Midostaurin (Rydapt®) given ‘positive opinion’ by the EMA CHMP for use in newly diagnosed FLT3+ AML patients

On 21st July 2017, the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) gave a positive opinion recommending approval for midostaurin (Rydapt®), a multi-kinase inhibitor, for the treatment of newly diagnosed Acute Myeloid Leukemia (AML) patients who are Fms Like Tyrosine Kinase 3 (FLT3) mutated-positive (FLT3+).1

This positive opinion follows the recent U.S. Food and Drug Administration (FDA) approval of Rydapt® for FLT3-mutated AML on 28th April 2017, which was reported here.

This recommendation of approval for midostaurin by the EMA CHMP was based on results from the phase III RATIFY trial (NCT00651261), which showed midostaurin in combination with standard chemotherapy significantly prolonged the survival of young adult patients with FLT3 mutated AML. More results of the study were reported here.2

The drug manufacturers, Novartis, highlighted that if Rydapt® is approved by the European Commission (EC), it will be “indicated in combination with standard daunorubicin and cytarabine induction and high-dose cytarabine consolidation chemotherapy, and for patients in complete response, followed by Rydapt® single agent maintenance therapy, for adult patients with newly diagnosed AML who are FLT3 mutation-positive”.

  1. GlobeNewswire: Novartis receives positive CHMP opinion for Rydapt® (midostaurin) for newly diagnosed FLT3-mutated acute myeloid leukemia (AML) and three types of advanced systemic mastocytosis (SM). 2017 Jul 21. https://globenewswire.com/news-release/2017/07/21/1055640/0/en/Novartis-receives-positive-CHMP-opinion-for-Rydapt-midostaurin-for-newly-diagnosed-FLT3-mutated-acute-myeloid-leukemia-AML-and-three-types-of-advanced-systemic-mastocytosis-SM.html [Accessed 2017 Jul 24].
  2. Stone R.M. et al. Midostaurin plus Chemotherapy for Acute Myeloid Leukemia with a FLT3 Mutation. N Engl J Med. 2017 Jun 23. DOI: 10.1056/NEJMoa1614359. [Epub ahead of print].
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