The Center for Medicinal Products and Human Use (CHMP) of the European Medicine Agency (EMA) took a negative opinion on the marketing authorization application for masitinib as a treatment for people with ALS. A second opinion from the CHMP will be issued in July 2018 following a re-examination of the data with input from drug maker AB Science. In general, the EMA took issue with how the trial was conducted at some sites involved as well as some of the analysis conducted by the company of their data.

This announcement follows earlier release of encouraging results from the phase 2/3 trial, as well as the company preparing to launch a confirmatory phase 3 trial based on a proposed responder group. ALS TDI will monitor for other outcomes from EMA reviews and from the FDA regarding the proposed phase 3 trial, which has been speculated to include US enrollment sites.

According to the press release from CHMP, they based their decision to refuse authorization at this time on:

- CHMP found that the study did not show masitinib was effective at slowing down progression of the disease. CHMP recognized the normal vs fast progressor analysis done by AB Science but called the process "arbitrary" and one that "did not reflect clinical practice".

- CHMP inspectors found deficiencies in the the way that two study sites conducted the trial, which may have introduced bias in favor of masitinib.

- CMHP expressed concern that bias in favor of masitinib was introduced via the statistical methods used to account for data from patients who withdrew from treatment.

AB Science requested the re-examination by CHMP and will work to address these points. They expect a second opinion to be delivered by the end of July 2018.

Update: On May 28th, AB Science, makers of masitinib, announced that they have changed their mind and will not be asking the EMA to reconsider their application with responses to the issues raised.  Rather, the company is planning to speak with EMA about pathways available for potential approval, and potentially, submit an entirely new application for marketing authorization.

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