The European Medicines Agency (EMA) rejected Amylyx Pharmaceuticals’ marketing authorisation application (MAA) for its amyotrophic lateral sclerosis (ALS) drug Albrioza (AMX0035).

This negative recommendation arrives following the agency’s prior negative opinion on the drug in June 2023, resulting in its reexamination.

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The EMA’s Committee for Medicinal Products for Human Use (CHMP) gave a negative opinion surrounding the drug in its 9-12 October meeting. In the EMA’s first negative opinion, the agency said that it had concerns that “the main study did not show convincingly that Albrioza was effective in slowing down the worsening of the disease”, as per a 13 October press release.

The European agency also claimed that the survival data was unreliable due to its collection and analysis methods. During the reexamination, the agency concluded that these initial issues were not adequately addressed, leading to a refusal, based on the 13 October press release.

In these decisions, the CHMP considered opinions from ALS patient associations and expert groups among others.

Amylyx confirmed that the negative decision would not affect patients in Albrioza clinical trials. The approval decision was made based on results from the Phase II CENTAUR clinical trial (NCT03127514).

The US-headquartered company will continue to focus on the completion of the Phase III PHOENIX trial (NCT05021536), which was initiated before submitting the MAA, as per the press release. This will provide additional data to support the drug’s approval. The company said that it plans to seek EU approval as quickly as possible with topline Phase III results expected in mid-2024.

In June 2022, Health Canada approved the drug, which was soon followed by a US approval. While the drug is marketed under the name Relyvrio in the US, it is known as Albrioza in Canada and the EU.

Albrioza consists of two active substances, sodium phenylbutyrate and ursodoxicoltaurine, which cause a reduction of damage to nerve cells. The EMA granted the therapy an orphan medicine designation in June 2020.

In the 13 October press release, Amylyx’s Europe, the Middle East and Africa (EMEA) head Stéphanie Hoffmann-Gendebien said: “We share the frustration felt by the European ALS community, who has no time to wait for new, safe, and effective treatment options.”

This was in response to a statement from the European Organization for Professionals and Patients with ALS (EUpALS), which 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.”

Currently, the only approved ALS therapy in the EU is Sanofi’s Rilutek (Riluzole).