RARE Daily

FDA Approves Bristol Myers Squibb’s Vidaza for Rare Pediatric Blood Cancer

May 24, 2022

The U.S. Food and Drug Administration approved Bristol Myers Squibb’s azacytidine, originally marketed as Vidaza by Celgene, for pediatric patients with newly diagnosed juvenile myelomonocytic leukemia, a rare blood cancer.

Efficacy was evaluated in AZA-JMML-001, an international, multicenter, open-label study to evaluate the pharmacokinetics, pharmacodynamics, safety, and activity of azacitidine prior to hematopoietic stem cell transplantation (HSCT) in 18 pediatric patients with juvenile myelomonocytic leukemia (JMML). Patients were treated with intravenous azacitidine daily on Days 1-7 of a 28-day cycle for a minimum of 3 cycles and a maximum of 6 cycles, provided patients did not have disease progression or were ready for HSCT between Cycles 4 and 6.

The main efficacy outcome measures were clinical complete remission (cCR) or clinical partial remission (cPR) according to the International JMML response criteria at 3 months (Cycle 3, Day 28). Responses must have been sustained for at least 4 weeks either in the 4-week period preceding or succeeding Cycle 3, Day 28. A total of 9 patients had confirmed clinical responses. Of these 9 patients, there were 3 cCR and 6 cPR. The median time to response was 1.2 months (range 0.95-1.87 months). The proportion of patients undergoing HSCT was 94 percent and the median time to HSCT was 4.6 months (range 2.8-19 months).

Most common adverse reactions (>30 percent) occurring in pediatric patients with JMML were pyrexia, rash, upper respiratory tract infection, and anemia.

This review used the Assessment Aid, a voluntary submission from the applicant to facilitate the FDA’s assessment. The FDA approved this application 1 month ahead of the FDA goal date.

The application was granted priority review and breakthrough designation.

Author: Rare Daily Staff

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