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Novartis gets speedy FDA review for midostaurin

Leukaemia treatment could become first targeted therapy for 25 years

NovartisNovartis has picked up an FDA priority review for leukaemia drug candidate midostaurin, setting up a possible US approval for the drug in the first half of 2017.

The US regulator has started its review of midostaurin (PKC412) for newly-diagnosed FLT3-mutated acute myeloid leukaemia (AML) and advanced systemic mastocytosis and - if all goes according to plan - the drug could become the first targeted therapy for a cancer whose treatment has remained unchanged for a quarter of a century.

The European Medicines Agency (EMA) has also started a review of the drug, and both regulators are assessing it alongside a companion diagnostic that can be used to determine FLT3 status.

The treatment of AML is still limited to conventional chemotherapy, which achieves long-term survival rates of 25-50% in patients below the age of 60, and only 5-15% in older patients.

In trials, the drug has been shown to improve overall survival by 23% when added to standard chemotherapy in AML patients whose cancer cells exhibit FLT-3 mutations, earning it a breakthrough designation from the FDA.

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. FLT3 mutations are seen in around a third of all patients, and are associated with a poorer prognosis.

Analysts at Credit Suisse are predicting sales of around $260m for the drug in 2019 if it claims approval for both AML and ASM. That would make it a relatively minor product for Novartis but still a major player in the AML market, which is predicted to be worth around $900m in 2020 by market research firm GBI.

Prospects for improved AML drugs are looking increasingly bright, however, with several other candidates coming through the pipeline. These include improved chemotherapies such as CPX-351 (liposomal cytarabine and daunorubicin) from Celator Pharmaceuticals as well as targeted drugs such as Ambit Biosciences' FLT3 inhibitor quizartinib and Roche's new BCL-2 inhibitor Venclexta (venetoclax).

Article by
Phil Taylor

15th November 2016

From: Regulatory

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