After an about-face on a PhIII trial, FDA sets up an adcomm to review controversial ALS drug next month
Following a U-turn from the FDA, the agency’s Peripheral and Central Nervous System Drugs Advisory Committee will meet on March 30 for an all-day teleconference to discuss what could be only the third ALS drug approved since 1995.
Although the potential drug from Amylyx, known as AMX0035, may work, the FDA previously sought another trial to confirm early positive results. The agency had asked Amylyx to conduct a Phase III study last April to see whether Phase II data from 2020 held up.
But then, roughly five months later, the agency reversed course, saying the new study wouldn’t be needed for review. Amylyx submitted its ALS drug for US approval in November after meeting with regulators, and it was granted priority review the next month. The expected PDUFA date is June 29.
The biotech previously published its Phase II/III trial results in the New England Journal of Medicine, showing AMX0035 could slow the onset of ALS against a standard-of-care-based placebo. Based on a functional rating scale, which Amylyx used to measure its primary endpoint, AMX0035 showed a statistically significant reduction in disease progression over about six months.
The 137-patient study showed how those in the active arm scored on average 2.32 points higher on the survey than placebo after 24 weeks, good for a p-value of p=0.03.
While the results weren’t definitive, and ALS is historically a tough field for R&D and a fatal disease where most patients live an average of two to five years after receiving their diagnoses, the results were good enough for officials in the EU and Canada to consider approval.
While it’s unknown how the FDA or its outside advisors will view the final data package, the announcement of the meeting is another sign that FDA is adopting more relaxed approval standards in neuro.
Similarly to Biogen’s controversial Alzheimer’s drug approval, the FDA’s neuro head Billy Dunn, who’s being investigated for an improperly close relationship with Biogen, helped drive the shift by the agency with Amylyx.
In an interview with Endpoints News in September, Amylyx co-CEO Josh Cohen said multiple conversations with US regulators, specifically Dunn’s group in the neuro division, led to the agency to change its mind. By Cohen’s retelling, the FDA had been excited about the Phase II CENTAUR data but was interested in seeing more data before moving forward.
Helping with a potential drug launch, should the FDA OK the drug, the Massachusetts-based Amylyx IPO-ed last month, raising $190 million after pricing its stock at the midrange of the offering.
The company is led by a pair of former college roommates — Cohen and Justin Klee — who got some key early support from the late Henri Termeer.