Amylyx Pharmaceuticals’ amyotrophic lateral sclerosis (ALS) drug Relyvrio will reach the market carrying a premium $158,000 a year price without a requirement that the company produce the clinical data to support it.

Approval of the Amylyx drug was a traditional FDA approval as opposed to an accelerated one. The accelerated approval pathway permits a drug to reach the market with less clinical evidence than is required of a standard review. But retaining approval status is contingent on additional data produced from a larger study. Because Relyvrio received a traditional approval, Amylyx is not required to complete a confirmatory study. In summary documents explaining the full approval based on a single clinical trial, the FDA said it was exercising “regulatory flexibility” in light of the life-threatening nature of ALS and the substantial unmet need.

Nevertheless, a Phase 3 test is already underway and Amylyx will complete it. That study was a condition for the drug’s June approval in Canada, co-CEO Justin Klee said during a Friday conference call. He added that Amylyx expects a confirmatory study will also be a requirement in Europe, where the drug is still under review. Klee reiterated a commitment made during an FDA advisory committee meeting earlier this month, during which he said the company would withdraw the drug from the market if it fails in the larger study.

“We will always do what is right for patients and right for the community,” Klee said during the call. “Why would we keep a drug on the market that doesn’t help people? But the data we have today is that Relyvrio shows an impact on function and survival in a disease that is rapidly progressive and universally fatal, so we have a responsibility to the community to deliver.”

The $158,000 a year wholesale price of Relyvrio falls just below the annual cost of Radicava, a Mitsubishi Tanabe Pharma ALS drug that was approved by the FDA in 2017. But Relyvrio’s price is still far higher than the $9,100 to $30,700 price range in which treatment with the drug would be cost effective, according to calculations by the Institute for Clinical and Economic Review (ICER). In a report issued earlier this month, the drug price watchdog group said drugmakers should price new medications based on their therapeutic value, not according to the price of existing medications.

“This is especially important since new drugs are anticipated to be used in combination with other very expensive drugs, creating the highest risk for financial toxicity due to health care costs,” the report said.

In the clinical testing of Relyvrio, dosing of the drug was in addition to standard of care ALS treatments, including Radicava and riluzole, a now generic tablet. The Amylyx drug is comprised of two compounds, the combination of which is meant to improve neuron survival. ICER noted that one of those pharmaceutical ingredients is widely available as a nutritional supplement and it’s still unclear whether that inexpensive component could work as well as on its own. In light of that uncertainty, the ICER report said an alternative approach could be short-term pricing, in which a lower price closer to the cost of production is set until the benefits of treatment can be adequately evaluated.

Relyvrio is a powder that comes in a packet. The powder is dissolved in water and the drug is taken by mouth. Though Amylyx’s formulation is proprietary, the two main components of the drug are not. That means those ingredients could be obtained by those looking to compound them to make their own Relyvrio. Co-CEO Joshua Cohen said Amylyx believes it has strong intellectual property and the company will work with its attorneys to enforce it.

Margaret Olinger, Amylyx’s chief commercial officer, said Relyvrio’s wholesale price is not what most patients should expect to pay. For patients who have commercial insurance, the company will work to provide financial assistance to bring copays to zero. She added that drug companies can’t offset copayments for those on Medicare and Medicaid, but Amylyx is working to ensure that patients on those program have access to the drug as well. For uninsured or under-insured ALS patients, Olinger said Amylyx intends to provide Relyvrio at no cost.

Preliminary results for Relyvrio’s Phase 3 test in ALS are expected in 2024. In addition to continuing that study, Klee said Amylyx also plans to study the drug in other neurodegenerative disorders. The drug has reached mid-stage testing in Alzheimer’s disease.

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