Is FDA Ready to Make Another Sarepta- and Biogen-Like Decision?
(Thursday, April 7, 2022)
In July 2016, FDA approved Sarepta’s DMD drug, and in June 2021, it approved Biogen’s Aduhelm for Alzheimer’s disease, both times going against the vote of its Advisory Committees. Now FDA is being lobbied again by patient groups to approve Amylyx’s AMX0035 for ALS despite a no-vote from the Advisory Committee. Is FDA ready for another one of its controversial decisions? It is hard to know if the previous controversial decisions helped the patients. There is limited data on the benefits of Sarepta’s DMD drugs for the patient. Sarepta, however become a multi-billion-dollar corporation based on its first approval. Biogen has not been so fortunate. Aduhelm was severely panned by practically all health groups and got strictly limited coverage by CMS, curtailing its use in patients and the resulting sales. It is still early for Biogen as the drug may recover its market eventually as it is the only drug approved by FDA for Alzheimer’s disease. Amylyx’s ALS drug fits the above plan like a glove. The clinical trial with the drug did not show major improvement, but the patient groups want hope more than a treatment and are lobbying hard for it. Ironically, ALS patients are using Aduhelm’s approval to make their case for the drug. Apparently scientific and medical controversy has little influence on the patients’ desire to have a treatment even if it is not proven to work. Amylyx is obviously bullish about the product despite the negative vote by the Ad Comm making passionate (read cheesy) case about saving patients dying of ALS waiting for FDA approval, side-stepping the fact that the drug has shown no evidence that it would actually save any patient’s life. So, the decision falls back to the FDA. Does it repeat the precedence it created with previous drug approvals despite poor evidence, or does it this time decide to be scientifically objective and not emotionally subjective in its decisions. There will be controversy either way. FDA decision should be based on facts. Regulatory flexibility specifically asks for promising preliminary data for the drug. By using the regulatory flexibility for non-promising drugs, FDA only creates more incentives for drug companies trying to monetize patient’s desperation. At least, by following its own standards FDA would not be a party to companies reaping billions on the back of patients while patients keep dying for non-working therapies.
Dr. Mukesh Kumar
Founder & CEO, FDAMap
Linkedin: Mukesh Kumar, PhD, RAC