The A.L.S. Association is urging the Food and Drug Administration to expedite its review process and grant approval as soon as the company applies for it — but to require rigorous follow-up studies, since the original study was a Phase 2 trial instead of the larger and longer Phase 3, which is often required for approval. The association also wants the company that makes the treatment, Amylyx, a Massachusetts start-up the students founded, to seek the agency’s permission to provide the drug for compassionate use while it is still being evaluated.
Experts who weren’t involved in the study said the data was encouraging, but that important unanswered questions remained about the potential therapy. One unknown is what benefit the drug would have when compared with patients who never received it at all and received only placebo for 30 months, said Dr. Robert Miller, director of clinical research at Forbes Norris MDA/A.L.S. Research Center at California Pacific Medical Center. Still, he said he considered the results a “base hit single.”
The study involved patients who developed symptoms within 18 months before the trial and