AB Science files for conditional Marketing Authorization to EMA for ALS treatment

 AB Science SA (Euronext - FR0010557264 - AB) today announced that it has filed an application for conditional Marketing Authorization to the European Medicines Agency (EMA) for Alsitek (masitinib) in the treatment of amyotrophic lateral sclerosis (ALS). This application has now been validated by EMA and review by the Committee for Medicinal Products for Human Use (CHMP) has begun. The CHMP has a target of 210 active evaluation days to review the application.

The application is based on results from the phase 2/3 AB10015 study and its long-term survival follow-up. Study AB10015 was a randomized, double-blind, placebo-controlled trial over a 48-week treatment period, conducted with 394 ALS patients and evaluating Alsitek in combination with riluzole versus riluzole alone. Detailed results from study AB10015 and its long-term survival analysis have been published in the journals Amyotrophic Lateral SclerosisandFrontotemporal Degeneration [1] and Therapeutic Advances in Neurological Disorders [2].

This decision to file followed a pre-submission meeting held with the CHMP Rapporteur during which new data generated with Alsitek in ALS were presented, in particular, a clinical benefit in terms of a 25-month increase in median overall survival for patients with moderate ALS, which is a cohort that closely resembles newly diagnosed patients. During the pre-submission meeting, AB Science also presented how issues listed in the previous CHMP assessment for Alsitek on ALS (EMA/406203/2018) were resolved, in particular:

• The mode of action of Alsitek in ALS, which has been well-demonstrated and published in peer-reviewed publications [3-8].
• A remonitoring of all efficacy and safety data and a complete reassessment of the Alsitek safety database.
• Additional analyses for the primary efficacy endpoint, imputing all missing data for early discontinuation, and a conservative analysis imputing missing data with a penalty for patients who discontinued Alsitek for lack of efficacy or toxicity. These analyses were positive, and all showed a treatment effect in favor of Alsitek and convergent with the primary analysis.
• Long-term follow-up survival data showing a significant benefit in favor of Alsitek in moderate ALS patients (between group difference in median OS of +25 months, hazard ratio 0.56 (95%CI [0.32;0.96])).

https://www.biospace.com/article/releases/ab-science-announces-that-it-has-filed-an-application-for-conditional-marketing-authorization-to-ema-for-masitinib-in-the-treatment-of-als/