Health Canada has rubber-stamped an experimental therapy to treat people with amyotrophic lateral sclerosis, also known as Lou Gehrig’s disease, under an accelerated approval program for drugs that show promise but lack data demonstrating their effectiveness, reports The New York Times. The conditional approval requires that the treatment’s developer, Amylyx Pharmaceuticals, provide further evidence that the treatment works, but it will be available in as soon as six weeks to private payers and possibly within several months to those seeking it under Canada’s public health system.
The treatment, AMX0035 (marketed as Albrioza), is composed of two drugs—tauroursodeoxycholic acid and sodium phenylbutyrate—that protect nerve cells by blocking stress signals within mitochondria and protein-synthesizing organelles called endoplasmic reticula, according to ALS News Today.
The Times reports that, to date, the treatment’s effectiveness has been measured in a Phase 2 clinical trial involving 137 participants, as well as an open-label extension study, which ...
