Cambridge

Cambridge Company's Newly Approved ALS Drug Offers Hope for Families

The FDA approved the drug based on a small study in part because there are few treatment options for ALS

A Cambridge company has won approval for a new drug to treat ALS. For many families, it brings a new sense of hope.

The new drug from Cambridge-based Amylyx Pharmaceuticals Inc. is called Relyvrio.

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 “People with ALS are some of the most strong and incredible people,” said Joshua Cohen, co-CEO and cofounder of Amylyx pharmaceuticals.

Cohen and Justin Klee are the company’s CEOs. They say the drug, which can be taken as powder in single-use sachets, for oral suspension, can increase lifespan by about five months.

“In our studies there was shown to be an impact on function, so slowing disease progression as well as a difference in overall survival so people lived longer,” Klee said.

Thursday’s approval still comes with questions. The FDA approved it based on results from a small, mid-stage study, as opposed to two large studies, or one with very strong results.

The urgency, they say, is because of the limited treatments available for ALS.

But that still brings hope to many, including Nancy Frates, whose son Peter Frates was an inspiration behind the viral ice bucket challenge.

“I’m just full of hope and joy,” she said.

The former Boston College baseball star was a driving force for the challenge to raise money for ALS before he passed from the disease in 2019.

"If you’re a family who may quite possibly be looking at an ALS diagnosis, take hold of every bit of hope that this brings and hang on to it because - is this the cure? It’s not the cure and we know that. But it’s another step towards the cure,” she said.

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