Amylax Pharmaceutical, Inc., based in Massachusetts, has received a negative response from the Committee for Medicinal Products for Human Use (CHMP) of European Medicines Agency (EMA) ) for the second time on the marketing authorisation application (MAA) for the anti-sclerosis medication AMX0035 (sodium phenylbutyrate and ursodoxicoltaurine or taurursodiol), marketed as Albrioza, despite being approved by the United States Food and Drug Agency.This verdict resulted from a thorough four-month re-examination of the initial negative opinion issued by CHMP in June 2023.

Albrizoa, an oral medication aimed at addressing amyotrophic lateral sclerosis (ALS), a rare neurological condition associated with motor neuron degeneration, recently faced a re-examination after an initial unfavourable opinion by CHMP in June 2023. The comprehensive four-month review ultimately led to this decision.

The drug, designed to combat the debilitating effects of ALS, a condition affecting the nerve cells responsible for voluntary muscle movements like chewing, walking, and speaking, was initially met with a challenging evaluation.

Albrizoa's manufacturer is now emphasising the ongoing Phonix Phase 3 studies, which were initiated before the submission of the Marketing Authorization Application (MAA). This pivotal research is expected to shed more light on the medication's safety and efficacy. The company plans to file for approval in the European Union, with results anticipated by mid-2024.

It may be recalled that the drug had previously received conditional authorization from Health Canada in June 2022 and secured approval from the US Food and Drug Administration (USFDA) in September 2022, marking significant milestones in its journey towards addressing ALS.

General Manager and Head of EMEA at Amylyx, Stephanie Hoffmann-Gendebien, said, “We share the frustration felt by the European ALS community, which has no time to wait for new, safe, and effective treatment options."

"All options will be continuously explored to prompt the company’s mission to deliver AMX0035 to ALS patients in Europe as soon as possible," she added.

In a statement, the European Organisation for Professionals and Patients with ALS (EUpALS) Patients and Carers Expert Board said, “We are disappointed to learn of this outcome, as it is a further setback for the more than 30,000 people living with ALS and their loved ones in Europe who have not seen therapeutic progress for this devastating disease in over 25 years.”

The Centres for Disease Control and Prevention reported that ALS affects more than 31,000 individuals in the United States. According to a study published in Rare India Diseases, ALS affects 6 out of every 100,000 people in India.

The co-CEOs of Amylyx Pharma, Joshua Cohen, and Justin Klee, issued a joint statement saying, “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 United States, thousands of people have been prescribed AMX0035 in North America. ALS has no geographical boundaries, and we are working with urgency towards providing timely, broad, and sustainable access to AMX0035 for eligible people living with ALS who may benefit.”