Novartis issues favourable results for PKC412 (midostaurin) study

Novartis said The New England Journal of Medicine (NEJM) has published data for an PKC412 (midostaurin) study that showed an overall response rate of 60% in patients with advanced systemic mastocytosis (SM).

The Swiss pharmaceuticals titan said the median duration of response for all responders in the primary efficacy population was 24.1 months.

Advanced SM is a rare disease that is characterised by the accumulation of abnormal mast cells, a type of white blood cell, in the bone marrow, liver, spleen and other organs, leading to organ damage.

Patients with advanced SM have a poor prognosis, with overall survival varying between less than 6 months to 3.5 years, depending on subtype, and currently there is no approved treatment for the majority of patients.

The pivotal Phase II study, CPKC412D2201, was the largest and longest-running prospective trial ever conducted in this rare disorder. A total of 116 people with advanced SM were enrolled.