Second Time’s The Charm: Amylyx’s ALS Drug Wins US FDA Panel Nod
In switching their votes from the first panel review in March, advisory committee members cited supportive additional analyses of a survival benefit, framing of the issues in the context of unmet need and regulatory flexibility, and the company’s statement that it would withdraw AMX0035 if the Phase III PHOENIX trial does not succeed.
You may also be interested in...
A November 2022 refuse-to-file letter cited clinical and statistical issues for the stem cell therapy, which failed its Phase III clinical efficacy endpoint. However, BrainStorm has consistently asserted that NurOwn demonstrated benefit in patients with less advanced disease at baseline, and it is encouraged by regulatory flexibility FDA recently has shown for the neurodegenerative disease.
Dunn is retiring after an 18-year career that included involvement in some high-profile and controversial drug approval decisions, not the least of which is Biogen’s Alzheimer’s treatment Aduhelm; deputy director Teresa Buracchio takes over the neurology office on an acting basis.
The US FDA’s use of outside expert advisory committees rebounded from historic lows in 2022 – but just barely. Will Commissioner Califf’s ‘update’ of the process finally mean more committee meetings in 2023?