Ionis Pharmaceuticals (IONS) has upcoming FDA regulatory milestones for Zilganersen and ION582. 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 Ionis Pharmaceuticals (IONS).
Over the past two years, Ionis Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
Pelacarsen, Zilganersen, Olezarsen, ION337, Eplontersen, ION582, and BIIB080/IONIS-MAPT. 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.
Pelacarsen FDA Regulatory Events
Pelacarsen is a drug developed by Ionis Pharmaceuticals for the following indication: Cardiovascular disease and elevated levels of Lp(a).
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:
- Pelacarsen
- Announced Date:
- September 4, 2026
- Indication:
- Cardiovascular disease and elevated levels of Lp(a)
Announcement
Royalty Pharma plc announced an update on Novartis’ Phase 3 results from the HORIZON outcomes trial for pelacarsen, an antisense oligonucleotide studied in patients with atherosclerotic cardiovascular (CV) disease and elevated lipoprotein(a), or Lp(a).
Read Announcement
Zilganersen FDA Regulatory Timeline and Events
Zilganersen is a drug developed by Ionis Pharmaceuticals for the following indication: for the treatment of children and adults Alexander disease (AxD).
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:
- Zilganersen
- Announced Date:
- September 3, 2026
- Indication:
- for the treatment of children and adults Alexander disease (AxD).
Announcement
Ionis Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved ZANVASTRO™ (zilganersen) for the treatment of Alexander disease (AxD) in pediatric and adult patients. ZANVASTRO is the first and only disease modifying treatment for AxD, an ultra-rare, progressive and often fatal neurological disorder that can affect motor, cognitive, autonomic and gastrointestinal function.
Read Announcement- Drug:
- Zilganersen
- Announced Date:
- April 21, 2026
- Indication:
- for the treatment of children and adults Alexander disease (AxD).
Announcement
Ionis Pharmaceuticals, Inc. announced additional positive results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive and often fatal neurological condition with no approved disease-modifying treatments.
AI Summary
Ionis Pharmaceuticals reported additional positive pivotal study results for zilganersen in children and adults with Alexander disease (AxD), a rare, progressive and often fatal neurological disorder with no approved disease‑modifying treatments. The data suggest potential benefit across multiple AxD symptom domains and on patients’ overall health.
Key secondary measures favored zilganersen: 32% of treated patients rated their most bothersome symptom as “much better” versus 0% on control, and only 5% rated it “much worse” versus 31% on control. On global measures, 83% of zilganersen patients reported improvement or no change in disease severity (vs. 76% control), and 21% felt “much better” overall (vs. 0% control). Additional endpoints showed consistent signals for improvements in communication, swallowing, gastrointestinal and autonomic symptoms.
An exploratory analysis found a 33.6% reduction in plasma GFAP at Week 61 (nominal p=0.003). Zilganersen’s safety profile was favorable, with most adverse events mild or moderate and fewer serious events than control (37.5% vs. 47.1%). A PDUFA action date is set for September 22, 2026.
Read Announcement- Drug:
- Zilganersen
- Announced Date:
- March 23, 2026
- Target Action Date:
- September 22, 2026
- Indication:
- for the treatment of children and adults Alexander disease (AxD).
Announcement
Ionis Pharmaceuticals, Inc announced that The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of September 22, 2026.
AI Summary
Ionis Pharmaceuticals announced that the U.S. Food and Drug Administration has set a Prescription Drug User Fee Act (PDUFA) target action date of September 22, 2026, for its investigational therapy zilganersen. This date is the FDA’s target for completing its review and making a regulatory decision on the application for zilganersen.
Zilganersen is an investigational antisense medicine being developed to treat Alexander disease (AxD), a rare, progressive neurological disorder caused by mutations in the GFAP gene. People with AxD can have motor, speech, and swallowing problems, seizures, and developmental delays, and treatment options are limited.
Ionis Neurology leads the company’s work on therapies for rare neurological diseases. The PDUFA date gives physicians, patients, and investors a clear regulatory timeline as Ionis awaits the FDA’s review and potential next steps. Outcomes will depend on the FDA review process and may change.
Read Announcement- Drug:
- Zilganersen
- Announced Date:
- March 23, 2026
- Indication:
- for the treatment of children and adults Alexander disease (AxD).
Announcement
Ionis Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the New Drug Application (NDA) for zilganersen, an investigational RNA-targeted medicine for Alexander disease (AxD), a rare, progressive and often fatal neurological condition.
AI Summary
Ionis Pharmaceuticals announced that the U.S. Food and Drug Administration has accepted for Priority Review its New Drug Application for zilganersen, an investigational RNA-targeted medicine for Alexander disease (AxD). The agency set a PDUFA date of September 22, 2026, when it must complete its review.
Priority Review means the FDA will review the application on an accelerated timeline compared with standard reviews. Acceptance of the NDA marks a key regulatory step that allows the agency to evaluate clinical and safety data and decide whether the drug can be approved for use.
Zilganersen is being developed as an RNA-targeted therapy aimed at addressing the underlying biology of AxD, a rare, progressive and often fatal neurological disorder. If approved, the medicine could become a treatment option for patients with this severe disease and reflects Ionis’s focus on therapies for neurological conditions.
Read Announcement- Drug:
- Zilganersen
- Announced Date:
- December 2, 2025
- Target Action Date:
- Q1 2026
- Estimated Target Date Range:
- January 1, 2026 - March 31, 2026
- Indication:
- for the treatment of children and adults Alexander disease (AxD).
Announcement
Ionis Pharmaceuticals, Inc announced that On track to submit new drug application (NDA) in Q1 2026 –
AI Summary
Ionis Pharmaceuticals said it is on track to submit a new drug application (NDA) for zilganersen in Q1 2026 and is working to start an Expanded Access Program (EAP) in the U.S. Zilganersen is an investigational antisense oligonucleotide designed to reduce excess GFAP protein caused by disease‑causing variants in the GFAP gene. Ionis plans to continue working with regulators to make the medicine available to people with Alexander disease.
Topline results from the pivotal Phase 1–3 study showed zilganersen 50 mg stabilized gait speed on the 10‑Meter Walk Test at week 61 versus control (mean difference 33.3%, p=0.0412) with favorable safety and tolerability and consistent benefits on key secondary measures. The global trial enrolled 54 participants aged 1.5–53 years, mostly children, randomized 2:1 with dosing every 12 weeks and included long‑term open‑label follow‑up. Alexander disease is a rare, progressive neurological disorder with no approved disease‑modifying treatments.
Read Announcement- Drug:
- Zilganersen
- Announced Date:
- December 2, 2025
- Indication:
- for the treatment of children and adults Alexander disease (AxD).
Announcement
Ionis Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to zilganersen for the treatment of Alexander disease (AxD), a rare, progressive and often fatal neurological condition.
AI Summary
Ionis Pharmaceuticals announced the FDA has granted Breakthrough Therapy designation to zilganersen for Alexander disease (AxD), a rare, progressive and often fatal neurological disorder that can cause loss of mobility and problems with walking, speaking, swallowing and breathing. There are currently no approved disease‑modifying treatments for AxD, and the designation is intended to speed review of therapies that show substantial potential benefit.
The designation is supported by topline results from a global pivotal trial in children and adults. Zilganersen 50 mg, dosed every 12 weeks, produced a statistically significant and clinically meaningful stabilization in gait speed on the 10‑Meter Walk Test at week 61 versus control (mean difference 33.3%, p=0.0412). The medicine showed favorable safety and tolerability and consistent benefit on key secondary measures.
Ionis plans to submit a new drug application in Q1 2026 and is working to initiate an Expanded Access Program in the U.S., aiming to make this potential first disease‑modifying treatment available to patients in urgent need.
Read Announcement- Drug:
- Zilganersen
- Announced Date:
- September 22, 2025
- Target Action Date:
- Q1 2026
- Estimated Target Date Range:
- January 1, 2026 - March 31, 2026
- Indication:
- for the treatment of children and adults Alexander disease (AxD).
Announcement
Ionis Pharmaceuticals, Inc announced that NDA submission planned in Q1 2026
AI Summary
Ionis Pharmaceuticals announced positive topline results from a pivotal study of zilganersen in children and adults with Alexander disease, a rare and often fatal neurological condition. This is the first investigational medicine to show a meaningful disease-modifying effect in this disorder.
In the trial, zilganersen 50 mg led to a 33.3% improvement in walking speed at week 61 compared to control, meeting the primary goal with a statistically significant result. Participants also showed consistent benefits in measures like symptom severity and global impressions, and most side effects were mild or moderate.
Ionis plans to submit a new drug application to the U.S. Food and Drug Administration in Q1 2026. The company is also evaluating an Expanded Access Program to provide early treatment options, with detailed data set to be presented at an upcoming medical conference.
Read Announcement- Drug:
- Zilganersen
- Announced Date:
- September 22, 2025
- Indication:
- for the treatment of children and adults Alexander disease (AxD).
Announcement
Ionis Pharmaceuticals, Inc announced positive topline results from the pivotal study of zilganersen in children and adults living with Alexander disease (AxD), a rare, progressive and often fatal neurological condition with no approved disease-modifying treatments.
AI Summary
Ionis announced positive topline results from the pivotal study of zilganersen in children and adults with Alexander disease (AxD), a rare progressive and often fatal neurological disorder without approved disease-modifying treatments.
Zilganersen 50 mg showed a 33.3% stabilization in gait speed on the 10-Meter Walk Test versus control at week 61 (p=0.0412) and had a favorable safety profile.
It also met key secondary endpoints, showing slowed progression or improvement in Most Bothersome Symptom, Patient Global Impression of Change and Clinician Global Impression scores.
These data mark the first time an investigational drug has shown a disease-modifying effect in AxD, offering hope for preserving walking, speaking and swallowing functions.
Ionis plans to submit a New Drug Application to the FDA in Q1 2026 and is evaluating an Expanded Access Program, with full results to be presented at medical conferences.
Read Announcement- Drug:
- Zilganersen
- Announced Date:
- October 1, 2024
- Indication:
- for the treatment of children and adults Alexander disease (AxD).
Announcement
Ionis Pharmaceuticals, announced that the U.S. Food and Drug Administration (FDA) has granted zilganersen Fast Track designation for the treatment of children and adults with an ultra-rare, progressive and ultimately fatal neurological disorder known as Alexander disease (AxD).
AI Summary
Ionis Pharmaceuticals has received Fast Track designation from the U.S. Food and Drug Administration (FDA) for zilganersen, an investigational medicine aimed at treating Alexander disease (AxD) in both children and adults. AxD is an ultra-rare, progressive neurological disorder that currently has no approved treatment and often leads to fatal outcomes. Zilganersen is designed to target the underlying process that causes harmful protein accumulation in brain cells, which contributes to the disease. By granting Fast Track designation, the FDA will help expedite the development and review of this promising treatment, addressing a critical unmet medical need for patients suffering from AxD. The pivotal Phase 1-3 study, conducted across 13 sites in eight countries, has already completed enrollment, and topline data is expected to be released in the second half of 2025.
Read Announcement
Olezarsen (Formerly IONIS-APOCIII-L) FDA Regulatory Timeline and Events
Olezarsen (Formerly IONIS-APOCIII-L) is a drug developed by Ionis Pharmaceuticals for the following indication: Severe hypertriglyceridemia.
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:
- Olezarsen (Formerly IONIS-APOCIII-L)
- Announced Date:
- August 20, 2026
- Indication:
- Severe hypertriglyceridemia
Announcement
Ionis Pharmaceuticals, Inc announced that new data in the management of severe hypertriglyceridemia (sHTG) will be presented at the European Society of Cardiology (ESC) Congress 2026, taking place August 28 – 31 in Munich.
Read Announcement- Drug:
- Olezarsen (Formerly IONIS-APOCIII-L)
- Announced Date:
- June 24, 2026
- Indication:
- Severe hypertriglyceridemia
Announcement
Ionis Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved TRYNGOLZA® (olezarsen) as an adjunct to diet to reduce triglycerides (TG) and the risk of acute pancreatitis in adults with severe hypertriglyceridemia (sHTG: TG greater than or equal to 500 mg/dL). TRYNGOLZA is available in a 50 mg or 80 mg dose and is self-administered once monthly via an autoinjector.
AI Summary
Ionis Pharmaceuticals said the U.S. Food and Drug Administration has approved TRYNGOLZA (olezarsen) for adults with severe hypertriglyceridemia, or sHTG, as an add-on to diet. The treatment is meant to lower triglyceride levels and reduce the risk of acute pancreatitis in patients with triglycerides of 500 mg/dL or higher. Ionis says TRYNGOLZA is the only treatment for sHTG specifically approved to help reduce pancreatitis risk.
TRYNGOLZA will come in 50 mg and 80 mg doses and is given once a month with a self-injected autoinjector. The company said the drug showed strong triglyceride lowering in its clinical program and had a favorable safety profile overall. The most common side effects in sHTG patients were injection site reactions and higher liver enzyme levels. Ionis expects TRYNGOLZA to be available in the U.S. in July.
Read Announcement- Drug:
- Olezarsen (Formerly IONIS-APOCIII-L)
- Announced Date:
- February 26, 2026
- Indication:
- Severe hypertriglyceridemia
Announcement
Ionis Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental New Drug Application (sNDA) for olezarsen for severe hypertriglyceridemia (sHTG). The FDA has set a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2026.
AI Summary
Ionis Pharmaceuticals announced that the U.S. Food and Drug Administration has accepted a supplemental New Drug Application (sNDA) for olezarsen to treat severe hypertriglyceridemia (sHTG). The application was granted Priority Review, and the FDA set a Prescription Drug User Fee Act (PDUFA) target action date of June 30, 2026.
Olezarsen is an investigational RNA-targeted medicine designed to reduce the body’s production of apoC-III, a liver protein that helps control triglyceride levels in the blood. By lowering apoC-III, olezarsen aims to reduce triglycerides and potentially lower the risk of complications linked to very high triglyceride levels.
The Priority Review designation means the FDA will review the sNDA on an accelerated timeline. Ionis and stakeholders will await the agency’s decision by the June 30, 2026 PDUFA date, which will determine whether olezarsen can move forward for this indication.Read Announcement
- Drug:
- Olezarsen (Formerly IONIS-APOCIII-L)
- Announced Date:
- December 1, 2025
- Indication:
- Severe hypertriglyceridemia
Announcement
Ionis Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to olezarsen as an adjunct to diet to reduce triglyceride (TG) levels in adults with severe hypertriglyceridemia (sHTG) (TG greater than or equal to 500 mg/dL).
AI Summary
Ionis Pharmaceuticals announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation to olezarsen as an adjunct to diet to reduce triglyceride (TG) levels in adults with severe hypertriglyceridemia (sHTG) (TG ≥500 mg/dL). Breakthrough designation is intended to speed the review of treatments for serious conditions that show early evidence of substantial improvement over available options.
The decision was based on Phase 3 CORE and CORE2 study results showing olezarsen reduced triglycerides by up to 72% versus placebo and cut acute pancreatitis events by 85%, with favorable safety and tolerability. Nearly 90% of patients treated with olezarsen achieved TG levels below 500 mg/dL, a key threshold linked to lower pancreatitis risk. Current therapies often only modestly lower triglycerides and do not reliably prevent acute pancreatitis.
Ionis said it is on track to submit a supplemental new drug application to the FDA by the end of the year. If approved, olezarsen could provide a new treatment option for people with dangerously high triglycerides.
Read Announcement- Drug:
- Olezarsen (Formerly IONIS-APOCIII-L)
- Announced Date:
- October 27, 2025
- Indication:
- Severe hypertriglyceridemia
Announcement
Ionis Pharmaceuticals, Inc announced that detailed data from the landmark pivotal Phase 3 CORE and CORE2 studies of olezarsen in people with severe hypertriglyceridemia (sHTG) will be presented during a late-breaking session, titled "Groundbreaking Trials in Cardiometabolic Therapeutics", at the American Heart Association (AHA) Scientific Sessions in New Orleans.
AI Summary
Ionis Pharmaceuticals announced that detailed data from its pivotal Phase 3 CORE and CORE2 studies of olezarsen in people with severe hypertriglyceridemia will be shared at the American Heart Association Scientific Sessions in New Orleans. This late-breaking session, called “Groundbreaking Trials in Cardiometabolic Therapeutics,” will feature the presentation “Olezarsen in Patients with Severe Hypertriglyceridemia: The CORE-TIMI 72a and CORE2-TIMI 72b Trials” by Dr. Nicholas Marston on Saturday, November 8 from 9:40 to 9:50 a.m. ET. The data showed a statistically significant drop in fasting triglycerides and fewer acute pancreatitis events, along with good safety and tolerability.
Ionis will also host a webcast at 3:00 p.m. ET on November 8 to discuss these results. Interested viewers can access the live webcast online, with a replay available for a limited time.
Read Announcement- Drug:
- Olezarsen (Formerly IONIS-APOCIII-L)
- Announced Date:
- September 19, 2025
- Indication:
- Severe hypertriglyceridemia
Announcement
Ionis Pharmaceuticals, Inc announced that TRYNGOLZA® (olezarsen) has been approved in the European Union (EU) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS). .
AI Summary
Ionis and Sobi announced that TRYNGOLZA® (olezarsen) has been approved in the EU as an adjunct to diet for adult patients with genetically confirmed familial chylomicronemia syndrome (FCS).
The approval follows a positive CHMP opinion and is based on the Phase 3 Balance study. At 80 mg, TRYNGOLZA cut fasting triglyceride levels by a statistically significant amount at six months, sustained through 12 months, and reduced acute pancreatitis events, while maintaining a favorable safety profile.
FCS is a rare genetic disorder marked by extremely high triglyceride levels and a high risk of life-threatening acute pancreatitis. In Europe, it affects about 13 people per million.
TRYNGOLZA is an RNA-targeted therapy that lowers apoC-III production in the liver. Sobi will commercialize TRYNGOLZA across the EU, offering a new adjunct therapy to help FCS patients lower their risk of severe complications.
Read Announcement- Drug:
- Olezarsen (Formerly IONIS-APOCIII-L)
- Announced Date:
- September 2, 2025
- Indication:
- Severe hypertriglyceridemia
Announcement
Ionis Pharmaceuticals Announced Topline Results From Phase 3 CORE and CORE2 Studies Of Olezarsen For Severe Hypertriglyceridemia, Olezarsen Demonstrated A Highly Statistically Significant Reduction In Fasting Triglycerides Of Up To 72% And Acute Pancreatitis Events Of 85% With Favorable Safety And Tolerability
AI Summary
Could you please provide the FDA-related news article detailing Ionis Pharmaceuticals’ Phase 3 CORE and CORE2 study results for Olezarsen? I’ll then summarize it in about 140 words, in HTML paragraphs, at a high school reading level.
Read Announcement- Drug:
- Olezarsen (Formerly IONIS-APOCIII-L)
- Announced Date:
- July 25, 2025
- Target Action Date:
- Q4 2025
- Estimated Target Date Range:
- October 1, 2025 - December 31, 2025
- Indication:
- Severe hypertriglyceridemia
Announcement
Ionis Pharmaceuticals, Inc and Sobi announced that European Commission decision expected by Q4 2025
AI Summary
Ionis Pharmaceuticals and Sobi announced that the EMA’s Committee for Medicinal Products for Human Use (CHMP) gave a positive opinion for TRYNGOLZA® (olezarsen) as a treatment for familial chylomicronemia syndrome (FCS) in adults. The recommendation is based on the Phase 3 Balance study, which showed a significant drop in triglycerides and a substantial cut in acute pancreatitis events sustained over 12 months. The opinion now goes to the European Commission, with an approval decision expected by Q4 2025.
Brett P. Monia, Ph.D., chief executive officer of Ionis, said these results show their commitment to expand access to TRYNGOLZA globally. With strong reductions in triglycerides and fewer pancreatitis events, TRYNGOLZA could make a meaningful difference for people with FCS in Europe.
Sobi, which holds rights to market TRYNGOLZA in the EU, will use its experience from distributing Waylivra® to support a successful launch. Both companies look forward to the final decision later next year.
Read Announcement- Drug:
- Olezarsen (Formerly IONIS-APOCIII-L)
- Announced Date:
- July 25, 2025
- Indication:
- Severe hypertriglyceridemia
Announcement
Ionis Pharmaceuticals, Inc and Sobi® today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion of TRYNGOLZA® (olezarsen) as an adjunct to diet in adult patients for the treatment of genetically confirmed familial chylomicronemia syndrome (FCS).
AI Summary
Ionis Pharmaceuticals and Sobi today announced that the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) has given a positive opinion for TRYNGOLZA® (olezarsen) as an add-on to diet for adults with genetically confirmed familial chylomicronemia syndrome (FCS). The recommendation follows results from the Phase 3 Balance study, which showed that TRYNGOLZA significantly lowered triglyceride levels at six months and sustained this reduction through 12 months, along with a substantial decrease in acute pancreatitis events and a favorable safety profile.
The positive opinion is now with the European Commission, which is expected to make a final decision by the fourth quarter of 2025. If approved, TRYNGOLZA would provide a new treatment option for the rare and serious condition of FCS, where patients struggle to clear high levels of fat from their blood and face life-threatening complications.
Sobi, as Ionis’s commercial partner in Europe, plans to use its experience in rare lipid disorders to help bring TRYNGOLZA to patients, aiming to reduce disease burden and improve quality of life for people with FCS.
Read Announcement- Drug:
- Olezarsen (Formerly IONIS-APOCIII-L)
- Announced Date:
- May 19, 2025
- Indication:
- Severe hypertriglyceridemia
Announcement
Ionis Pharmaceuticals, Inc. announced positive topline results from the Essence study of olezarsen in people with moderate hypertriglyceridemia (fasting triglycerides ≥150 mg/dL to <500 mg/dL) with or at risk for atherosclerotic cardiovascular disease (ASCVD).
AI Summary
Ionis Pharmaceuticals announced positive topline results from the Essence study, which evaluated olezarsen in nearly 1,500 people with moderate hypertriglyceridemia (fasting triglyceride levels between 150 mg/dL and less than 500 mg/dL) who have or are at risk for atherosclerotic cardiovascular disease. Participants, already on standard lipid-lowering therapies, received monthly doses of 50 mg or 80 mg of olezarsen. The study met its primary endpoint by showing a statistically significant 58% to 61% reduction in triglyceride levels compared to placebo at six months, with almost all patients achieving normal triglyceride levels below 150 mg/dL.
Olezarsen also met all key secondary endpoints and demonstrated a favorable safety and tolerability profile, with mainly mild injection site reactions reported. These encouraging results support the potential of olezarsen as a treatment option and contribute to the exposure database ahead of further pivotal trials for severe hypertriglyceridemia expected in 2025.
Read Announcement- Drug:
- Olezarsen (Formerly IONIS-APOCIII-L)
- Announced Date:
- December 19, 2024
- Indication:
- Severe hypertriglyceridemia
Announcement
Ionis Pharmaceuticals, Inc. announced that that the U.S. Food and Drug Administration (FDA) has approved TRYNGOLZA™ (olezarsen) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a rare, genetic form of severe hypertriglyceridemia (sHTG) that can lead to potentially life-threatening acute pancreatitis (AP).
AI Summary
Ionis Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved TRYNGOLZA™ (olezarsen) as an adjunct to diet for reducing triglycerides in adults with familial chylomicronemia syndrome (FCS). FCS is a rare genetic disorder that causes dangerously high triglyceride levels and can lead to life-threatening acute pancreatitis. With this approval, TRYNGOLZA becomes the first treatment in the U.S. specifically designed to manage FCS, offering a new option for patients who previously struggled with strict dietary restrictions.
The treatment is self-administered once a month using an auto-injector, and clinical trials have shown it significantly lowers triglyceride levels and reduces episodes of acute pancreatitis. This approval marks a significant advancement in the care for FCS patients, providing hope for improved quality of life and a reduction in serious, debilitating complications associated with the disease.
Read Announcement- Drug:
- Olezarsen (Formerly IONIS-APOCIII-L)
- Announced Date:
- December 19, 2024
- Indication:
- Severe hypertriglyceridemia
Announcement
Ionis Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved TRYNGOLZA™ (olezarsen) as an adjunct to diet to reduce triglycerides in adults with familial chylomicronemia syndrome (FCS), a rare, genetic form of severe hypertriglyceridemia (sHTG) that can lead to potentially life-threatening acute pancreatitis (AP).
AI Summary
Ionis Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has approved TRYNGOLZA™ (olezarsen) as an adjunct to diet for reducing triglycerides in adults with familial chylomicronemia syndrome (FCS). FCS is a rare genetic disorder that causes severe hypertriglyceridemia and can lead to acute pancreatitis, a life-threatening condition. The approval marks a breakthrough since TRYNGOLZA is the first treatment in the U.S. specifically aimed at significantly lowering triglyceride levels, thereby reducing the risk of dangerous pancreatitis episodes when used alongside a low-fat diet.
The medication is designed to be self-administered once a month via an auto-injector, offering patients a more manageable approach to managing their condition. Ionis Pharmaceuticals is optimistic that this approval will pave the way for further innovative treatments to help those affected by FCS.
Read Announcement
ION337 FDA Regulatory Events
ION337 is a drug developed by Ionis Pharmaceuticals for the following indication: Dravet syndrome.
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:
- ION337
- Announced Date:
- July 13, 2026
- Indication:
- Dravet syndrome
Announcement
Ionis Pharmaceuticals, Inc announced that the first participant has been dosed in the Phase 1-2 ASCEND study of ION337, an investigational RNA-targeted medicine for people living with Dravet syndrome, a rare, severe and lifelong neurological disorder.
AI Summary
Ionis Pharmaceuticals said the first participant has been dosed in the Phase 1-2 ASCEND study of ION337, an investigational RNA-targeted medicine for people with Dravet syndrome. Dravet syndrome is a rare, severe, lifelong brain disorder that can cause frequent seizures and other serious health problems. ION337 is designed to help increase production of the NaV1.1 protein, which is reduced in some people with the condition because of certain SCN1A gene changes.
The medicine uses next-generation N-Methylacetamide modifications, or NMA technology, which Ionis says may improve the strength of splice-modulating antisense oligonucleotides and help the drug work for longer periods. That could support less frequent dosing, which may be easier for patients. Ionis says the study will help evaluate ION337 as a possible treatment option for Dravet syndrome.
Read Announcement- Drug:
- ION337
- Announced Date:
- July 13, 2026
- Indication:
- Dravet syndrome
Announcement
Ionis Pharmaceuticals, Inc announced that the first participant has been dosed in the Phase 1-2 ASCEND study of ION337, an investigational RNA-targeted medicine for people living with Dravet syndrome, a rare, severe and lifelong neurological disorder.
AI Summary
Ionis Pharmaceuticals said the first participant has been dosed in the Phase 1-2 ASCEND study of ION337, an experimental RNA-targeted medicine for people with Dravet syndrome, a rare and severe lifelong brain disorder. The study will test the treatment in patients whose condition is linked to certain SCN1A gene changes. ION337 is designed to raise levels of the NaV1.1 protein, which is too low in many people with Dravet syndrome. The medicine uses next-generation N-methylacetamide, or NMA, technology to improve how strongly antisense oligonucleotides work and to help the drug last longer in the body, which could allow less frequent dosing. Ionis said the program reflects its ongoing focus on neurological diseases with high unmet medical need.
Read Announcement
Eplontersen FDA Regulatory Events
Eplontersen is a drug developed by Ionis Pharmaceuticals for the following indication: Treatment for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
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:
- Eplontersen
- Announced Date:
- July 9, 2026
- Indication:
- Treatment for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
Announcement
Ionis Pharmaceuticals, Inc and partner AstraZeneca announced that the CARDIO-TTRansform Phase 3 trial for eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) did not meet the primary efficacy endpoint of the composite outcome of cardiovascular (CV) mortality and recurrent CV clinical events up to Week 140 compared with placebo.
AI Summary
Ionis Pharmaceuticals and AstraZeneca said the Phase 3 CARDIO-TTRansform trial of eplontersen in patients with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM) did not meet its main goal. The study failed to show a benefit over placebo for the combined outcome of cardiovascular death and repeated cardiovascular events through Week 140.
In a planned subgroup analysis, patients taking eplontersen without stabilizer therapy had a nominally significant result, with a hazard ratio of 0.71 for the same combined outcome. However, patients who were already on stabilizer therapy at the start of the study did not show a treatment effect.
Even though the primary endpoint was missed, several secondary, imaging, and biomarker measures favored eplontersen. The drug also produced large and lasting reductions in transthyretin levels. Overall, eplontersen was well tolerated and had a safety profile similar to earlier findings. The companies said they will continue reviewing the full data set and plan to present results to the scientific community in August 2026.
Read Announcement- Drug:
- Eplontersen
- Announced Date:
- March 10, 2025
- Indication:
- Treatment for hereditary transthyretin-mediated amyloid polyneuropathy (ATTRv-PN).
Announcement
Ionis Pharmaceuticals, Inc. announced that Ionis and AstraZeneca's WAINZUA (eplontersen) has been approved in the European Union (EU) for the treatment of hereditary transthyretin-mediated amyloidosis in adult patients with stage 1 or stage 2 polyneuropathy, commonly referred to as hATTR-PN or ATTRv-PN.
AI Summary
Ionis Pharmaceuticals, Inc. announced that its RNA-targeted medicine WAINZUA (eplontersen), developed with AstraZeneca, has been approved in the European Union for treating hereditary transthyretin-mediated amyloidosis in adults with stage 1 or stage 2 polyneuropathy (hATTR-PN or ATTRv-PN). This approval follows a positive opinion from the Committee for Medicinal Products for Human Use and is based on the NEURO-TTRansform Phase 3 trial. The trial demonstrated that over 66 weeks, patients receiving WAINZUA experienced consistent improvements in neuropathy impairment and quality of life compared to an external placebo. Notably, WAINZUA is the only approved treatment in the EU that can be self-administered monthly via an auto-injector, offering a convenient and effective option for patients managing this debilitating disease.
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ION582 FDA Regulatory Timeline and Events
ION582 is a drug developed by Ionis Pharmaceuticals for the following indication: Angelman syndrome.
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:
- ION582
- Announced Date:
- July 6, 2026
- Target Action Date:
- H2 2027
- Estimated Target Date Range:
- July 1, 2027 - December 31, 2027
- Indication:
- Angelman syndrome
Announcement
Ionis Pharmaceuticals, Inc announced Topline data from REVEAL anticipated in 2H 2027 -
AI Summary
Ionis Pharmaceuticals said topline data from its REVEAL study is expected in the second half of 2027. The company is also preparing to move obudanersen into a Phase 3 study called CHAMPION, which will test the treatment in people with Angelman syndrome who have uniparental disomy or imprinting defect genotypes. Ionis said the CHAMPION trial remains on track to begin before the end of 2026.
These updates show Ionis is continuing to develop obudanersen as a possible treatment for Angelman syndrome. The company is focusing on later-stage testing and expects important data and trial progress over the next two years.
Read Announcement- Drug:
- ION582
- Announced Date:
- July 6, 2026
- Indication:
- Angelman syndrome
Announcement
Ionis Pharmaceuticals, Inc. announced that it has completed enrollment in the pivotal cohort (Cohort 1) of the global Phase 3 REVEAL study evaluating obudanersen (ION582), an investigational RNA-targeted medicine for people living with Angelman syndrome (AS), a serious and rare neurodevelopmental disorder associated with significant impairments in communication, physical function and cognition.
AI Summary
Ionis Pharmaceuticals said it has finished enrolling the main group of patients in its global Phase 3 REVEAL study of obudanersen, also known as ION582, for Angelman syndrome. Angelman syndrome is a rare brain disorder that can seriously affect speech, movement, and thinking. The company said the study is moving forward as planned, and it expects topline results in the second half of 2027.
Ionis also plans to begin another Phase 3 study called CHAMPION by the end of 2026. That study will test obudanersen in people with Angelman syndrome who have uniparental disomy or imprinting defect genotypes. Together, these studies are meant to help show whether the medicine may improve life for people living with this challenging condition.
Read Announcement- Drug:
- ION582
- Announced Date:
- September 9, 2025
- Indication:
- Angelman syndrome
Announcement
Ionis Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy designation to ION582 for the treatment of Angelman syndrome (AS), a rare neurological disease that often presents in infancy and is characterized by profound intellectual disability, impaired communication, motor impairment and debilitating seizures.
AI Summary
Ionis Pharmaceuticals announced that the U.S. Food and Drug Administration has granted Breakthrough Therapy designation to ION582 for the treatment of Angelman syndrome (AS), a rare neurological disease.
Angelman syndrome often appears in infancy and causes severe intellectual disability, lack of speech, motor impairment, balance problems and debilitating seizures. There are currently no approved disease-modifying treatments for AS.
“This Breakthrough Therapy designation underscores the severity of this disease and the significant unmet need for treatment,” said Holly Kordasiewicz, Ph.D., Senior Vice President of Neurology at Ionis. “It also highlights the potential of ION582 to deliver meaningful benefits for individuals with Angelman syndrome.”
The designation is based on positive results from the Phase 1/2 HALOS study, which showed consistent clinical improvements in communication, cognition and motor function, along with favorable safety and tolerability.
Ionis launched the global Phase 3 REVEAL study earlier this year. Enrollment of children and adults with a maternal UBE3A gene deletion or mutation is on track to finish in 2026.
ION582 is an investigational RNA-targeted antisense medicine designed to boost UBE3A protein expression and address the root cause of Angelman syndrome.
Read Announcement- Drug:
- ION582
- Announced Date:
- June 11, 2025
- Indication:
- Angelman syndrome
Announcement
Ionis Pharmaceuticals, Inc. announced that the first participant has been dosed in the global Phase 3 REVEAL study, which is designed to evaluate the efficacy and safety of ION582, an investigational medicine for the treatment of people living with Angelman syndrome (AS), a serious and rare neurodevelopmental disorder that leads to significant physical and cognitive impairments.
AI Summary
Ionis Pharmaceuticals, Inc. announced a major milestone as the first participant was dosed in its global Phase 3 REVEAL study. This study is testing the safety and effectiveness of the investigational medicine ION582 for people with Angelman syndrome (AS), a rare and serious neurodevelopmental disorder that causes significant physical and cognitive challenges.
The trial is a randomized, double-blind, and placebo-controlled study designed to enroll about 200 children and adults with AS who have a maternal UBE3A gene deletion or mutation. Over a 52‑week period, participants will be given quarterly doses of either ION582 or a placebo, and eligible patients will later have the chance to receive the drug for up to two additional years.
This important step reflects Ionis’s commitment to advancing new treatments for individuals with Angelman syndrome, who currently have no approved disease-modifying therapies.
Read Announcement- Drug:
- ION582
- Announced Date:
- June 11, 2025
- Indication:
- Angelman syndrome
Announcement
Ionis Pharmaceuticals, Inc. announced that Completion of enrollment anticipated in 2026
AI Summary
Ionis Pharmaceuticals, Inc. has announced a major milestone in its Phase 3 REVEAL study evaluating ION582 for Angelman syndrome. The global, randomized, double-blind, placebo-controlled trial is designed to assess the safety and efficacy of ION582, an investigational RNA-targeted antisense medicine, in children and adults with specific UBE3A gene mutations or deletions. A key detail from the announcement is that the completion of enrollment for this study is anticipated in 2026. During the 52-week treatment period, approximately 200 participants will be randomized to receive either ION582 or a placebo, followed by a long-term extension where eligible participants will receive ION582 for up to two years. The study aims to measure improvement, particularly in expressive communication, which is a challenging symptom for those with Angelman syndrome. This important effort reflects Ionis’s ongoing commitment to addressing the needs of underserved patient communities.
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BIIB080/IONIS-MAPT FDA Regulatory Events
BIIB080/IONIS-MAPT is a drug developed by Ionis Pharmaceuticals for the following indication: Mild Alzheimer's 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:
- BIIB080/IONIS-MAPT
- Announced Date:
- May 14, 2026
- Indication:
- Mild Alzheimer's disease
Announcement
Biogen Inc. announced compelling topline results from the Phase 2 CELIA study evaluating diranersen (BIIB080), an investigational antisense oligonucleotide (ASO) therapy targeting tau, in individuals with early Alzheimer's disease.
AI Summary
Biogen reported strong topline results from the Phase 2 CELIA study of diranersen (BIIB080), an investigational antisense oligonucleotide therapy for early Alzheimer’s disease. The drug is designed to lower tau by targeting MAPT mRNA, which may help reduce the buildup of abnormal tau linked to brain damage and memory loss.
In the study, three intrathecal dose schedules were tested over 76 weeks: 60 mg every 24 weeks, 115 mg every 24 weeks, and 115 mg every 12 weeks. Biogen said the safety and tolerability profile was generally consistent with earlier results, with similar rates of side effects across dose groups. Serious adverse events were seen more often at the highest dose.
An ongoing long-term extension study is still tracking the drug’s safety, tolerability, and lasting effects in people with early Alzheimer’s disease.
Read Announcement- Drug:
- BIIB080/IONIS-MAPT
- Announced Date:
- April 2, 2025
- Indication:
- Mild Alzheimer's disease
Announcement
Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to BIIB080, an investigational antisense oligonucleotide (ASO) therapy targeting tau, for the treatment of Alzheimer's disease.
AI Summary
Biogen Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to its investigational therapy BIIB080. This novel antisense oligonucleotide (ASO) is designed to target tau protein, which plays a key role in Alzheimer’s disease. Fast Track designation is granted to therapies that address serious conditions and unmet medical needs, allowing for a faster review process. Dr. Priya Singhal, Head of Development at Biogen, stated that the FDA’s decision underlines the urgent need for new treatments targeting tau pathology. BIIB080 is the first tau-targeting ASO to move into clinical development for Alzheimer’s disease and is currently being evaluated in a global Phase 2 study, offering hope for innovative treatment options to patients and their families.
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Donidalorsen (formerly IONIS-PKK-LRx) FDA Regulatory Timeline and Events
Donidalorsen (formerly IONIS-PKK-LRx) is a drug developed by Ionis Pharmaceuticals for the following indication: Hereditary angioedema (HAE).
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:
- Donidalorsen (formerly IONIS-PKK-LRx)
- Announced Date:
- February 27, 2026
- Indication:
- Hereditary angioedema (HAE)
Announcement
Ionis Pharmaceuticals, Inc announced new data to be presented for DAWNZERA™ (donidalorsen), the first and only RNA-targeted medicine for hereditary angioedema (HAE), including new analyses from the Phase 3 OASIS-HAE and OASISplus studies.
AI Summary
Ionis Pharmaceuticals announced it will present new data for DAWNZERA™ (donidalorsen), the first and only RNA-targeted medicine for hereditary angioedema (HAE). The materials include new analyses from the Phase 3 OASIS-HAE and OASISplus studies. These analyses are intended to deepen understanding of donidalorsen’s effects across patient groups and trial endpoints, including additional efficacy and safety evaluations from the pivotal studies.
The company says the new analyses may help clinicians and patients evaluate DAWNZERA’s benefit-risk profile. Important safety information notes DAWNZERA is contraindicated in people with a history of serious hypersensitivity to the drug or its ingredients; hypersensitivity reactions, including anaphylaxis, have been reported. The most common side effects (≥5%) include injection site reactions, upper respiratory tract infection, urinary tract infection, and abdominal discomfort.
Read Announcement- Drug:
- Donidalorsen (formerly IONIS-PKK-LRx)
- Announced Date:
- January 21, 2026
- Indication:
- Hereditary angioedema (HAE)
Announcement
Ionis Pharmaceuticals, Inc and Otsuka Pharmaceutical Co., Ltd. announced that the European Commission (EC) has approved DAWNZERA™ (donidalorsen) in the European Union (EU) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.
AI Summary
Ionis Pharmaceuticals and Otsuka announced the European Commission has approved DAWNZERA (donidalorsen) in the European Union for routine prevention of recurrent hereditary angioedema (HAE) attacks in adults and adolescents 12 years and older. The decision followed a positive opinion from the Committee for Medicinal Products for Human Use.
The approval was based on Phase 3 OASIS-HAE and OASISplus results, where DAWNZERA produced significant, sustained reductions in mean monthly HAE attack rate, including a 94% overall reduction at one year in the OASISplus open‑label extension. DAWNZERA is an RNA‑targeted medicine that targets plasma prekallikrein and is self‑administered by subcutaneous autoinjector every four or eight weeks.
HAE is a rare genetic disorder that causes recurrent, sometimes life‑threatening swelling in areas such as the face, throat, abdomen and extremities, affecting about 1 in 50,000 people. Otsuka holds exclusive rights to commercialize DAWNZERA across Europe and Asia‑Pacific; Ionis is eligible for a $15 million milestone payment and tiered royalties up to 30% on net sales.
Read Announcement- Drug:
- Donidalorsen (formerly IONIS-PKK-LRx)
- Announced Date:
- November 14, 2025
- Indication:
- Hereditary angioedema (HAE)
Announcement
Ionis Pharmaceuticals, Inc. and Otsuka Pharmaceutical Co., Ltd. (Otsuka) today announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency has adopted a positive opinion of DAWNZERA™ (donidalorsen) for the routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.
AI Summary
Ionis Pharmaceuticals and Otsuka announced that the CHMP of the European Medicines Agency has adopted a positive opinion for DAWNZERA™ (donidalorsen) for routine prevention of recurrent hereditary angioedema (HAE) attacks in adults and adolescents 12 years and older. The opinion is now with the European Commission for final approval.
The CHMP recommendation was based on Phase 3 OASIS-HAE and OASISplus trials, which showed significant and sustained reductions in mean monthly HAE attack rates. Results included positive outcomes when patients self-administered the drug using an autoinjector, supporting the breadth of clinical evidence for DAWNZERA.
The European Commission is expected to decide in Q1 2026. Otsuka holds exclusive rights to develop and commercialize donidalorsen across Europe and the Asia Pacific region.
Safety notes: DAWNZERA is contraindicated in patients with serious hypersensitivity to the drug. Hypersensitivity, including anaphylaxis, has been reported. Common adverse reactions include injection site reactions, upper respiratory and urinary tract infections, and abdominal discomfort.
Read Announcement- Drug:
- Donidalorsen (formerly IONIS-PKK-LRx)
- Announced Date:
- July 21, 2025
- Indication:
- Hereditary angioedema (HAE)
Announcement
Ionis Pharmaceuticals, Inc. announced that positive results from the Phase 3 OASISplus prospective switch study of donidalorsen in patients with hereditary angioedema (HAE) were published in The Journal of Allergy and Clinical Immunology (JACI) In Practice.
AI Summary
Ionis Pharmaceuticals announced that the Phase 3 OASISplus prospective switch study of donidalorsen in patients with hereditary angioedema (HAE) was published in The Journal of Allergy and Clinical Immunology In Practice. The study looked at people moving from existing preventive treatments to donidalorsen.
During a 10-week baseline on prior therapies, patients recorded their usual attack rates. After switching to donidalorsen for 16 weeks, they saw no increase in breakthrough attacks. In fact, the average monthly HAE attack rate dropped an additional 62% compared to baseline.
Survey data showed 84% of patients preferred donidalorsen over their previous treatment. They cited better disease control, quicker dosing, and fewer injection-site reactions. Donidalorsen was generally well tolerated, with most side effects being mild or moderate.
Donidalorsen is under review by the U.S. Food and Drug Administration as a potential first-in-class preventative therapy for HAE. A decision is expected by the August 21, 2025 PDUFA date.
Read Announcement- Drug:
- Donidalorsen (formerly IONIS-PKK-LRx)
- Announced Date:
- February 20, 2025
- Indication:
- Hereditary angioedema (HAE)
Announcement
Ionis Pharmaceuticals, Inc. announced that it will present additional data from the pivotal Phase 3 OASIS and OASISplus studies, as well as three year data from the Phase 2 open-label extension (OLE) study of donidalorsen, the company's investigational RNA-targeted prophylactic medicine for hereditary angioedema (HAE).
AI Summary
Ionis Pharmaceuticals recently announced that it will share new clinical data on donidalorsen—a promising RNA-targeted prophylactic medicine for hereditary angioedema (HAE). The company plans to present findings from its pivotal Phase 3 OASIS and OASISplus studies, as well as three-year data from a Phase 2 open-label extension study. These studies show that donidalorsen may help reduce the frequency of HAE attacks while significantly improving patients’ quality of life. Notably, the data suggest that patients switching from other prophylactic treatments experienced fewer attacks and felt better overall. In addition, the ease of donidalorsen administration, which can be done monthly or every two months using an autoinjector, offers a convenient treatment option. The data presentations are scheduled for the 2025 AAAAI/WAO Joint Congress in San Diego, California.
Read Announcement
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