adults with autosomal dominant hypocalcemia type 1 (ADH1). Encaleret is an investigational oral negative allosteric modulator of the calcium-sensing receptor.
The trial enrolled 70 adults randomized 2:1 to encaleret or conventional therapy. At Week 24, 76% of encaleret patients hit target serum (8.3–10.7 mg/dL) and urine calcium levels compared with 4% on standard care (p<0.0001). In a key secondary analysis, 91% of encaleret patients achieved intact PTH above the lower limit versus 7% on conventional therapy (p<0.0001).
By Day 3, 71% of encaleret patients reached normal serum calcium, and by Week 20, 98% were within target ranges versus 33% with standard care. Encaleret also reduced 24-hour urine calcium by 200 mg/day on average and was well tolerated, with no study-drug discontinuations.
BridgeBio plans to submit a New Drug Application in the first half of 2026 and will start registrational studies of encaleret in chronic hypoparathyroidism and pediatric ADH1 next year.
Read Announcement- Drug:
- Encaleret
- Announced Date:
- September 2, 2025
- Indication:
- Autosomal Dominant Hypocalcemia Type 1 (ADH1)
Announcement
BridgeBio Pharma, Inc. announced today that an oral presentation of Phase 2 data in post-surgical hypoparathyroidism and two poster sessions on skeletal dysplasia data will be shared at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting 2025, taking place in Seattle, WA from September 5 - 8, 2025.
AI Summary
BridgeBio Pharma, Inc. announced that it will share new data at the American Society for Bone and Mineral Research (ASBMR) Annual Meeting 2025, held in Seattle, WA from September 5 – 8, 2025.
First, the company will deliver an oral presentation on Phase 2 results for its investigational oral therapy in post-surgical hypoparathyroidism. These data highlight how the drug performed in patients who develop low blood calcium after their parathyroid glands are removed during surgery.
In addition, BridgeBio will host two poster sessions focusing on skeletal dysplasia, a group of rare bone growth disorders. These posters will present the latest preclinical and clinical findings, offering insights into how the company’s treatment candidates could help children and adults with abnormal bone development.
The presentations underscore BridgeBio’s commitment to finding new treatments for rare bone and mineral diseases and to advancing patient care through scientific research.
Read Announcement
BBO-11818 FDA Regulatory Timeline and Events
BBO-11818 is a drug developed by BridgeBio Pharma for the following indication: Potent panKRAS Inhibitor.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- BBO-11818
- Announced Date:
- April 22, 2026
- Indication:
- Potent panKRAS Inhibitor
Announcement
BridgeBio Oncology Therapeutics, Inc today presented new preclinical data for BBO-11818, a selective, orally bioavailable non-covalent inhibitor that targets mutant KRAS in both the ON (active GTP-bound) and OFF (inactive GDP-bound) states with robust anti-tumor activity in KRAS-mutant preclinical models.
AI Summary
BridgeBio Oncology Therapeutics presented new preclinical data for BBO-11818, a selective, orally bioavailable, non‑covalent inhibitor that targets mutant KRAS in both the ON (GTP‑bound) and OFF (GDP‑bound) states. In laboratory studies, BBO‑11818 potently suppressed MAPK signaling and strongly inhibited cell proliferation in KRAS‑mutant cell lines. Its selectivity and oral dosing profile may make it practical for further clinical testing.
In animal tumor models, BBO‑11818 showed robust anti‑tumor activity as a monotherapy and increased benefit when used in combination with other treatments across multiple KRAS‑mutant tumor types. The company expects to report updated Phase 1 clinical data in the second half of 2026. If clinical results match these preclinical findings, BBO‑11818 could become a promising option for treating cancers driven by diverse KRAS mutations.
Read Announcement- Drug:
- BBO-11818
- Announced Date:
- April 20, 2026
- Target Action Date:
- H2 2026
- Estimated Target Date Range:
- July 1, 2026 - December 31, 2026
- Indication:
- Potent panKRAS Inhibitor
Announcement
BridgeBio Oncology Therapeutics, Inc. announced that the Updated Phase 1 clinical data are expected in the second half of 2026
AI Summary
BridgeBio Oncology Therapeutics announced that updated Phase 1 clinical data for BBO-11818 are expected in the second half of 2026. BBO-11818 is being evaluated in the Phase 1 KONQUER-101 trial in subjects with locally advanced unresectable or metastatic KRAS‑mutant solid tumors. The company says the timing should give clinicians and researchers more detailed information on the agent’s early profile.
Early Phase 1 findings reported a confirmed partial response in pancreatic cancer with BBO-11818 monotherapy. Anti‑tumor activity was seen across dose levels and tumor types, with greater tumor reductions at higher doses and a generally favorable, differentiated safety profile during dose escalation. Responses across multiple tumor types suggest potential broader activity against KRAS‑mutant cancers.
The updated data due in late 2026 are expected to include longer follow‑up, more patients, and deeper analyses of response rates, duration of response, and adverse events. Those results should help guide dosing decisions, patient selection, and next steps in development.
Read Announcement- Drug:
- BBO-11818
- Announced Date:
- April 20, 2026
- Indication:
- Potent panKRAS Inhibitor
Announcement
BridgeBio Oncology Therapeutics, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to BBO-11818 for the treatment of adult patients with advanced KRAS-mutant pancreatic ductal adenocarcinoma.
AI Summary
BridgeBio Oncology Therapeutics, Inc. announced that the U.S. Food and Drug Administration has granted Fast Track designation to BBO-11818 for the treatment of adult patients with advanced KRAS‑mutant pancreatic ductal adenocarcinoma. The designation recognizes the potential of BBO-11818 to address a serious disease with limited treatment options.
BBO-11818 is being evaluated in the Phase 1 KONQUER-101 trial in patients with locally advanced unresectable or metastatic KRAS‑mutant solid tumors. Updated Phase 1 clinical data are expected in the second half of 2026. Preliminary results released in January 2026 showed a confirmed partial response in pancreatic cancer, anti‑tumor activity across dose levels and tumor types, tumor reductions at higher doses, and a generally favorable, differentiated safety profile during dose escalation.
Fast Track designation is intended to help speed the development and review of promising therapies for serious conditions that may fill unmet medical needs, potentially enabling more frequent interactions with the FDA and expedited review timelines.
Read Announcement- Drug:
- BBO-11818
- Announced Date:
- October 23, 2025
- Indication:
- Potent panKRAS Inhibitor
Announcement
BridgeBio Oncology Therapeutics, Inc. announced new preclinical data on BBO-11818 demonstrating its potential as a potent panKRAS inhibitor targeting mutant KRAS in both the ON (active GTP-bound) and OFF (inactive GDP-bound) states, with selectivity over HRAS and NRAS.
AI Summary
BridgeBio Oncology Therapeutics announced preclinical data on BBO-11818, a potent panKRAS inhibitor that binds mutant KRAS in both the ON (GTP-bound) and OFF (GDP-bound) states. It shows over 1,000-fold selectivity against NRAS, HRAS, and BRAF-mutant cell lines. In KRASG12D and KRASG12V mutant cell assays, BBO-11818 effectively suppressed MAPK signaling, blocked ERK phosphorylation, and inhibited cell growth at single-digit nanomolar concentrations.
In mouse xenograft models of pancreatic, non-small cell lung, and colorectal cancer, oral dosing produced significant tumor regressions with good pharmacokinetics. Combining BBO-11818 with BBOT’s RAS:PI3Kα breaker (BBO-10203) further decreased tumor cell proliferation and increased apoptosis. Enhanced anti-tumor activity was also seen with cetuximab or anti-PD-1 therapy, leading to complete regressions in a KRASG12D model.
These results highlight BBO-11818’s strong potential as both a standalone and combination treatment for KRAS-driven cancers.
Read Announcement
Acoramidis (ATTRibute-CM) FDA Regulatory Timeline and Events
Acoramidis (ATTRibute-CM) is a drug developed by BridgeBio Pharma for the following indication: Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM).
This drug is approved by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- March 23, 2026
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma, Inc. announced today that long-term efficacy and safety data in patients with ATTR-CM from the open-label extension (OLE) trial of ATTRibute-CM, its Phase 3 study of acoramidis, will be shared in a late-breaking oral presentation at the American College of Cardiology (ACC) Annual Scientific Sessions & Expo, taking place in New Orleans, Louisiana on March 28-30, 2026.
AI Summary
BridgeBio Pharma announced that long-term efficacy and safety results from the open-label extension of ATTRibute-CM, its Phase 3 study of acoramidis, will be presented in a late-breaking oral session at the American College of Cardiology Annual Scientific Sessions in New Orleans, March 28–30, 2026. The oral presentation, titled “Long-Term Survival Benefits and Disease Stabilization with Acoramidis in Patients with ATTR-CM,” will be delivered by Prem Soman, M.D., Ph.D., on Monday, March 30 at 2:33 pm CT.
Additional data will appear as posters: Sarah Cuddy, M.D., will present long-term effects on serum transthyretin and KCCQ-OS on Saturday, March 28 at 12:30 pm CT; Jan Griffin, M.D., will report on the link between early sTTR increases and long-term heart-failure health status on March 28 at 3:30 pm CT; and Jill Waldron will present U.S. treatment patterns and preferences on March 30 at 9:30 am CT.
These presentations will share extended safety and clinical outcome findings for acoramidis (Attruby), a transthyretin stabilizer used to treat ATTR-CM and reduce cardiovascular death and related hospitalizations.
Read Announcement- Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- January 12, 2026
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma, Inc. today provided updates on its commercial progress for Attruby (acoramidis), status of late-stage pipeline programs, and anticipated 2026 milestones
AI Summary
BridgeBio reported strong commercial traction for Attruby (acoramidis), with preliminary unaudited net product revenue of $146.0 million in Q4 2025 and $362.4 million for the full year. As of December 31, 2025, 6,629 unique patient prescriptions were written by 1,632 prescribers, and the company says Attruby is rapidly becoming a first-choice therapy for newly diagnosed ATTR-CM patients. BridgeBio had approximately $587.5 million in cash, cash equivalents, and marketable securities, which it says positions the company to sustain Attruby’s growth and potentially launch three additional medicines globally.
On the late-stage pipeline, BridgeBio announced a new TTR amyloid depleter antibody program expected to enter the clinic in 2027–2028. Interim FORTIFY results for BBP-418 in LGMD2I/R9 showed broad benefit and a 2.6‑point NSAD improvement at 12 months; the company plans to submit an NDA in the first half of 2026. For encaleret, more than 1,700 ADH1 patients have been identified and an NDA based on CALIBRATE is expected in H1 2026; RECLAIM‑HP phase 3 is planned to start in summer 2026. PROPEL 3 achieved LPLV with topline results expected by end of Q1 2026, and ACCEL 2/3 achieved LPI for its Phase 2 portion.
Read Announcement- Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- September 22, 2025
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma, Inc. announced today that one late breaking clinical trials oral presentation, one oral presentation, and three poster sessions will be shared at the Heart Failure Society of America (HFSA) Annual Scientific Meeting (ASM) 2025, taking place in Minneapolis, MN from September 26 - 29, 2025.
AI Summary
BridgeBio Pharma, Inc. announced it will share one late-breaking oral presentation, one oral presentation, and three poster sessions at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2025 in Minneapolis, MN, from September 26–29.
The late-breaking oral presentation, “Effect of Acoramidis on Recurrent and Cumulative Cardiovascular Outcomes in ATTR-CM,” will be delivered by Dr. Ahmad Masri on Sunday, September 28 at 10:15 a.m. CT.
An oral presentation by Dr. Lily Stern, “Continuous Acoramidis Treatment Significantly Reduced Risk of All-cause Mortality and Cardiovascular-related Hospitalization at Month 42,” is set for Saturday, September 27 at 1:33 p.m. CT.
Poster sessions include: “Acoramidis Mitigates the Rise in NT-proBNP Levels” by Dr. Nitasha Sarswat (Saturday 7:45 a.m. CT); “Effect of Acoramidis on Cardiac Conduction Abnormalities” by Dr. Brett Sperry (Saturday 1:21 p.m. CT); and “State-Level Differences in Incidence of ATTR-CM in US Veterans” by Dr. Sandesh Dev (Sunday 12:15 p.m. CT).
Read Announcement- Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- August 25, 2025
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma, Inc. announced today that one rapid-fire oral presentation on additional open-label extension data from ATTRibute-CM and two ePosters with ATTRibute-CM data at Month 30 will be shared at the European Society of Cardiology (ESC) Congress 2025, taking place in Madrid, Spain from August 29 - September 1, 2025.
AI Summary
BridgeBio Pharma, Inc. announced that it will share new long-term data on acoramidis at the 2025 European Society of Cardiology Congress in Madrid, Spain, from August 29 to September 1. A rapid-fire oral presentation on the open-label extension of the ATTRibute-CM study will show acoramidis’s impact on cardiovascular mortality at 42 months. Dr. Kevin Alexander from Stanford University will present on August 30 at 1:15 pm CEST (7:15 am ET).
Two ePosters will highlight Month 30 findings from ATTRibute-CM. Dr. Nitasha Sarswat of UChicago Medicine will discuss improvements in NT-proBNP compared with placebo on August 31 at 4:15 pm CEST (10:15 am ET). On August 29 at 3:15 pm CEST (9:15 am ET), Dr. Julian Gillmore of University College London’s Centre for Amyloidosis will present data on changes in NAC ATTR staging.
Read Announcement- Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- March 27, 2025
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma, Inc. announced the Japanese Ministry of Health, Labour and Welfare has approved acoramidis, under the brand name Beyonttra, for the treatment of adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM).
AI Summary
BridgeBio Pharma, Inc. announced that Japan’s Ministry of Health, Labour and Welfare has approved acoramidis, marketed as Beyonttra, for treating adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM). ATTR-CM is a progressive heart disease that leads to heart failure, and acoramidis works by stabilizing the transthyretin protein. In a Japanese Phase 3 study, patients treated with acoramidis showed 0% mortality over a 30‐month period, and the treatment was well-tolerated. The approval was further supported by positive findings from a global Phase 3 trial, which reported early benefits as soon as three months into treatment, along with significant reductions in the risk of all-cause mortality and cardiovascular hospitalizations. This milestone marks an important advancement for patients in Japan, offering a new treatment option for a severe and life-threatening condition, with commercialization efforts planned soon.
Read Announcement- Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- February 11, 2025
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma, Inc. announced the European Commission has granted marketing authorization in the European Union (EU) for acoramidis, under the brand name BEYONTTRA™, for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
AI Summary
BridgeBio Pharma, Inc. announced that the European Commission has granted marketing authorization in the European Union for its drug acoramidis, marketed as BEYONTTRA™, to treat adults with cardiomyopathy caused by wild-type or variant transthyretin amyloidosis (ATTR-CM). This approval is based on the positive results from the Phase 3 ATTRibute-CM study. In the study, acoramidis showed rapid benefits by significantly reducing the risk of all-cause mortality and cardiovascular-related hospitalizations compared to placebo. Patients experienced clear improvements in as little as three months, with a 42% reduction in severe events and a 50% drop in cardiovascular hospitalizations over 30 months. Bayer will lead the launch and all commercial activities in the EU, with the drug scheduled for release in the first half of 2025, providing a promising new treatment option for those affected by this progressive and life-threatening condition.
Read Announcement- Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- December 13, 2024
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma, Inc. announced today that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion recommending marketing authorization in the European Union (EU) for acoramidis for the treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).
AI Summary
BridgeBio Pharma, Inc. announced that the Committee for Medicinal Products for Human Use (CHMP) has given a positive opinion recommending marketing authorization in the European Union (EU) for acoramidis. This small molecule, taken orally, is designed to treat adult patients with wild-type or variant transthyretin amyloidosis with cardiomyopathy (ATTR-CM). The recommendation is based on encouraging Phase 3 ATTRibute-CM study results, where acoramidis demonstrated significant benefits in reducing cardiovascular-related hospitalizations and improving patient outcomes. The final decision is expected from the European Commission within the coming months. BridgeBio, which holds the U.S. rights, is partnering with Bayer, who will manage the European market launch. This collaboration leverages Bayer’s expertise in cardiovascular disease to bring an important new treatment option to patients suffering from this progressive and potentially fatal condition.
Read Announcement- Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- November 22, 2024
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma, Inc. announced that the U.S. Food and Drug Administration (FDA) approved Attruby™ (acoramidis), an orally-administered near-complete (≥90%) stabilizer of Transthyretin (TTR) for the treatment of adults with ATTR-CM to reduce cardiovascular death and cardiovascular-related hospitalization.
AI Summary
BridgeBio Pharma, Inc. announced that the FDA approved Attruby™ (acoramidis), a new oral treatment for adults with transthyretin-mediated cardiac amyloidosis (ATTR-CM). Attruby is unique as the first and only approved therapy that specifically stabilizes over 90% of the TTR protein. By doing so, it helps maintain TTR’s natural role of transporting thyroxine and vitamin A while significantly reducing risks of cardiovascular death and hospitalizations. In the Phase 3 ATTRibute-CM study, patients experienced benefits as early as three months, with a 42% reduction in a combined risk of death and hospitalizations, and a 50% reduction in cardiovascular-related hospital events over 30 months compared to placebo. BridgeBio also announced a commitment to offer Attruby free for life to patients who participated in the clinical trial, underlining its dedication to improving patient outcomes and hope in managing ATTR-CM.
Read Announcement- Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- September 30, 2024
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma, Inc presented a post-hoc analysis evaluating the effect of acoramidis on the composite endpoint of ACM and recurrent CVH events in its Phase 3 ATTRibute-CM study in ATTR-CM at the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2024.
AI Summary
At the HFSA Annual Scientific Meeting 2024, BridgeBio Pharma presented a post-hoc analysis from its Phase 3 ATTRibute-CM trial. The study evaluated acoramidis, a near-complete stabilizer of transthyretin (TTR), in patients with ATTR-CM. Results demonstrated that acoramidis reduced the composite endpoint of all-cause mortality (ACM) and recurrent cardiovascular-related hospitalization events by 42% compared to placebo at Month 30. An early and sustained improvement was observed, with event separation starting as early as Month 3.
The findings highlight the potential of acoramidis to improve clinical outcomes and overall quality of life for patients with ATTR-CM. By focusing on data transparency and detailed analysis, BridgeBio aims to support physicians in selecting effective treatment options. This analysis reinforces the promise of acoramidis as a meaningful therapy for managing the serious implications of ATTR-CM.
Read Announcement- Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- August 30, 2024
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma, IncBridgeBio Pharma, Inc that additional data on clinical outcomes from ATTRibute-CM, its Phase 3 study of acoramidis in ATTR-CM, will be presented at the European Society of Cardiology (ESC) Congress 2024, taking place in London, United Kingdom on August 30 – September 2, 2024 and the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2024, taking place in Atlanta, Georgia on September 27 - 30, 2024.
AI Summary
BridgeBio Pharma, Inc. announced that it will share fresh clinical outcomes data from its Phase 3 ATTRibute-CM study of acoramidis at two major scientific meetings in 2024. The company will present additional results at the European Society of Cardiology (ESC) Congress, held in London from August 30 to September 2, 2024. This session will focus on the observed increase in serum TTR levels with acoramidis treatment in patients with transthyretin amyloid cardiomyopathy (ATTR-CM) and will be moderated by Dr. Mathew S. Maurer from Columbia University Irving Medical Center.
Later, BridgeBio will discuss findings on improved clinical outcomes using a post hoc recurrent event analysis at the Heart Failure Society of America (HFSA) Annual Scientific Meeting in Atlanta from September 27 to 30, 2024. The session, led by Dr. Daniel P. Judge, promises important insights into the treatment benefits of acoramidis in this patient group.
Read Announcement- Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- August 29, 2024
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma, Inc. announced that additional data on clinical outcomes from ATTRibute-CM, its Phase 3 study of acoramidis in ATTR-CM, will be presented at the European Society of Cardiology (ESC) Congress 2024, taking place in London, United Kingdom on August 30 – September 2, 2024 and the Heart Failure Society of America (HFSA) Annual Scientific Meeting 2024, taking place in Atlanta, Georgia on September 27 - 30, 2024.
AI Summary
BridgeBio Pharma, Inc. announced that it will share additional clinical outcome data from its Phase 3 study, ATTRibute-CM, which examines the use of acoramidis in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). This new data will be presented at two key 2024 conferences. At the European Society of Cardiology (ESC) Congress 2024 in London, United Kingdom (August 30 – September 2), a moderated poster will discuss an increase in serum TTR levels observed with acoramidis treatment, presented by Dr. Mathew S. Maurer from Columbia University Irving Medical Center. In addition, at the Heart Failure Society of America (HFSA) Annual Scientific Meeting in Atlanta, Georgia (September 27–30), Dr. Daniel P. Judge from the Medical University of South Carolina will present an oral abstract on how acoramidis improves clinical outcomes, sharing further insights through a post hoc recurrent event analysis from ATTRibute-CM.
Read Announcement- Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- May 13, 2024
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma announced positive results of four new analyses from its Phase 3 ATTRibute-CM study of acoramidis in ATTR-CM at the European Society of Cardiology (ESC) Heart Failure Congress 2024. ATTRibute-CM was designed to study the efficacy and safety of acoramidis, an investigational, next-generation, orally-administered, highly potent, small molecule stabilizer of TTR.
AI Summary
At the ESC Heart Failure Congress 2024, BridgeBio Pharma shared positive results from four new analyses of its Phase 3 ATTRibute-CM study. The study focused on acoramidis, an investigational drug that is taken orally and is designed as a small molecule stabilizer of the TTR protein. This protein plays a key role in the development of ATTR-CM, a serious heart condition caused by transthyretin amyloidosis.
The new data from ATTRibute-CM highlights the efficacy and safety of acoramidis in treating patients with ATTR-CM. These promising results add to evidence that acoramidis could reduce the heart complications linked to this disease. BridgeBio Pharma believes that the findings are an important step forward in developing a treatment that could greatly improve the lives of patients suffering from this life-threatening condition.
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LGMD2I/R9 FDA Regulatory Events
LGMD2I/R9 is a drug developed by BridgeBio Pharma for the following indication: Monogenic autosomal recessive.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- LGMD2I/R9
- Announced Date:
- March 11, 2026
- Indication:
- Monogenic autosomal recessive
Announcement
BridgeBio Pharma, Inc. today presented additional positive data from the interim analysis of FORTIFY, the Phase 3 clinical trial of oral BBP-418, in individuals with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).
AI Summary
BridgeBio presented interim Phase 3 FORTIFY data showing oral BBP-418 produced broad, consistent benefits in limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). The company reported positive effects across key clinical endpoints and prespecified subgroups. BBP-418 separated from placebo on the 100-meter timed test (100MTT), with ambulation improvements evident as soon as three months.
BridgeBio plans to submit a new drug application (NDA) to the FDA in the first half of 2026 and expects a U.S. launch in late 2026 or early 2027 if approved. If successful, BBP-418 could be the first approved therapy for LGMD2I/R9 and may represent the first approval for any form of LGMD.
The interim analysis also included new efficacy and safety findings presented in a late‑breaking oral at the MDA conference, plus one additional oral presentation and four posters to further understanding of BBP-418 and the disease.
Read Announcement- Drug:
- LGMD2I/R9
- Announced Date:
- June 18, 2024
- Indication:
- Monogenic autosomal recessive
Announcement
BridgeBio Pharma, Inc. announced it has surpassed its interim analysis enrollment target and expects topline interim data from its Phase 3 registrational study (FORTIFY) in individuals with LGMD2I/R9 in 2025.
AI Summary
BridgeBio Pharma, Inc. has reached its interim analysis enrollment target for its Phase 3 FORTIFY study, a major step in its research for treating LGMD2I/R9 with the investigational oral therapy BBP-418. The FORTIFY study is a randomized, double-blind, placebo-controlled trial designed to evaluate the safety and effectiveness of BBP-418 in individuals with this rare and debilitating genetic disease.
The company expects to release top-line interim data from the study in 2025. This milestone is important as the interim analysis, planned at 12 months, will use a validated measure of glycosylated alpha-dystroglycan as a potential surrogate endpoint to support Accelerated Approval. BridgeBio’s progress continues as it enrolls participants across multiple regions including the U.S., UK, Europe, and Australia.
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BBP-418 FDA Regulatory Timeline and Events
BBP-418 is a drug developed by BridgeBio Pharma for the following indication: Limb Girdle Muscular Dystrophy Type 2i (LGMD2i).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- BBP-418
- Announced Date:
- March 4, 2026
- Indication:
- Limb Girdle Muscular Dystrophy Type 2i (LGMD2i)
Announcement
BridgeBio Pharma, Inc. announced today that additional data from the interim analysis of FORTIFY, the Phase 3 clinical trial of BBP-418 in patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), will be shared in a late-breaking oral presentation at the MDA Clinical and Scientific Conference, taking place in Orlando, Florida on March 8-11, 2026.
AI Summary
BridgeBio Pharma announced that additional data from the interim analysis of FORTIFY, its Phase 3 trial of BBP-418 for patients with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9), will be presented as a late-breaking oral presentation at the Muscular Dystrophy Association (MDA) Clinical and Scientific Conference. The company says the interim analysis met key efficacy endpoints, and the new data will provide more detail on those results and on BBP-418’s potential benefit for people with LGMD2I/R9.
The late-breaking oral presentation is scheduled for Wednesday, March 11 at 2:00 pm ET in Orlando, Florida, during the March 8–11, 2026 conference. The presenter is Katherine Mathews, M.D., Professor of Pediatrics and Neurology at the University of Iowa’s Roy J. and Lucille A. Carver College of Medicine. The data are interim and will be discussed at the session.
Read Announcement- Drug:
- BBP-418
- Announced Date:
- October 27, 2025
- Indication:
- Limb Girdle Muscular Dystrophy Type 2i (LGMD2i)
Announcement
BridgeBio Pharma, Inc. today reports positive topline results from FORTIFY, the Company's Phase 3 pivotal study of BBP-418 in individuals living with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9).
AI Summary
BridgeBio Pharma today reported positive topline results from FORTIFY, its randomized, double-blind, placebo-controlled Phase 3 study of BBP-418 in people with limb-girdle muscular dystrophy type 2I/R9 (LGMD2I/R9). The trial met all primary and secondary interim analysis endpoints with a safety profile consistent with earlier studies.
The primary endpoint, glycosylated alpha-dystroglycan (αDG), increased 1.8-fold from baseline in BBP-418–treated participants at three months (p < 0.0001) and remained significantly improved at 12 months versus placebo (p < 0.0001).
At 12 months, treated individuals showed an 82% reduction in serum creatine kinase, a marker of muscle damage (p < 0.0001). They also experienced a 0.14 m/s gain in walking speed (100MTT) from baseline and a 0.27 m/s advantage over placebo (p < 0.0001), plus a ~3% rise in forced vital capacity from baseline and ~5% over placebo (p = 0.0071). BBP-418 was well tolerated with no unexpected safety issues.
BridgeBio plans to submit a New Drug Application to the FDA in the first half of 2026.
Read Announcement- Drug:
- BBP-418
- Announced Date:
- October 24, 2025
- Target Action Date:
- October 27, 2025
- Indication:
- Limb Girdle Muscular Dystrophy Type 2i (LGMD2i)
Announcement
BridgeBio Pharma, Inc. to Report Phase 3 Results for Small Molecule BBP-418 in LGMD2I/R9 FORTIFY Study
AI Summary
BridgeBio Pharma will report interim Phase 3 topline results for BBP-418, its small molecule therapy in the FORTIFY study for Limb-girdle Muscular Dystrophy Type 2I/R9 (LGMD2I/R9). Management will host a webinar on October 27, 2025, at 8:00 am ET to share these results, followed by a Q3 2025 earnings call on October 29 at 4:30 pm ET.
Investors can join the live webcasts through the “Events and Presentations” page on BridgeBio’s website. Replays of both sessions will be available for 90 days after the events.
BBP-418 is designed to improve muscle strength and slow disease progression in LGMD2I/R9, a genetic condition caused by FKRP mutations. Positive interim data could support full data release and discussions with regulators.
Founded in 2015, BridgeBio focuses on discovering and developing transformative genetic medicines, advancing programs from early research through clinical trials.
Read Announcement- Drug:
- BBP-418
- Announced Date:
- September 30, 2024
- Indication:
- Limb Girdle Muscular Dystrophy Type 2i (LGMD2i)
Announcement
BridgeBio Pharma, Inc announced, on LGMD Awareness Day, the completion of enrollment of FORTIFY, the Company's Phase 3 registrational study of BBP-418 in individuals with LGMD2I/R9..
AI Summary
On LGMD Awareness Day, BridgeBio Pharma announced that enrollment in its Phase 3 FORTIFY study for BBP-418 has been completed ahead of target. The trial, which tests BBP-418 in individuals with LGMD2I/R9, is a randomized, double-blind, placebo-controlled study designed to evaluate both the safety and efficacy of the investigational oral therapy. A key goal of the study is a planned interim analysis at 12 months, which will assess a potential biomarker surrogate endpoint—glycosylated alpha-dystroglycan (αDG). BridgeBio believes that positive findings from this analysis could support an Accelerated Approval pathway for BBP-418 in the U.S., potentially making it the first approved treatment for LGMD2I/R9. Topline data from the interim analysis is expected to be released in 2025, bringing new hope to patients awaiting effective treatment options.
Read Announcement- Drug:
- BBP-418
- Announced Date:
- June 18, 2024
- Indication:
- Limb Girdle Muscular Dystrophy Type 2i (LGMD2i)
Announcement
BridgeBio Pharma, Inc. announced it has surpassed its interim analysis enrollment target and expects topline interim data from its Phase 3 registrational study (FORTIFY) in individuals with LGMD2I/R9 in 2025.
AI Summary
BridgeBio Pharma, Inc. announced that it has surpassed its interim enrollment target for the Phase 3 FORTIFY study evaluating BBP-418 in individuals with LGMD2I/R9, a rare and serious genetic disorder that primarily affects muscle function. The FORTIFY study is a randomized, double-blind, placebo-controlled trial designed to assess both the safety and efficacy of BBP-418. With a planned interim analysis at 12 months focused on glycosylated alpha-dystroglycan levels—a key molecular marker—the company expects to release top-line interim results in 2025. These results could support the pursuit of Accelerated Approval by the FDA, offering hope for a faster path to providing a much-needed treatment for patients living with this debilitating condition. The study is actively enrolling participants across the U.S., UK, Europe, and Australia.
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Infigratinib PROPEL3 FDA Regulatory Events
Infigratinib PROPEL3 is a drug developed by BridgeBio Pharma for the following indication: Infigratinib in Children with Achondroplasia.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Infigratinib PROPEL3
- Announced Date:
- February 12, 2026
- Indication:
- Infigratinib in Children with Achondroplasia
Announcement
BridgeBio Pharma, Inc. announced positive topline results from PROPEL 3, the global Phase 3 pivotal study of oral infigratinib in children living with achondroplasia. BridgeBio will host an investor call on February 12, 2026 at 8:00 am ET to discuss these results.
AI Summary
BridgeBio announced positive topline results from PROPEL 3, a global Phase 3 trial of oral infigratinib in children with achondroplasia. The study met its primary endpoint: change from baseline in annualized height velocity (AHV) at Week 52 (p<0.0001). The mean treatment difference versus placebo was +2.10 cm/year; the least-squares (LS) mean treatment difference was +1.74 cm/year.
In a pre-specified exploratory analysis of children younger than 8 years (over 50% of participants), infigratinib produced a statistically significant improvement in body proportionality versus placebo (LS mean difference -0.05, p<0.05). The key secondary endpoint—change in height Z-score at Week 52—was met (p<0.0001) with an LS mean increase of +0.41 SD. The drug was well tolerated: no drug-related serious adverse events or discontinuations; three mild, transient cases (4%) of hyperphosphatemia without dose changes; and no retinal or corneal events linked to FGFR1/2 inhibition.
PROPEL 3 was a one-year, global, randomized 2:1 double-blind placebo-controlled study in children aged 3 to under 18 years with open growth plates. BridgeBio plans NDA and MAA submissions in the second half of 2026, will accelerate development in hypochondroplasia, and is enrolling the observational run-in for the Phase 3 trial. BridgeBio will hold an investor call on February 12, 2026 at 8:00 am ET to discuss the results.
Read Announcement- Drug:
- Infigratinib PROPEL3
- Announced Date:
- September 17, 2024
- Indication:
- Infigratinib in Children with Achondroplasia
Announcement
BridgeBio Pharma, Inc. announced that the FDA has granted Breakthrough Therapy Designation to oral infigratinib under development for children with achondroplasia.
AI Summary
BridgeBio Pharma, Inc. recently announced that the FDA has granted Breakthrough Therapy Designation to its oral drug infigratinib, now under development for children with achondroplasia. This special status is based on promising preliminary data from the Phase 2 PROPEL 2 trial. In one study cohort, children treated with infigratinib showed a statistically significant increase in their annualized height velocity—gaining about 2.5 centimeters per year at both 12 and 18 months. Additionally, the data demonstrated significant improvements in body proportionality, which is an important factor for overall health. The Breakthrough Therapy Designation will allow BridgeBio to work more closely with the FDA, expediting the development and regulatory review process. If approved, infigratinib could become the first oral treatment option for children with achondroplasia, offering families a new and promising therapeutic approach.
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Autosomal dominant hypocalcemia type 1 FDA Regulatory Events
Autosomal dominant hypocalcemia type 1 is a drug developed by BridgeBio Pharma for the following indication: calcium‐sensing receptor gene (CASR).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- autosomal dominant hypocalcemia type 1
- Announced Date:
- October 28, 2025
- Indication:
- calcium‐sensing receptor gene (CASR).
Announcement
BridgeBio Pharma announced plans to release topline results of the autosomal dominant hypocalcemia type 1 (ADH1) CALIBRATE Phase 3 trial before the market opens on Wednesday, October 28, 2025.
AI Summary
BridgeBio Pharma, a biopharmaceutical company dedicated to genetic diseases, said it will release the topline results of its autosomal dominant hypocalcemia type 1 (ADH1) CALIBRATE Phase 3 trial before the market opens on Wednesday, October 28, 2025. These results are expected to provide key insights into the safety and efficacy of the investigational therapy in patients with ADH1.
The company’s management team will host a conference call at 8:00 a.m. ET the same day to review the data. Investors and analysts can access the live webcast on the “Events and Presentations” page of the BridgeBio website. A replay will be available online for 90 days following the event.
Founded in 2015, BridgeBio focuses on discovering, testing, and delivering transformative medicines for genetic diseases. Its pipeline spans from early research programs to advanced clinical trials, with a goal of bringing genetic treatments to patients as quickly as possible. For more information, visit bridgebio.com.
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Infigratinib (PROPEL 2) FDA Regulatory Events
Infigratinib (PROPEL 2) is a drug developed by BridgeBio Pharma for the following indication: Achondroplasia (ACH).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- Infigratinib (PROPEL 2)
- Announced Date:
- November 18, 2024
- Indication:
- Achondroplasia (ACH)
Announcement
BridgeBio Pharma, Inc. announced that positive 18-month results from PROPEL 2, a Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia, were published as an original research article in the New England Journal of Medicine (NEJM) today
AI Summary
BridgeBio Pharma recently announced that positive 18‑month results from its Phase 2 PROPEL 2 trial of infigratinib in children with achondroplasia have been published as an original research article in the New England Journal of Medicine. In this trial, children in Cohort 5 who received a daily oral dose of 0.25 mg/kg of infigratinib demonstrated a statistically significant increase in annualized height velocity (AHV), with a mean change from baseline of +2.50 cm/year at Month 18. These findings also revealed notable improvements in height Z‑score and body proportionality. The encouraging data highlight both the potential effectiveness and the favorable safety profile of infigratinib, an investigational oral small molecule designed to inhibit FGFR3 signaling. These results underscore the promise of infigratinib as a best‑in‑class treatment option for enhancing growth and function in children with achondroplasia.
Read Announcement- Drug:
- Infigratinib (PROPEL 2)
- Announced Date:
- June 4, 2024
- Indication:
- Achondroplasia (ACH)
Announcement
BridgeBio Pharma, Inc. announced sustained positive results from PROPEL 2, a Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia, demonstrating continued potential best-in-class efficacy and an encouraging safety profile.
AI Summary
BridgeBio Pharma has announced sustained positive results from its Phase 2 PROPEL 2 trial evaluating infigratinib, an oral investigational therapy for children with achondroplasia. The study demonstrated a sustained and statistically significant increase in annualized height velocity, with a +2.51 cm/year increase at Month 12 and +2.50 cm/year at Month 18 (p=0.0015). In addition, there was a significant improvement in body proportionality; the mean upper to lower body segment ratio improved from 2.02 at baseline to 1.88 at Month 18 (p=0.001). Importantly, infigratinib was well-tolerated with no treatment-related adverse events observed in this cohort, supporting its potential as a best-in-class therapy. These encouraging efficacy and safety results point to meaningful benefits for children with achondroplasia, as the company continues to expand its development programs to address unmet needs in this patient population.
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BBP-631 FDA Regulatory Events
BBP-631 is a drug developed by BridgeBio Pharma for the following indication: Congenital Adrenal Hyperplasia (CAH).
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- BBP-631
- Announced Date:
- September 10, 2024
- Indication:
- Congenital Adrenal Hyperplasia (CAH)
Announcement
BridgeBio Pharma, Inc. announced topline results from the Phase 1/2 open-label ADventure study investigating BBP-631, the Company's investigational adeno-associated virus (AAV) 5 gene therapy, for the treatment of congenital adrenal hyperplasia (CAH).
AI Summary
BridgeBio Pharma, Inc. announced positive topline results from its Phase 1/2 open-label ADventure study investigating BBP-631, an investigational adeno-associated virus (AAV) 5 gene therapy for congenital adrenal hyperplasia (CAH). In the study, researchers evaluated the safety, tolerability, and early signs of effectiveness of BBP-631 in patients with CAH. The preliminary data shows that the gene therapy may help address some of the key challenges associated with CAH by targeting the genetic root of the condition. This innovative approach aims to provide a long-term solution by delivering a functional copy of the necessary gene, potentially improving hormonal balance and reducing related complications. These topline results are an encouraging step forward for BBP-631, and they support further clinical development to better understand the therapy’s potential benefits for patients suffering from this challenging endocrine disorder.
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BBP-812 FDA Regulatory Events
BBP-812 is a drug developed by BridgeBio Pharma for the following indication: Canavan disease.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- BBP-812
- Announced Date:
- September 10, 2024
- Indication:
- Canavan disease
Announcement
BridgeBio Pharma, Inc. announced that the United States Food and Drug Administration (FDA) has granted Regenerative Medicine Advanced Therapy (RMAT) designation to BBP-812, an investigational intravenous (IV) adeno-associated virus serotype 9 (AAV9) gene therapy for the treatment of Canavan disease.
AI Summary
BridgeBio Pharma recently announced that the FDA has granted Regenerative Medicine Advanced Therapy (RMAT) designation to its investigational gene therapy, BBP-812. This intravenous treatment, using an adeno-associated virus serotype 9 (AAV9), is being developed to treat Canavan disease, a rare and fatal neurodegenerative disorder that affects young children. The RMAT designation speeds up development by allowing BridgeBio earlier and more frequent interactions with the FDA, which could help pave the way for an accelerated approval process.
The decision was based on preliminary clinical data from the CANaspire Phase 1/2 trial. In this trial, patients receiving BBP-812 showed functional improvements, suggesting that the therapy may address critical needs for those with Canavan disease. BridgeBio’s leadership expressed gratitude to the trial participants, reinforcing their commitment to bringing this potentially life-changing treatment to affected families as quickly as possible.
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