EU CHMP recommends refusal on application of Masipro (masitinib) in systemic mastocytosis- AB Science

The Committee for Medicinal Products for Human Use (CHMP) adopted a negative opinion, recommending the refusal of the marketing authorisation for the medicinal product Masipro (masitinib), from AB Science, intended for the treatment of systemic mastocytosis. The company may request a re-examination of the opinion within 15 days of receipt of notification of this negative opinion.

The CHMP was concerned about the reliability of the study results because a routine GCP (good clinical practice) inspection at the study sites revealed serious failings in the way the study had been conducted. In addition, major changes were made to the study design while the study was ongoing, which made the results difficult to interpret. Finally, data on the safety of the medicine were limited and there were concerns regarding the medicine�s side effects, including neutropenia (low levels of white blood cells) and harmful effects on the skin and liver, which were of relevance particularly because the medicine was to be used long term. Therefore, at that point in time, the CHMP was of the opinion that the benefits of Masipro did not outweigh its risks and recommended that it be refused marketing authorisation.