BREAKING: Amylyx shares routed on skeptical adcomm docs — but did the FDA leave an open door on unmet ALS need? – Endpoints News

Federal health regulators announced a negative report on Monday regarding an experimental drug closely monitored to treat the debilitating condition that is known as Lou Gehrig’s Disease after months of campaigning by advocates for patients to approve.

The drug produced by Amylyx Pharmaceuticals has become a unifying cause for those suffering from the fatal neurodegenerative disease ALS family members and even members of Congress who have joined forces in pushing for the Food and Drug Administration to allow the drug.

However, regulators stated in a study that the study was “not convincing,” due to missing information, mistakes with the enrollment process and other issues. The next day, an advisory group of FDA advisers will hold an unbinding vote on whether or not the drug is a good idea to approve.

The event is closely watched as a sign of the FDA’s attitude towards new drugs that have no evidence and its capacity to stand up to pressure from outside.

The FDA’s stance against the drug could create the possibility of tension in the public forum on Wednesday which will feature more than a dozen ALS patients and their advocates are scheduled to address. The FDA will take into consideration the recommendations of its advisory panel prior to making a decision about the drug, which is expected to be made by June.

ALS is also known as amyotrophic Lateral degeneration of nerve cells, is a cause of death required to talk, walk and swallow, and eventually breathe. The treatment isn’t available, and the majority of patients end up dying within 3 to 5 years.

Amylyx’s product is a mixture of two drugs that are older that are prescribed for liver problems and the dietary supplement used in tradition Chinese medicine. Amylyx has registered the combination as powder form and claims that the chemicals prevent cells from dying early.

However, the reviewers concluded that the medication produced “only moderate” impact on slowing down the progress of the disease in a 137-patient mid-stage study that reviewers claimed was plagued by issues with implementation and analysis. In general, FDA approval requires two massive studies or a single study that has the “very convincing” impact on the survival of patients.

In the FDA document, the agency’s regulators “strongly advised” Amylyx complete a large trial in late-stage to prove the effectiveness of the drug prior to approving. This study is expected to conclude in 2024.

After more talks with regulators over the summer, Amylyx decided to submit its product to be approved based on the initial study as well as the survival data collected afterward. The company claimed that the results revealed that patients who received the drug lived for one year longer than patients who took a placebo drug.

However, the FDA declares that the study results are not reliable due to issues with tracking participants over a long period of time.

This is the first time that FDA has convened the advisory panel since ignoring its recommendations in June of last year and approved Biogen’s Alzheimer’s medication, Aduhelm. Three members of the panel resigned following the controversial decision, and congressional investigators began a probe into the handling by FDA of the review process for the drug. Similar to the current situation the FDA was under pressure from groups representing patients to approve.

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