Amylyx Pharmaceuticals, Inc. today announced that the U.S. Food and Drug Administration (FDA) has accepted for review its New Drug Application (NDA) for AMX0035 (sodium phenylbutyrate (PB) and taurursodiol (TURSO; also known as ursodoxicoltaurine)) for the treatment of amyotrophic lateral sclerosis (ALS). The FDA has granted Priority Review and assigned a Prescription Drug User Fee Act date for AMX0035 of June 29, 2022, the target date by which the FDA intends to complete its review and take action on the NDA. The Agency noted that it is currently planning to hold an advisory committee meeting to discuss the application. Amylyx additionally is preparing to submit an Expanded Access Program (EAP) to the FDA for launch in the United States in the coming months for patients who are ineligible for participation in the global Phase 3 PHOENIX clinical trial.
Asia Jami was diagnosed with Lou Gehrig's disease six years ago. Amyotrophic lateral sclerosis (ALS) is a progressive neurodegenerative disease that impacts nerve cells in the brain and the spinal cord.
Many Americans first learned about amyotrophic lateral sclerosis (ALS) when baseball legend Lou Gehrig said he was the luckiest man on the face of the earth.
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In 2002, my dad, Martin Doctoroff, died of ALS. At the time, we thought it was a tragic fluke, as is the case with the vast majority of ALS patients. But when his brother, Mike, was diagnosed in 2008, it was clear the disease wasn’t random. Ever since then, my family has been living with a spectre that was always vaguely present. Now, unfortunately, it appears very likely that I have ALS, too.
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The fatal nerve disease has few treatments. But drug developers are hopeful that genetic technologies and a better understanding of biology will bring new options.
A man with Amyotrophic Lateral Sclerosis (ALS) had the advance of his disease slowed, and even halted altogether, thanks to a new trial that could offer hope to the 12,000 to 15,000 patients who currently suffer from the disease.