- Amylyx has developed a drug for ALS that could slow down the disease and extend patients’ lives.
- The biotech will now file for approval, following new guidance from the FDA
- The FDA had asked Amylyx for more data, but reversed course shortly after approving a controversial Alzheimer’s drug.
Americans with a rare and fatal brain disorder were devastated to learn in April that a promising drug wouldn’t be available in the US for at least a couple of years.
But on Wednesday, entrepreneurs Joshua Cohen and Justin Klee announced a change in course, following new conversations with the US Food and Drug Administration.
Their company, Amylyx Pharmaceuticals, will ask the FDA this year to approve its treatment for amyotrophic lateral sclerosis, also known as ALS or Lou Gehrig’s disease.
Amylyx’s drug could cut a person’s risk of death by 44% over three years, according to mid-stage trial data. That kind of effect is better than anything ALS researchers and advocates have seen in years. But the FDA strongly recommended that the biotech run another trial, Cohen told Insider in April.
The change came as a surprise. Companies don’t often get differing advice from the FDA within the span of a few months. Plus, the regulatory agency is still dealing with the fallout from its decision to approve a treatment for another brain disorder — Alzheimer’s disease — early.
Experts say it shows shifts within the regulatory agency that are emboldening drug companies to move for earlier approval of their treatments.
The Amylyx-FDA conversations shifted one month after a controversial drug decision
At least one major event happened between Amylyx’s conversations with the FDA this spring and the company’s meeting with the agency in July: the FDA made a controversial decision to approve another brain disorder drug.
The drug in question, Biogen’s Alzheimer’s treatment Aduhelm, has been allowed onto the market based on one successful late-stage study. The FDA normally wants to see data from two trials showing a medication works. Instead, it is allowing Biogen to confirm Aduhelm works by running another trial sometime in the next nine years.
Cohen and Klee said they don’t yet know whether Amylyx will be asked to run a confirmatory trial. The company had already put together plans for a new trial based on the FDA’s feedback in the spring.
The Aduhelm decision changed the drug world, industry consultant and neuroscientist Eric Siemers said. It led three external FDA advisors to resign in protest, and placed other officials in hot water.
“It’s really interesting that they’re continuing to push the envelope. You’ve got two congressional committees investigating the Aduhelm approval, and questions of whether Billy Dunn will keep his job,” Siemers said, referring to the head of the FDA’s neuroscience branch. “The speculation I’ve heard was that the FDA was going to have to dial back.”
The FDA said in a statement that it can’t comment on any potential applications.
“The agency continues to recognize the critical unmet medical need for new, effective treatments for complex neurological diseases, such as ALS,” an FDA spokesperson said. “We are committed to working with sponsors of novel therapies and the ALS community to facilitate development and approval of agents to treat this devastating disease.”
The Alzheimer’s drug decision is emboldening neuroscience companies
The whole regulatory environment is subject to change right now, regulatory consultant and neurobiologist Kevin Phelan told Insider. But he doesn’t attribute that to the Aduhelm decision.
The agency has been bending its rules over the last few years, particularly when it comes to allowing drugs to hit the market based on a single significant study. In 2016, it approved Sarepta Therapeutics’ treatment for another rare neurological disease based on a single, small trial. In 2019, the agency released draft guidance laying out the conditions for getting a drug approved based on a single late-stage study.
What the Aduhelm decision may have done was made neuroscience companies like Amylyx willing to push the FDA a bit.
“If Amylyx took a look at Aduhelm and said, it seems like nothing is impossible, even if it’s a less than 50% chance, let’s go ahead,’ I can see that being an easier corporate decision,” Phelan said.
Companies working on other brain disorders will likely follow suit.
“Everybody is going to have exactly these questions: Does this mean Huntington’s disease, things are different now? For Parkinson’s disease?” Siemers said.