Novartis wins US approval of Rydapt for rare type of AML
- Pharma giant Novartis last week secured approval from the Food and Drug Administration for its targeted therapy Rydapt (midostaurin) in two rare diseases, FLT3-mutated acute myeloid leukemia (AML) and a blood disorder called advanced systemic mastocytosis (SM).
- Rydapt is the first new drug okayed for use in treating FLT3-mutated AML in nearly three decades. The FDA approved the drug in combination with chemotherapy for patients diagnosed by a companion diagnostic made by Invivoscribe Technologies, Inc.
- Novartis' drug is also the first and only approved therapy for advanced SM, a debilitating group of disorders that can lead to organ damage, low blood counts, weight loss and severe itching.
While AML is the most common adult leukemia, the aggressive cancer also has the lowest survival rate of any adult leukemias.
The National Cancer Institute estimates 21,400 new cases of AML will be detected in the U.S. this year, with just under 11,000 deaths. Roughly a third of AML patients will have an FMS-like tyrosine kinase 3 (FLT) gene mutation, which can make the cancer grow faster, lead to higher relapse rates and lower survival rates.
While Novartis' Rydapt is the first to market for FLT3-mutated AML, other drugs are coming up through development.
Japanese drugmaker Daiichi Sankyo is currently testing quizartinib as maintenance monotherapy as well as paired with induction and consolidation chemotherapy in Phase 3 trials. On the biotech front, Arog Pharmaceuticals' crenolanib in Phase 3 in relapsed and refractory FLT3-positive AML.
The FDA designated Rydapt a Breakthrough Therapy for FLT3-mutated AML, while granting Priority Review and Fast Track status for the advanced SM indication. Novartis received Orphan Drug Designations for both indications.
In addition to the U.S., Novartis has submitted Rydapt for approval in Europe and other filings are underway globally.
Rydapt is Novartis' second cancer drug approved for use in the U.S. this year, following an FDA okay for the closely watched Kisqali (ribociclib) in mid-March.
The two approvals come roughly a year after Novartis carried out a major corporate restructuring, separating Novartis Oncology into a separate unit from Novartis Pharmaceuticals. The new organization was designed to reflect the increased emphasis on oncology for the Swiss pharma.
Generic competition has hit Novartis' top-seller Gleevec (imatinib) hard, raising the stakes for pipeline development. Immuno-oncology, unsurprising, is a particular focus.
CEO Joe Jimenez expects Novartis' CAR-T hopeful CTL019 to be "big business in oncology," and the company is also pushing ahead with PD-1 inhibitor and 15 second generation I/O agents in early studies.
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