FDA Votes to Approve Experimental ALS Drug

Facts

• After voting against approval back in Mar., a Food and Drug Admin. (FDA) advisory panel on Wed. voted 7–2 to recommend approval of an experimental ALS drug, coming with strong support from patients and advocates.
• The medication, called AMX0035, is a combination of two older drugs: one being a prescription medication for liver disease and the other a dietary supplement used in traditional Chinese medicine.
• The same panel’s original rejection of the drug cited a lack of “substantially persuasive” data. This time, however, its maker Amlyx Pharmaceuticals reportedly showed it could extend life by around 10 months.
• Though not a cure, it purportedly slows the progression of ALS by preventing the premature death of special cells needed for walking, speaking, and eating. Advocates say it’s a major advancement for the roughly 30k ALS patients in the US.
• With the manufacturer set to complete its large trial next year or in early 2024, the company agreed to voluntarily withdraw the drug if approved now but it fails to show effectiveness later. The FDA, which doesn’t have to follow outside recommendations, is expected to make a decision on Sept. 29.
• This comes in the wake of the FDA’s controversial approval of Alzheimer’s Aduhelm last year, in which the agency disregarded negative reviews by advisors and now faces Congressional investigations.

Sources: Washington Post, CBS, Reuters, and NBC.

Narratives

• Narrative A, as provided by Medpage Today. Between the current unmet needs of ALS patients and Amlyx’s promise to withdraw the drug if shown ineffective, this was the right decision by the advisory panel. With no cure for this devastating disease and few other medications on the market, it’s only fair that patients be given the chance to try and extend their lives as much as possible.
• Narrative B, as provided by Tandd. Though patients and advocates understandably want any and every possible treatment available, the FDA should be skeptical of a drug that hasn’t completed its trials and a company’s promise to voluntarily withdraw it from the market if it fails in order to receive approval. There’s a reason it was declined in the past.