The FDA granted accelerated approval to Biogen’s treatment for a...

The FDA granted accelerated approval to Biogen’s treatment for a rare type of amyotrophic lateral sclerosis, or ALS, giving a small subset of patients a new and much-needed treatment option.Biogen’s drug, tofersen, to be marketed as Qalsody, is meant for the 1% to 2% of ALS patients who have a mutation called SOD1. That equates to about 330 people in the US, with 120 patients newly diagnosed each year, according to Biogen.Biogen didn’t immediately provide the price of the drug, saying in a statement that “Qalsody will be priced within a range comparable to other recently launched ALS treatments.” Amylyx Pharmaceuticals’ Relyvrio, which was approved in September, is priced at $158,000 per year.

Perhaps the more interesting thing for ATH is that:

While Qalsody is for only a tiny group of people, the FDA’s decision to clear it using accelerated approval has far-reaching implications, both for ALS and for other neurodegenerative diseases. It shows the FDA’s increasingly flexible approach to new drugs for devastating neurodegenerative diseases that have limited treatment options, as well as its willingness to use a biomarker for accelerated approval despite mixed clinical evidence.

This news was published by Endpoints News.

The ATH434 phase 2 study has however good clinical evidence tools but in the future, this may be important.