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AMX 0035 filed with EU for amyotrophic lateral sclerosis.

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Published:6th Jan 2022
Amylyx Pharmaceuticals announced it has submitted a Marketing Authorization Application (MAA) to the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for AMX 0035 (sodium phenylbutyrate (PB) and ursodoxicoltaurine (TURSO; also known as taurursodiol)) for the treatment of amyotrophic lateral sclerosis (ALS).

The MAA submission to the EMA is based on data from the CENTAUR trial, a randomized, double-blind, placebo-controlled Phase II clinical trial conducted at 25 centers of the Northeast ALS Consortium (NEALS), evaluating 137 adults with ALS. In this trial, participants receiving AMX 0035 demonstrated a statistically significant reduction in clinical decline at the end of the 6-month randomized phase as measured by the Revised ALS Functional Rating Scale (ALSFRS-R), the most widely used scale in clinical practice and in ALS clinical trials.

In a survival analysis conducted in all randomized participants from the CENTAUR trial who were followed for up to three years, which included participants who continued to receive AMX 0035 in an open-label extension phase during the follow-up period, participants who started on AMX 0035 during the placebo-controlled phase of CENTAUR demonstrated a 44% lower risk of death compared to those who started on placebo during the placebo-controlled phase (HR 0.56; 95% CI, 0.34-0.92). Median survival duration through the open-label long-term follow-up phase was 25.0 months (95% CI, 19.0-33.6 months) in the group that started on AMX 0035 and 18.5 months (95% CI, 13.5-23.2 months) in the group that started on placebo, a 6.5-month difference. Overall, reported rates of adverse events and discontinuations were similar between AMX0035 and placebo groups during the 24-week randomized phase.

Condition: Amyotrophic Lateral Sclerosis/Lou Gehrig's Diseas
Type: drug

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