After pressure from patients, ALS drug reviewed by FDA

pharmafile | March 25, 2022 | News story | Medical Communications  

The FDA is meeting next week to review a drug closely watched for ALS, also known as Lou Gehrig’s disease, followed by months of lobbying by patient groups and congressional lawmakers. This is in spite of many experts, including the agency’s own outside advisers, stating that the Alzheimer’s drug is unlikely to help patients.

The review has therefore raised the question, how much evidence do patients living with a terminal illness need, to be able to access an experimental drug?

For many battling ALS, the answer is that any drug which may extend, or improve life, is worth trying.

Amyotrophic lateral sclerosis is a progressive nervous system control disease, that affects nerve cells in the brain and spinal cause, causing loss of muscle control. It is a deadly neurodegenerative disease, though some people diagnosed live with it for many years.

Early symptoms of ALS include stiff muscles, muscle weakness, twitches, and gradual increasing weakness. Disease progression leads to difficulty in speaking, swallowing and breathing, and eventual respiratory failure.

The FDA is meeting as a result of patient pressure to publicly review evidence from a small, mid-stage study of Amylyx Pharmaceuticals’ drug. The partial data is already debated by experts.

Regulators have informed Amylyx that it will need to conduct a large, confirmatory study before seeking approval. However, following months of intense lobbying from ALS patients, and their representatives, in US Congress, the FDA has shared that the drug could be submitted based on the smaller study.

The drug appears to modestly slow patient decline.

Traditionally, the FDA requires two large, late-stage studies for approval. However, for deadly diseases such as terminal cancer, and ALS, one study showing promising early results is often accepted.

Some exerts are concerned that the FDA is displaying a pattern of succumbing to external pressure to accept weaker evidence, which damages the agency’s scientific credibility, and opens the door to ineffective treatments.

Ana Ovey

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