It is generally illegal for Americans to import drugs that are not approved in the US for personal use. But the FDA website lists some exceptions that may apply to Albrioza, even if the drug doesn’t have serious safety concerns and when it comes to treating “a serious condition for which there is no effective treatment available in the United States.”
dr. Angela Genge, director of the Montreal Neurological Institute’s ALS Global Center for Excellence, which has received compensation from Amylyx for serving on an advisory board, said U.S. patients would be legally able to get Albrioza in Canada if prescribed by a Canadian physician and obtained from a Canadian pharmacy, although they would not qualify for insurance coverage under Canada’s public or private system.
In an interview, Mr. Cohen and Mr. Klee declined to disclose the price Amylyx is considering for Albrioza, as it is still under negotiation. They said the therapy would be available to people paying privately in about six weeks, but it would take longer, possibly months, for people to get coverage under the Canadian public system. Amylyx has already provided Albrioza for free under compassionate-use agreements to 250 patients in the United States, they said.
Until last summer, the FDA had recommended that Amylyx not apply for approval until the drug completed its Phase 3 trial, but in July, officials began suggesting that Amylyx apply for approval using existing data. The timing followed vociferous pressure from ALS advocacy groups in the wake of the approval of the new Alzheimer’s drug, Aduhelm, which has been controversial because many experts said there was insufficient data that Aduhelm worked.
In the phase 2 study, two-thirds of 137 participants received Albrioza, and over 24 weeks they experienced a 25 percent slower decline than the participants who received placebo — 2.32 points less on a 48-point ALS scale that rates 12 physical abilities , including walking, speaking, swallowing, dressing, handwriting, and breathing.
The open-label extension study involved 90 of those patients, including 34 from the placebo group, who started taking the medication about seven months after those who had received it from the start. Those who received treatment the longest had an average of 4.8 months more time before being hospitalized, put on a ventilator or dying, Amylyx reported. Researchers involved in the study released more data last month suggesting additional benefit.
