US Pharmaceutical company withdraws ALS drug following failed trial

Amylyx Pharmaceuticals made a significant announcement on Thursday, revealing its decision to withdraw its approved treatment for amyotrophic lateral sclerosis (ALS) after clinical data indicated the drug’s ineffectiveness. The company stated it would discontinue market authorizations for Relyvrio/Albrioza, the respective brand names of the medication in the US and Canadian markets.

In a joint statement, the company’s co-CEOs, Joshua Cohen and Justin Klee, expressed the difficulty of the decision, emphasizing their commitment to individuals affected by ALS and other neurodegenerative diseases. Additionally, the company disclosed plans to reduce its workforce by approximately 70%, shifting focus to an alternative experimental drug for ALS treatment and exploring repurposing options for Relyvrio.

The decision follows the outcome of a clinical trial involving 664 ALS patients, which reported no significant differences in outcomes between those treated with the drug and those who received a placebo. ALS, commonly known as Lou Gehrig’s disease, is a debilitating condition that progressively damages nerve cells in the brain and spinal cord, with most patients succumbing to the disease within five years of diagnosis.

The approval of Relyvrio by the US Food and Drug Administration (FDA) in 2022 sparked controversy due to its reliance on data from a single trial involving a limited number of participants. Despite acknowledging residual uncertainty about the drug’s effectiveness, the FDA deemed the approval appropriate given the severity of ALS and the lack of effective treatments.

Patient advocacy groups played a pivotal role in advocating for Relyvrio’s approval, with a petition garnering tens of thousands of signatures urging the FDA to act. However, concerns over the drug’s exorbitant pricing and regulatory delays in Europe underscored the challenges faced by ALS patients and their advocates.

Following the withdrawal of Relyvrio, attention now turns to the limited treatment options available for ALS patients. Existing treatments such as Riluzole and Edaravone offer modest benefits, while recent advancements like tofersen provide hope for targeted gene therapy treatments.

In response to Amylyx’s decision, the ALS Association commended the company for prioritizing patient welfare while ensuring continued access to the drug for those who find it beneficial. The association emphasized the importance of swiftly making safe and potentially effective treatments available, pending further research to validate their efficacy.

As the ALS community grapples with the setback, efforts continue to accelerate research and development of innovative therapies to address the urgent medical needs of ALS patients worldwide.