The U.S. Food and Drug Administration approved melphalan (Evomela) for 2 indications: use as a high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation in patients with multiple myeloma and for the palliative treatment of patients with multiple myeloma who are not eligible for oral therapy.
This is the first drug to be approved for high-dose conditioning for patients with multiple myeloma.
“The approval of EVOMELA marks the first new formulation of melphalan approved by the FDA, since its initial approval in 1964,” said Dr Parameswaran Hari, Armand J. Quick/William F. Stapp professor of hematology at the Medical College of Wisconsin, director of the Adult Blood and Marrow Transplant Program at Froedtert Hospital and the section head of hematologic malignancies and transplantation, in the Division of Hematology and Oncology in the Department of Medicine.
The approval was based on its bioequivalence to the standard melphalan (Alkeran) formulation, which was demonstrated in a phase 2 trial.
Melphalan is extensively used in the treatment of multiple myeloma and is the main drug in conditioning therapy pre-transplant. The new formulation does not contain propylene glycol and is reconstituted and admixed with normal saline. Captisol technology allows the mixture to be stable for 5 hours at room temperature following reconstitution.
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In terms of safety, the most common adverse events associated with this formulation of melphalan was decreased neutrophil and white blood cell counts in all patients, decreased lymphocyte and platelet counts (98%), diarrhea (93%), nausea (90%), fatigue (77%), hypokalemia (74%), anemia (66%), and vomiting (64%).
- FDA grants Spectrum Pharmaceuticals approval of EVOMELATM (melphalan) for injection [news release]. Spectrum Pharmaceuticals Inc. March 15, 2016. http://investor.sppirx.com/releasedetail.cfm?ReleaseID=960622. Accessed March 22, 2016.