APPROVALS

Most patients with ALS die within three to five years and multiple trials for various ALS drugs and treatments have failed in recent years, so the development and approval of a drug that can extend life by even a few months can only be a positive step for those suffering from ALS.

Initially, the advisory panel recommended that the FDA reject the drug, arguing that Amylyx hadn’t made a convincing case for the efficacy of the drug. However, after pleas from patients and their families, the FDA reconvened the panel this month. More data and further analyses were presented, suggesting that the drug will allow patients with ALS to live for up to 11 months longer than previously expected.

Only two drugs treating ALS have been approved since 1995, and neither is particularly effective, so this controversial approval is a significant step in the treatment of ALS.

Despite the uncertainty around the drug and the controversy of its approval, patients with ALS are eager to use the drug. Marc Litt, a retired cardiologist who was diagnosed with ALS in 2019, states: “Obviously, I realise any treatment won’t cure me now, but if my quality of life can be stabilised, I could enjoy some time with my grandson with what’s left.” 

Calaneet Balas, President and CEO of the ALS Association, responded to the indecision around the approval of the drug, stating: “We cannot allow perfection to stand in the way of real progress towards turning ALS from a fatal disease into a liveable one. The FDA’s own ALS guidance acknowledges that people with ALS are willing to accept greater risk for the possibility of some benefit.”