Novartis PKC412 (midostaurin) new drug application (NDA) has received Priority Review from the US Food and Drug Administration (FDA).
The drug is an investigational, oral, multi-targeted kinase inhibitor in development to treat acute myeloid leukaemia (AML) in newly diagnosed adults with an FMS-like tyrosine kinase-3 (FLT3) mutation and for advanced systemic mastocytosis (SM).
Additionally, the FDA has accepted for review the company's premarket approval application (PMA) for the PKC412 (midostaurin) FLT3 companion diagnostic, developed in partnership with Invivoscribe Technologies.
The European Medicines Agency (EMA) has already accepted the marketing authorisation application for PKC412 to treat AML and SM.
Novartis Oncology chief executive officer Bruno Strigini said: "FLT3-mutated AML and advanced SM are devastating and rare diseases, with significant unmet needs due to limited existing treatment options.
"This regulatory designation signifies the importance of midostaurin as a potential therapy for these patients who haven't had the benefit of targeted medicines."
The NDA submission for PKC412 consists of data from the largest clinical trials performed to date in each indication.
Based on the data from the Phase III RATIFY trial (CALGB 10603) conducted in patients with FLT3-mutated AML, the drug was granted Breakthrough Therapy designation by the FDA.
The data indicated that the overall survival improved in patients treated with PKC412 and chemotherapy combination compared to patients treated with placebo and chemotherapy combination.
A Phase II single-arm study to examine the efficacy of PKC412 in patients with advanced SM demonstrated an overall response rate of 60% with a median duration of response of 24.1 months and a median overall survival of 28.7 months.
Novartis has launched a Global Individual Patient Programme (compassionate use programme) and an Expanded Treatment Protocol in the US, to provide access to PKC412 to eligible patients with newly diagnosed AML and advanced SM as the drug is still under evaluation.
Image: Bone marrow aspirate showing acute myeloid leukaemia. Photo: courtesy of VashiDonsk.