Peter Marks in FDA: accelerated approvals for rare diseases

https://endpts.com/qa-fdas-peter-marks-ready-to-encourage-more-accelerated-approvals-for-rare-diseases/


Marks: We’re going to be encouraging people in the direction of accelerated approval when they can, especially for rare diseases. Because for rare diseases, and especially things that are like neurodegenerative diseases, it could take a long time to get to clinical endpoints. I suspect we’ll continue to see more accelerated approvals in this rare disease, gene therapy space potentially also in the cell therapy space.



If ATH434 works in the randomized phase 2 study ( even in the open phase 2 study!) and when MSA is a rare, orphan disease, ATH should get ATH434 to the pharmacy, in my understanding. According to Stamler the price of ATH434 will be $100.000 - 150.000 / y., but it is still open.