The pharmaceutical company said it received notice from the French medicines agency Agence Nationale de la Sécurité des Médicaments, or ANSM, requesting the temporary suspension of ongoing masitinib clinical trials in France for mastocytosis until AB’s compliance is confirmed by an independent audit.
The company is working closely with ANSM to resolve some quality and safety procedures found after an inspection by the European Medicines Agency (EMA) to restart the recruitment of patients in clinical studies in France. The inspection showed deviations from the Good Clinical Practice (GCP) in the mastocytosis pivotal study (NCT00814073) and deviations related to the pharmacovigilance system.
The findings identified during this inspection do not affect the ALS studies, AB Science said.
The randomized, double-blind trial (NCT02588677) compared the effectiveness and safety of a combination of masitinib and riluzole (brand names Rilutek or Teglutik) with a combination of a placebo and riluzole.
Interim results showed that masitinib in combination with riluzole improved patients’ quality of life and ability to survive. Researchers used the ALS functional rating scale, which measures progression of the disability, quality of life, and survival to assess patients who had completed 48 weeks of treatment.
Masitinib is an orally administered therapy that targets mast cells and macrophages, important cells for immunity, inhibiting the activation of inflammatory processes. This modulatory effect indicates the drug can be effective against symptoms of inflammatory and CNS-associated diseases like ALS.
Last year, the EMA accepted a conditional marketing authorization application for masitinib to treat ALS. The drug was also designated an orphan drug by the EMA’s Committee for Orphan Medicinal Products (COMP).
The EMA’s decisions were based on data from the Phase 2/3 trial and on preclinical data showing neuroprotective effects of masitinib in ALS through the targeting of microglial cells. The data was published in the Journal of Neuroinflammation in a report titled, “Post-paralysis tyrosine kinase inhibition with masitinib abrogates neuroinflammation and slows disease progression in inherited amyotrophic lateral sclerosis.”
EMA is expected to give a final decision regarding the drug’s approval for the treatment of ALS before the end of the year.