This section highlights FDA-related milestones and regulatory updates for drugs developed by Amylyx Pharmaceuticals (AMLX).
Over the past two years, Amylyx Pharmaceuticals has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
AMX0035 and AMX0114. 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.
AMX0035 FDA Regulatory Timeline and Events
AMX0035 is a drug developed by Amylyx Pharmaceuticals for the following indication: To treat amyotrophic lateral sclerosis (ALS).
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:
- AMX0035
- Announced Date:
- August 27, 2025
- Indication:
- To treat amyotrophic lateral sclerosis (ALS)
Announcement
Amylyx Pharmaceuticals, Inc. announced the decision to discontinue the ORION program of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine]) in adults living with progressive supranuclear palsy (PSP).
AI Summary
Amylyx Pharmaceuticals announced it will discontinue the ORION program testing AMX0035—a fixed-dose combination of sodium phenylbutyrate and taurursodiol—in adults with progressive supranuclear palsy (PSP). This decision follows Phase 2b results showing no difference between AMX0035 and placebo on primary or secondary outcomes at Week 24.
Although AMX0035 continued to be generally well-tolerated and safety data aligned with earlier studies, the lack of efficacy led Amylyx to end both the Phase 2b trial and its open-label extension. The company will not move forward with a planned Phase 3 study in PSP.
Amylyx expressed gratitude to participants, families and study sites for their support. The company is now focusing on its pivotal Phase 3 LUCIDITY trial of avexitide, ongoing AMX0035 development in Wolfram syndrome, and early ALS studies of AMX0114, with a cash runway expected through 2026.
Read Announcement- Drug:
- AMX0035
- Announced Date:
- August 12, 2024
- Indication:
- To treat amyotrophic lateral sclerosis (ALS)
Announcement
Amylyx Pharmaceuticals, Inc announced the publication of exploratory analyses on cerebrospinal fluid (CSF) biomarkers from participants with Alzheimer's disease (AD) from the Phase 2 PEGASUS trial.
AI Summary
Amylyx Pharmaceuticals announced the publication of exploratory analyses on cerebrospinal fluid (CSF) biomarkers from participants with Alzheimer’s disease in the Phase 2 PEGASUS trial. The study explored how AMX0035, a combination of sodium phenylbutyrate and taurursodiol, may affect key biomarkers associated with neurodegeneration. Researchers observed that treatment with AMX0035 resulted in consistent changes in the CSF levels of several biomarkers, including those related to tau pathology, synaptic and neuronal degeneration, and gliosis. Specifically, reductions were seen in phosphorylated tau181 and total tau, as well as in biomarkers of neuronal damage such as neurogranin and FABP3. These results support the idea that AMX0035 could engage multiple pathological pathways involved in Alzheimer’s disease, offering further insight into its potential to slow or mitigate neurodegeneration in patients with varying severity of the disease.
Read Announcement- Drug:
- AMX0035
- Announced Date:
- August 2, 2024
- Indication:
- To treat amyotrophic lateral sclerosis (ALS)
Announcement
Amylyx Pharmaceuticals, Inc announced that Topline data for all 12 participants from Phase 2 HELIOS trial studying impact of AMX0035 on endocrine, metabolic, and neurodegenerative aspects of Wolfram syndrome anticipated fall 2024
AI Summary
Amylyx Pharmaceuticals announced that topline data is expected this fall 2024 from its Phase 2 HELIOS trial, which is studying the impact of AMX0035 on Wolfram syndrome. The trial involves 12 participants and focuses on the drug’s effects on endocrine, metabolic, and neurodegenerative aspects of the disease. Interim results from eight participants showed that AMX0035 may help stabilize or even improve pancreatic function, blood sugar control, and vision—areas that typically worsen over time in Wolfram syndrome. The full data release will include Week 24 assessments for all trial participants, providing important insights into the potential benefits of AMX0035. This study is a significant step toward addressing the urgent unmet needs of those affected by this rare and progressive condition, which is marked by early-onset diabetes, vision loss, and neurological decline.
Read Announcement- Drug:
- AMX0035
- Announced Date:
- August 2, 2024
- Indication:
- To treat amyotrophic lateral sclerosis (ALS)
Announcement
Amylyx Pharmaceuticals, Inc. announced the European Commission (EC), based on a positive opinion issued by the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency (EMA), has granted Orphan Drug Designation for AMX0035, Amylyx' proprietary, fixed-dose combination of sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine outside of the U.S.) for the treatment of Wolfram syndrome.
AI Summary
Amylyx Pharmaceuticals announced that the European Commission has granted Orphan Drug Designation for its drug AMX0035, following a positive opinion from the Committee for Orphan Medicinal Products of the European Medicines Agency. AMX0035 is a proprietary, fixed-dose combination of sodium phenylbutyrate and taurursodiol and is being developed for treating Wolfram syndrome—a rare, progressive, and life-threatening disorder. Wolfram syndrome causes childhood-onset diabetes, vision loss, hearing decline, and neurological problems, and there are currently no approved treatments for the disease.
This designation recognizes the potential of AMX0035 to provide a significant benefit over existing approaches. In addition to the designation, Amylyx is conducting a Phase 2 HELIOS trial to further evaluate the drug’s impact on endocrine, metabolic, and neurodegenerative aspects of Wolfram syndrome, with topline data expected this fall.
Read Announcement- Drug:
- AMX0035
- Announced Date:
- April 10, 2024
- Indication:
- Amyotrophic lateral sclerosis (ALS)
Announcement
-Amylyx Pharmaceuticals, Inc announced interim data from the ongoing Phase 2 HELIOS clinical trial of AMX0035 (sodium phenylbutyrate [PB] and taurursodiol [TURSO, also known as ursodoxicoltaurine]) in adults living with Wolfram syndrome, a rare, progressive genetic disease impacting approximately 3,000 people in the U.S
Read Announcement- Drug:
- AMX0035
- Announced Date:
- April 8, 2024
- Indication:
- Amyotrophic lateral sclerosis (ALS)
Announcement
Amylyx Pharmaceuticals, Inc announced the Company will host a virtual webcast with management and Fumihiko Urano, MD, PhD, Principal Investigator of the HELIOS clinical trial and the Samuel E. Schechter Professor of Medicine in the Division of Endocrinology, Metabolism & Lipid Research at Washington University School of Medicine in St.
AI Summary
Amylyx Pharmaceuticals has announced an upcoming virtual webcast where company management and Dr. Fumihiko Urano will discuss interim data from the HELIOS Phase 2 clinical trial. Dr. Urano, the principal investigator of the trial and the Samuel E. Schechter Professor of Medicine at Washington University School of Medicine in St. Louis, is a leading expert in Wolfram syndrome. The webcast, scheduled for April 10, 2024, at 1:30 pm ET, will provide an update on the study of AMX0035, an experimental treatment candidate that targets neurodegeneration in patients with Wolfram syndrome.
The session will offer investors and interested parties insights into the trial's safety, tolerability, and various functional measures. The live event can be accessed through Amylyx’s investor website, with a replay available for 90 days after the event, supporting the company’s commitment to transparent clinical updates in the neurodegenerative disease community.
Read Announcement
AMX0114 FDA Regulatory Events
AMX0114 is a drug developed by Amylyx Pharmaceuticals for the following indication: Treatment of Amyotrophic Lateral Sclerosis.
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:
- AMX0114
- Announced Date:
- June 3, 2025
- Indication:
- Treatment of Amyotrophic Lateral Sclerosis
Announcement
Amylyx Pharmaceuticals, Inc. announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to AMX0114, an investigational antisense oligonucleotide (ASO) targeting calpain-2 for the treatment of people living with amyotrophic lateral sclerosis (ALS).
AI Summary
Amylyx Pharmaceuticals announced that the FDA has granted Fast Track designation to its investigational AMX0114. This antisense oligonucleotide is designed to target calpain-2, a protein involved in the axonal degeneration seen in amyotrophic lateral sclerosis (ALS). Calpain-2 is known to play a key role in advancing ALS, a devastating and progressive disease with few treatment options.
The Fast Track status aims to facilitate a quicker development and review process by allowing more frequent interactions with the FDA. Preclinical studies have shown that AMX0114 can reduce calpain-2 activity, improve neuronal survival, and lower levels of nerve damage biomarkers. The company is moving forward with the Phase 1 LUMINA trial and anticipates early data later in 2025, marking an important step toward addressing the unmet needs of people living with ALS.
Read Announcement- Drug:
- AMX0114
- Announced Date:
- January 21, 2025
- Indication:
- Treatment of Amyotrophic Lateral Sclerosis
Announcement
Amylyx Pharmaceuticals, Inc announced that the U.S. Food and Drug Administration (FDA) has lifted the clinical hold placed on the Phase 1 clinical trial of AMX0114, an investigational antisense oligonucleotide (ASO) targeting calpain-2 for people living with amyotrophic lateral sclerosis (ALS).
AI Summary
Amylyx Pharmaceuticals announced that the FDA has lifted the clinical hold on its Phase 1 clinical trial for AMX0114, an investigational antisense oligonucleotide targeting calpain-2 in people with ALS. With approval now granted, the company is set to open trial sites across the U.S. for screening, enrollment, and dosing. The study, known as the LUMINA trial, will explore the safety and biological activity of AMX0114, assessing key ALS biomarkers such as neurofilament light levels. About 48 patients with ALS will be enrolled in a multicenter, randomized, placebo-controlled study, with participants receiving doses every four weeks. This milestone clears the way for further evaluation of AMX0114’s potential to slow axonal degeneration and offer a new treatment approach for those living with ALS, marking an important step forward in addressing unmet needs in this challenging disease.
Read Announcement
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