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Price of Cambridge biotech’s new ALS drug criticized by watchdog group

It says the $158,000 annual cost per patient isn’t justified, based on the medicine’s effectiveness.

Amylyx cofounders Joshua Cohen (left) and Justin KleeAssociated Press

The day after federal regulators approved its hotly debated new ALS drug, a Cambridge biotech on Friday set the annual list price at about $158,000 for the first year, drawing swift criticism from a nonprofit watchdog group.

Top executives at Amylyx Pharmaceuticals disclosed the price for Relyvrio in a conference call with analysts. In Canada, where the drug received its first approval in June, the company has proposed a price roughly equivalent to $165,000 a year.

Justin Klee and Josh Cohen, the company’s co-CEOs, said in a statement that Amylyx consulted every major US health insurer, doctors, and people living with amyotrophic lateral sclerosis, a devastating progressive neurological disorder. They also said that the price was less than Radicava, another ALS drug, approved by the US Food and Drug Administration in an oral form this year. That costs about $171,000 a year.

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“Taking all of that into account, we made the decision to price Relyvrio below the latest FDA-approved product available to people with ALS,” the executives said.

The company has promised to provide the drug at no cost to patients who are uninsured or underinsured and meet certain financial criteria and have exhausted other options.

Nonetheless, the price tag drew fire from a Boston-based drug-pricing watchdog group, the Institute for Clinical and Economic Review, or ICER. It had recently said that if the medicine were approved, it should be priced at $9,100 to $30,700 based on its clinical benefits to patients, which the organization said ranged from “small to substantial.”

“It’s not surprising, but it’s disappointing,” Dr. David Rind, ICER’s chief medical officer and an internal medicine specialist at Beth Israel Deaconess Medical Center, said of the price. A panel of experts assembled by ICER had considered the possibility that Amylyx might charge $160,000 to $170,000 for the drug and concluded that “at this price it was low value for the money,” Rind said.

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Amylyx’s small intermediate-stage clinical trial of 137 patients found that the drug prolonged survival by a median of 4.8 months compared with a placebo. In light of that and other considerations, an FDA advisory panel of independent medical experts voted in March that the firm had not proven the drug was effective enough to recommend approval. The panel reversed itself in September after patients with ALS and their families and advocates made impassioned pleas.

Amylyx is conducting a longer, larger trial to confirm the benefits of the medicine, with data expected in late 2023 or early 2024. In an unusual pledge, the company has vowed to remove the drug from the market if that study shows it isn’t successful.

ALS is a rare disease diagnosed in about 6,000 people a year in the United States, according to the Massachusetts chapter of the ALS Association. It affects the nerve cells in the brain and spinal cord, gradually robbing patients of the ability to speak, eat, and, finally, breathe.

Since 1995, only two drugs have been approved to treat the paralyzing and fatal disorder ― also known as Lou Gehrig’s disease ― and neither works very well. Amylyx developed a combination of two molecules, sodium phenylbutyrate and taurursodiol, that it believes helps delay the death of nerve cells.

The ALS Association, a Washington, D.C.-based nonprofit that donated $2.2 million for the development and testing of the drug, said Friday it was grateful that it won FDA approval.

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“We are glad that Amylyx is offering a financial assistance program and expect the company to work with payers to ensure everyone can access [Relyvrio] regardless of their financial situation,” said the association.

In recent years, drug companies have developed a number of cutting-edge treatments for rare diseases that carry six- and even seven-figure price tags. They include several gene therapies that patients need take only once and can transform their lives.

In 2019, ICER concluded that the price of Novartis’s gene therapy for spinal muscular atrophy, a rare degenerative neuromuscular disease that kills more infants than any other inherited disorder, was justifiable at $2.1 million because the one-time treatment was remarkably effective.



Jonathan Saltzman can be reached at jonathan.saltzman@globe.com.