The Food and Drug Administration (FDA) approved a new drug to treat ALS Thursday despite concerns from its own scientists that the drug isn’t effective.
The drug, Relyvrio from Amylyx Pharmaceuticals, is taken once daily for the first three weeks of treatment and twice daily thereafter. It demonstrated in one trial that it slows the decline in ALS patients‘ physical capabilities, according to the FDA. Its own panel of expert advisors, though, voted against recommending approval at a March meeting.
The approval of Relyvrio (#AMX0035) as a treatment for people with ALS inspires hope. We know that we will need many treatments to end ALS, and we are heartened to know that those impacted by this disease will now have another option. @US_FDA @AmylyxPharma
— ALS TDI (@ALSTDI) September 29, 2022
That panel has since reconvened and voted by a count of seven to two to recommend approval, despite admitting that data supporting the drug remained unconvincing. However, the regulators determined the drug was worth a shot to fight a devastating disease with few treatment options.
Amylyx has agreed to pull the drug from the market if an ongoing follow-up trial proves unsuccessful.
Another issue with the new treatment is its cost. Amylyx announced Friday it will charge at least $158,000 for a year’s worth of supply, scaling up to $163,000 after the first year.
At a cost of $12,500 per 28-day supply, patients might have a hard time getting insurance coverage for the medication. Another ALS medication already on the market, Radicava, costs $165,300 per year. Oftentimes insurance plans don’t cover it because of the exorbitant cost.
The market for ALS medications isn’t huge. There are about 32,000 patients suffering from the disease in the United States. At a cost of $163,000 per year, that represents a market worth as much as $5.2 billion. (RELATED: Swamp Personified: Top FDA Tobacco Official Resigns To Head To Major Cigarette Company)
However, the drug may be overpriced, even with high development costs and a small potential customer base. A fair price for the medication would be in the range of $9,100 to $30,600 per year, according to the Institute for Clinical and Economic Review, a nonprofit that works with insurers and pharmaceutical companies to estimate fair costs for newly-developed medicines.