Home Medizin Entfernen Sie das ALS-Medikament Relyvrio jetzt vom Markt

Entfernen Sie das ALS-Medikament Relyvrio jetzt vom Markt

von NFI Redaktion

The monitoring group Public Citizen is calling for the immediate removal of the drug Sodium Phenylbutyrate/Tauroursodeoxycholic Acid (Relyvrio) for the treatment of Amyotrophic Lateral Sclerosis (ALS), following its failure to meet the primary and secondary endpoints in the Phase 3 trial results.

As reported by Medical News from Medscape, Amylyx Pharmaceuticals announced the results on March 8th. „We are surprised and deeply disappointed by the PHOENIX results following the positive data from the CENTAUR trial,“ said the company’s Co-CEOs Justin Klee and Joshua Cohen in a statement. The company further stated that it will discuss options with the US Food and Drug Administration (FDA), including a potential voluntary withdrawal of the drug from the market.

The FDA granted accelerated approval to Relyvrio in September 2022 based on a single Phase 2 study. This was followed by an initial rejection by an advisory panel, which was later overturned in a rare second panel review.

The Phase 2 study results, combined with feedback from over 1300 ALS patients, lawyers, and physicians, seemed sufficient to convince the advisory panel to reverse its previous decision.

Public Citizen’s Health Research Group opposed the approval in 2022 and is now calling for a swift withdrawal of Relyvrio. „If the manufacturer does not immediately remove the drug from the market, the FDA should demand its removal,“ said Robert Steinbrook, MD, Director of Public Citizen’s Health Research Group, in a statement.

„The drug should be withdrawn,“ said Caleb Alexander, MD, Professor of Epidemiology and Medicine at the Johns Hopkins Bloomberg School of Public Health Center for Drug Safety and Effectiveness in Baltimore, Maryland.

Speaking as a member of the FDA Advisory Committee on Peripheral and Central Nervous System Drugs, Alexander voted against the approval twice.

„I’m sorry to see these results, but I don’t find them particularly surprising,“ he told Medical News from Medscape. „The evidence of this drug’s efficacy was not very convincing from the start, and it only managed to break through due to a series of somewhat cumbersome analyses,“ he said.

Public Citizen stated that when it came to the approval of Relyvrio, „the FDA capitulated to strong pressure from patients and advocates, many of whom had ties to Big Pharma.“

„It’s important to listen to patients, but ultimately these views should not necessarily be decisive, as the FDA’s responsibility is to follow the science and make decisions based on the safety or efficacy of a drug,“ said Steinbrook to Medical News from Medscape.

Alexander expressed uncertainty about the influence of patient representatives on approvals. „This product also had some pretty strong advocates within the FDA itself,“ he said. However, the engagement of advocates „is an important question,“ he added.

He pointed to the FDA’s process of granting accelerated approval to the Alzheimer’s therapy Aducanumab in 2021, largely promoted by patient representatives, only to be abandoned by its manufacturer Biogen in 2022 when Medicare refused to cover the therapy. The handling of Aducanumab by the agency had „set an unfortunate precedent that has put the FDA under tremendous pressure to show similar leniency with other products,“ he said.

At the time of publication, I Am ALS, one of the organizations heavily involved in advocating for Relyvrio’s approval, had not issued a public statement. Multiple calls to the organization’s headquarters in Washington, D.C. went unanswered.

Amylyx reported that approximately 3900 American patients are currently taking Relyvrio. The drug reportedly costs $158,000 per year. For the year ending December 31, Amylyx reported net sales of $381 million, primarily from US sales of Relyvrio.

It is unclear how thousands of patients could be withdrawn from Relyvrio.

„The logistics for this require fairly rapid discussions between the FDA, the company, and the clinicians treating patients,“ said Steinbrook. „This does not change the overall statement that the drug should be removed from the market if it is not helping patients,“ he added.

Alexander said that the „legal and regulatory complexities of withdrawing the product from the FDA and Amylyx were greatly simplified during the advisory committee meetings.“

He emphasized that the lack of efficacy shown by Relyvrio is not necessarily a sign of a flaw in the FDA’s process.

„This is a specific issue related to a particular product,“ said Alexander. „The FDA will continue to have to work on reconciling access with the evidence of safety and effectiveness.“

Alicia Ault is a freelance journalist based in St. Petersburg, Florida, whose work has appeared in publications such as JAMA and Smithsonian.com. You can find her on Twitter @aliciaault.

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