EMA committee leaning toward not recommending AMX0035 approval
A final opinion on the marketing application in the EU is expected June 19-22
A committee of the European Medicines Agency (EMA) is leaning toward not recommending a conditional approval of Amylyx Pharmaceuticals’ AMX0035 for treating amyotrophic lateral sclerosis (ALS) in the European Union.
The drug is already marketed in the U.S. under the name Relyvrio (sodium phenylbutyrate and taurursodiol) and in Canada as Albrioza, but has faced some hurdles in its regulatory review in the European Union.
Earlier this year, the EMA’s Committee for Medicinal Products for Human Use (CHMP) met with experts to evaluate the drug and provide recommendations for its approval. The committee instead sought more answers on major objections that remained.
A final opinion on the marketing authorization application is expected June 19-22, when CHMP holds its next meeting. While not binding, the opinion will be considered by the EMA when it makes its decision.
What happens next?
Should CHMP issue a negative opinion, the company plans to seek a formal re-examination based on available data from the CENTAUR trial (NCT03127514). A re-examination could take about four months.
“We disagree with the current view expressed by the CHMP and remain confident in the data from the CENTAUR trial. Should a negative opinion ultimately be issued, we intend to request a formal re-examination procedure,” Tammy Sarnelli, global head of regulatory affairs and clinical compliance at Amylyx, said in a company press release.
AMX0035 is an oral fixed-dose combination of sodium phenylbutyrate and taurursodiol, two compounds thought to protect nerve cells from cellular stress and help slow the progression of ALS symptoms.
Its approval, first with conditions in Canada and about a month later in the U.S., was based on data from CENTAUR, a Phase 2 clinical trial involving 137 adults with rapidly progressing ALS.
The data showed AMX0035 was well tolerated and significantly slowed functional disability progression compared with a placebo. The findings from CENTAUR and its open-label extension also showed a 10-month survival benefit from the therapy.
“There has not been a new product approved in the European Union for ALS in over 25 years,” said Stéphanie Hoffmann-Gendebien, Amylyx’s head and general manager of international markets. “We know from the European ALS community that there is a desperate need for new therapeutic options for this devastating disease, and timely access to safe and effective treatments is especially important.”
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