Amylyx’s ALS Drug Is Approved by the FDA


U.S. drug regulators approved

Amylyx

Pharmaceuticals Inc.’s new drug for amyotrophic lateral sclerosis, or ALS, giving patients a new option for slowing progression of the fatal disease.

The drug is the first that the Food and Drug Administration has approved for ALS, also known as Lou Gehrig’s disease, in five years and promises to extend the lives of patients by several months.

“It’s a fantastic step forward in our fight against ALS,” said Sabrina Paganoni, the study’s lead researcher and co-director of the Neurological Clinical Research Institute at Massachusetts General Hospital. “While the drug is not a cure for the disease, there is a significant impact on progression and survival.”

Amylyx, a Boston-area company founded by Brown University graduates who came up with the idea for the drug, called Relyvrio, while in college, didn’t immediately provide a price. The company said the drug should be available through specialty pharmacies in the next four to six weeks.

Amylyx’s ALS drug was cleared in June for sale in Canada under the name Albrioza.



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“Our priority now is to ensure that adults living with ALS in the U.S. whose doctors have prescribed Relyvrio can access it as quickly as possible,” said Amylyx Chief Commercial Officer

Margaret Olinger.

Amylyx shares rose about 10% to more than $33 in after-hours trading on Thursday; the stock is up 67% since the company’s initial public offering in January.

The approval comes as several drugmakers advance new ALS drugs, and after Canadian medicine regulators had cleared the drug for sale under the name Albrioza, prompting some desperate patients in the U.S. to look for ways to get Amylyx’s product.

“This approval provides another important treatment option for ALS, a life-threatening disease that currently has no cure,” said Billy Dunn, director of the FDA’s neuroscience drugs office.

In making its decision, the FDA was forced to juggle the patients’ demand for new treatments with the concerns of staff and some neuroscience experts about whether testing demonstrated Relyvrio truly works.

The agency also faced pressure to approve from patient-advocacy groups including the ALS Association, which contributed $2.2 million raised from the 2014 ice-bucket challenge to Relyvrio’s development.

“This is a victory for the entire ALS community, which came together to advocate for early approval,” said ALS Association Chief Executive Calaneet Balas.

The ALS Association isn’t eligible to receive sales royalties, but is eligible to be paid back 150% of its grant contribution, or about $3.3 million, Ms. Balas said.

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In Canada, Amylyx has proposed a price of about 224,000 Canadian dollars annually per patient, equivalent to around $165,000, though the final price that is negotiated in the country is subject to change.

People taking the Amylyx drug in combination with other approved ALS drugs had a 25% reduction in their disease progression over six months in a mid-stage, or Phase 2, study of 137 people.

The study measured the progression of people with ALS using questionnaires gauging the patients’ ability to perform physical functions such as walking, talking and swallowing.

Björn Oskarsson, an ALS specialist at the Mayo Clinic in Jacksonville, Fla., said he advised against approval in a public comment to the FDA earlier this year because of what he said were inconsistencies in Amylyx’s clinical trial results that warrant further investigation.

Yet he said he would likely prescribe it to patients. “This could very well work, and if you don’t approve it, there’s quite a few people who will not have gotten this drug who would have benefited from it,” said Dr. Oskarsson, who previously served on an Amylyx advisory board.

Amylyx initially submitted data to the FDA showing that its drug helped patients live 4.8 months longer on median than the placebo group in its Phase 2 trial.

Earlier this month, Amylyx presented a new analysis of patients in its Phase 2 trial that estimated patients receiving its drug would have lived 9.7 months longer on median than patients receiving placebos.

FDA reviewers, however, expressed skepticism of the analyses because the study wasn’t designed to measure survival alone, and relied in part on hypothetical estimates of how long placebo patients would have lived had they not been allowed to switch onto the drug after the study was completed.

The FDA extended its initial deadline for making a decision following a March meeting of an advisory committee that narrowly voted that the drug hadn’t been proven effective.

The agency then took the unusual step of convening a second advisory panel meeting for September to reconsider the drug and additional data analyses provided by Amylyx.

At the second hearing, the FDA advisers reversed course and voted 7-to-2 in favor of approving the drug after Amylyx pledged to voluntarily withdraw the drug from the market if a continuing follow-up study is unsuccessful.

“We will do what is right for patients, which includes voluntarily removing the product from the market,” Amylyx Co-Chief Executive

Justin Klee

said.

ALS is a relatively rare neurological disease affecting as many as 32,000 people in the U.S.

The condition progresses rapidly, destroying motor neurons in the spinal cord and brain, leading to a loss of movement, fatigue and problems swallowing that cause most patients to die within two to five years of diagnosis.

The most commonly used ALS treatment is riluzole, which is available generically after being approved in 1995 and is thought to prolong life by two to three months. The drug had an average per patient annual cost of $1,150 in 2020, according to Medicare data.

Relyvrio is a combination of two existing drugs, sodium phenylbutyrate and taurursodiol, which some ALS patients already take separately.

However, sodium phenylbutyrate can cost about $5,000 a month in the U.S. and isn’t covered by many insurers for the treatment of ALS. Taurursodiol is only available as an over-the-counter supplement and patients can’t be assured of its quality, doctors said.

Patients are expected to use Relyvrio with other currently approved ALS treatments, including riluzole and Mitsubishi Tanabe Pharma Corp.’s Radicava, which the FDA approved in 2017 based on a study showing it slowed disease progression.

It can be difficult, however, for patients to get Radicava covered by insurance because of its list price of about $165,300 annually per patient, doctors say.

Drug researchers have struggled to find effective treatments for ALS, though scientists say they have made progress understanding the molecular roots of the disease and several drugs are in development.

Biohaven Pharmaceutical Holding Co.

Ltd. said Thursday that its candidate, called verdiperstat, had failed in a study.

Earlier this month, the FDA said it was joining with the National Institutes of Health and nongovernmental researchers to advance the understanding of ALS and other rare neurodegenerative diseases and support the development of drugs to treat them.

The FDA is scheduled to decide on approval of

Biogen’s

experimental drug tofersen for a rare genetic type of ALS by Jan. 25. Biogen is seeking approval based on the drug’s ability to reduce a protein called neurofilament that is associated with the death of neurons, despite the drug’s failure in a Phase 3 study to slow patients’ disease progression.

Write to Joseph Walker at joseph.walker@wsj.com

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