FDA approves Evomela (captisol-enabled melphalan) for multiple myeloma, as conditioning treatment prior to stem cell transplantation and as a palliative treatment- Spectrum Pharmaceuticals

The FDA has granted approval of Evomela (captisol-enabled melphalan), from Spectrum Pharmaceuticals, for use in two indications: 1) use as a high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation (ASCT) in patients with multiple myeloma (MM), and 2) for the palliative treatment of patients with MM for whom oral therapy is not appropriate. This is the first product to be FDA-approved for the high-dose conditioning indication in MM.

Evomela was approved by FDA based on its bioequivalence to the standard melphalan formulation (Alkeran) in a Phase II clinical study (Aljitawi et al, Bone Marrow Transplant, 2014) via the 505(b)(2) regulatory pathway. Evomela has been granted Orphan Drug Designation by the FDA for its use as a high-dose conditioning regimen for patients with MM undergoing ASCT.

Comment: Evomela is a conditioning treatment for use in autologous transplant for patients with multiple myeloma and is a new IV formulation of melphalan (currently sold as Alkeran for Injection). This formulation completely avoids the use of propylene glycol, which has been used as a co-solvent in other formulations and has been reported to cause renal and cardiac side effects that limit the ability to deliver higher quantities of intended therapeutic compounds.