The regulatory agency approved Rydapt 25mg (ingredient: midostaurin), a treatment for rare FLT3-mutated acute myeloid leukemia (AML), as an orphan drug. AML is the most common blood cancer in adults and mutations in specific genes such as FLT3 are found in many cases of the disease, according to Novartis.

The Ministry of Food and Drug Safety on Thursday gave the nod to Rydapt in two indications -- one in combination with standard cytarabine and daunorubicin induction and high-dose cytarabine consolidation chemotherapy, and the other for aggressive systemic mastocytosis (ASM), systemic mastocytosis associated with a hematological neoplasm (SM-AHN), or mast cell leukemia.

In a trial on Rydapt in newly diagnosed AML patients in 177 medical institutions in 13 countries, including Europe, North America and Australia, patients who took the combination therapy of Rydapt and chemotherapy had statistically meaningful longer overall survival than those who received chemotherapy only.

Rydapt’s general adverse reactions include febrile neutropenia, nausea, mucositis, vomiting, headache, musculoskeletal pain, hyperglycemia, and upper respiratory

The U.S. Food and Drug Administration gave the green light to Rydapt in 2017 after a fast-track review.

Ryade is the first targeted therapy for AML in combination with chemotherapy. In January last year, the food and drug safety ministry designated the treatment as an orphan drug before the drug’s arrival in Korea.

lhs@docdocdoc.co.kr

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