Novartis has added EU approval its US registration for FLT3 inhibitor Rydapt, a new drug that brings years of stagnation in the development of new drugs for acute myeloid leukaemia (AML) to an end.
The EMA has cleared Rydapt (midostaurin) for use alongside chemotherapy in patients with newly-diagnosed adults with AML whose cancer cells carry the FLT3 genetic mutation.
The regulator also approved the drug for systemic mastocytosis (SM) and related conditions such as mast cell leukaemia, making it the first EU-approved therapy for this group of rare and life-threatening diseases.
AML is the most common form of acute leukaemia in adults and has a low survival rate, with just one in four patients still alive five years after diagnosis.
Treatment has relied on chemotherapy agents such as daunorubicin and cytarabine for more than two decades, with AML patients missing out on the revolution in targeted therapies that have improved treatment of many other haematological cancers.
The approval in AML is based on the results of the RATIFY trial, which showed a 23% reduction in the risk of death with Rydapt plus standard chemotherapy (induction with daunorubicin and cytarabine, followed by high-dose cytarabine alone) compared with placebo plus standard chemotherapy. The median overall survival in the two groups was approximately 75 months and 26 months, respectively.
The advanced SM indication was backed by two studies, with results from the larger phase II trial indicating that Rydapt-treated patients achieved an overall response rate of nearly 60%.
“Novartis is proud that we can deliver Rydapt, a breakthrough medicine, to patients with serious and hard-to-treat diseases where there are few treatment options,” said Bruno Strigini, chief executive of Novartis Oncology.
“For patients with FLT3-mutated AML, there have been no meaningful advancements in more than 25 years and with Rydapt they now have a targeted medicine that could significantly extend their lives.”
Midostaurin inhibits multiple kinases, including FLT3, which are thought to play a role in helping cancer cells to grow and multiply.
The FDA approved the drug for AML and advanced SM in April, and analysts at Credit Suisse have predicted that it could turn into a $250m-plus product – modest for Novartis but large in the contest of the highly-genericised AML market. Novartis could however see competition in future from FLT3 inhibitors in development at Astellas, Daiichi Sankyo and Arog Pharma.
Since Rydapt’s US approval, two other drugs have been cleared by the FDA for subpopulations of AML, further improving on the options available for patients.
Jazz Pharmaceuticals got the nod for Vyxeos – a fixed -dose combination of cytarabine and daunorubicin that became the first drug in the US for newly diagnosed therapy-related AML (t-AML) or AML with myelodysplasia-related changes (AML-MRC) – while Celgene/Agios claimed approval for Idhifa (enasidenib) as a treatment for AML patients with the IDH2 mutation.