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
Acoramidis, BBP-418, BBP-631, BBP-812, Infigratinib, Infigratinib, and LGMD2I/R9. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
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 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.
Read Announcement- Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- April 7, 2024
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma, Inc announced that Marketing Authorization Application (MAA) for acoramidis has been accepted by the European Medicines Agency (EMA), with an expected decision in 2025.
AI Summary
BridgeBio Pharma, Inc. announced that its Marketing Authorization Application (MAA) for acoramidis has been accepted by the European Medicines Agency (EMA). The EMA is expected to issue a decision on the application in 2025, marking an important milestone for the company as it works to bring this new treatment to patients with transthyretin amyloid cardiomyopathy (ATTR-CM).
Acoramidis is designed to stabilize the transthyretin protein nearly completely, which may help improve the structure and function of the heart. The decision from the EMA comes on the heels of positive results from the ATTRibute-CM Phase 3 trial that indicated potential cardiac improvements and even regression of cardiac amyloid in some patients. BridgeBio’s work with acoramidis reflects its commitment to developing transformative medicines for genetic diseases, and the upcoming EMA decision in 2025 is a key regulatory step in advancing this promising therapy.
Read Announcement- Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- April 7, 2024
- Estimated Event Date Range:
- November 29, 2024 - November 29, 2024
- Target Action Date:
- November 29, 2024
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma, Inc announced that BridgeBio's New Drug Application (NDA) has been accepted by the U.S. Food and Drug Administration (FDA) with a PDUFA action date of November 29, 2024
AI Summary
BridgeBio Pharma, Inc. announced that its New Drug Application (NDA) for acoramidis has been accepted by the U.S. Food and Drug Administration (FDA). The FDA set a Prescription Drug User Fee Act (PDUFA) action date of November 29, 2024, marking an important step forward for the company. Acoramidis is an investigational, orally administered drug designed to stabilize transthyretin (TTR) in patients with ATTR-CM, a condition that affects heart structure and function. Preliminary studies have shown that the drug may improve cardiac performance and potentially lead to regression of amyloid deposits in the heart. In addition to the U.S. approval process, BridgeBio’s Marketing Authorization Application (MAA) has been accepted by the European Medicines Agency, with a decision expected in 2025. These regulatory milestones support BridgeBio’s commitment to advancing new treatment options for patients with ATTR-CM.
Read Announcement - Drug:
- Acoramidis (ATTRibute-CM)
- Announced Date:
- April 7, 2024
- Indication:
- Symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM)
Announcement
BridgeBio Pharma, Inc. today presented results from the exploratory CMR imaging substudy of ATTRibute-CM, its Phase 3 trial of acoramidis in ATTR-CM.
AI Summary
BridgeBio Pharma, Inc. shared promising findings from an exploratory CMR imaging substudy of its Phase 3 ATTRibute-CM trial for acoramidis in patients with transthyretin amyloid cardiomyopathy (ATTR-CM). The study showed that patients treated with acoramidis experienced potential improvements in both cardiac structure and function compared to those who received a placebo. Specifically, measurements like the left ventricular mass remained stable or even improved, and cardiac function metrics such as the ejection fraction and stroke volume showed positive trends. These results suggest that near-complete transthyretin stabilization with acoramidis may lead to cardiac remodeling and even regression of amyloid deposits—a significant finding since this is the first prospective, longitudinal CMR imaging evaluation in a double-blind, placebo-controlled study for ATTR-CM.
Read Announcement
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:
- 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.
Read Announcement
BBP-631 - FDA Regulatory Timeline and 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.
Read Announcement
BBP-812 - FDA Regulatory Timeline and 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.
Read Announcement
Infigratinib (PROPEL 2) - FDA Regulatory Timeline and 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.
Read Announcement
Infigratinib PROPEL3 - FDA Regulatory Timeline and 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:
- 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.
Read Announcement
LGMD2I/R9 - FDA Regulatory Timeline and 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:
- 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.
Read Announcement
Free BBIO Stock Alerts