European approval of AMX0035 for ALS faces new hurdle in review
Developer Amylyx must answer 2nd round of questions from CHMP
Amylyx Pharmaceuticals is facing a new hurdle — a second round of questions from a European Medicines Agency (EMA) committee — in its quest to have AMX0035 approved as a treatment for amyotrophic lateral sclerosis (ALS) in Europe.
The questions, from the Committee for Medicinal Products for Human Use, known as CHMP, come after the company completed a scientific advisory group meeting, in which experts come together to evaluate a specific drug and provide recommendations for approval. However, the committee said there were still certain major objections to AMX0035 that should be addressed.
Amylyx will now respond to the questions, and expects an opinion from the committee by mid-2023. The EMA will consider that opinion in its decision on AMX0035’s potential approval.
While the company had previously said it was expecting a decision from the regulatory authority in the first half of 2023, updated timelines now point to “the third quarter of 2023 at the earliest,” Amylyx stated in press release announcing its financial results for 2022.
“We continue to engage with the European Medicines Agency as it reviews our marketing authorisation application for AMX0035,” said Joshua Cohen and Justin Klee, co-CEOs at Amylyx. “We remain focused on our efforts … to drive the broadest coverage possible for this important new therapeutic option.”
‘Much work remains,’ Amylyx CEOs say
AMX0035 is an oral fixed-dose combination of two small molecules — tauroursodeoxycholic acid and sodium phenylbutyrate — that protect nerve cells from damage induced by cellular stress.
The treatment is commercially available to treat people with ALS in Canada, under the name Albrioza. In the U.S., it was approved in September of last year and is marketed as Relyvrio.
“2022 was an exceptionally exciting year for Amylyx, culminating with the approval of Relyvrio in the U.S. following the approval with conditions for Albrioza in Canada, marking an important step toward delivering much-needed advancements in treatment options to the ALS community,” Cohen and Klee said.
Approvals of AMX0035 in North America were supported by data from CENTAUR (NCT03127514), a Phase 2 trial in which the therapy was found to significantly slow functional decline and extend survival in people with the neurodegenerative disease. That trial involved 137 people with recently diagnosed and rapidly progressing ALS.
Now, the company is conducting the PHOENIX Phase 3 trial (NCT05021536) to test the therapy in a larger group of patients. Enrollment was completed last month, with a total of 664 participants who will receive the medication or a placebo for 48 weeks (nearly one year).
The trial’s main goals are to assess AMX0035’s safety and changes in disease progression, as measured with the ALS Functional Rating Scale-Revised (ALSFRS-R). Secondary measures include changes in lung function, quality of life, and survival.
After completing the trial, patients will be able to join an open-label extension part and continue receiving treatment for two more years.
2022 was an exceptionally exciting year for Amylyx, culminating with the approval of Relyvrio in the U.S. following the approval with conditions for Albrioza in Canada, marking an important step toward delivering much-needed advancements in treatment options to the ALS community.
Earlier this year, Amylyx entered an exclusive license and distribution agreement with Neopharm, which, pending its approval, would make the Israel-based company responsible for regulatory submissions and commercialization of AMX0035 in certain Middle East regions. Those regions include Israel, Gaza, the West Bank, and the Palestinian Authority.
“Our commercial launch is off to a strong start, and we are encouraged by the engagement we have seen from physicians, people living with ALS, and payors,” Cohen and Klee said.
“While we are thrilled with this progress, we are also cognizant that much work remains to meet our goal of quick and efficient access for every eligible person living with ALS,” they added.