RARE Daily

CHMP Give Another Thumbs Down to Amylyx for ALS Drug Following Re-examination

October 13, 2023

Rare Daily Staff

The Committee for Medicinal Products for Human Use of the European Medicines Agency confirmed its initial negative opinion on the Marketing Authorization Application for Amylyx Pharmaceuticals AMX0035 for the treatment of the neurodegenerative condition amyotrophic lateral sclerosis.

The decision follows the conclusion of the CHMP’s formal re-examination procedure of an initial negative opinion adopted in June 2023.

ALS is a relentlessly progressive and fatal neurodegenerative disorder caused by motor neuron death in the brain and spinal cord. Motor neuron loss in ALS leads to deteriorating muscle function, the inability to move and speak, respiratory paralysis and eventually, death. More than 90 percent of people with ALS have sporadic disease, showing no clear family history. ALS affects approximately 29,000 people in the U.S. and more than 30,000 people are estimated to be living with ALS in Europe (European Union and United Kingdom). People living with ALS have a median survival of approximately two years from diagnosis.

AMX0035 was approved with conditions by Health Canada in June 2022 and granted a full approval by the U.S. Food and Drug Administration under the trade name Relyvrio in September 2022.

“AMX0035 is the first and only drug to show an effect on both function and survival in the same trial. Since the medication’s approval with conditions in Canada and full approval in the U.S., thousands of people have been prescribed AMX0035 in North America,” said Joshua Cohen and Justin Klee, Co-CEOs of Amylyx. “ALS has no geographical boundaries, and we are working with urgency toward providing timely, broad, and sustainable access to AMX0035 for eligible people living with ALS who may benefit.”

Amylyx said it continues to focus on the completion of the PHOENIX phase 3 clinical trial, which was initiated prior to its Marketing Authorization Application submission and will provide additional data on the efficacy and safety profile of AMX0035 in people living with ALS. If PHOENIX is supportive, Amylyx plans to seek approval in the EU as quickly as possible. Topline results are anticipated in mid-2024.

Photo: Justin Klee, Co-CEOs of Amylyx

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