Ligand Pharmaceuticals Incorporated announced that Spectrum Pharmaceuticals, Inc., a biotechnology company with fully integrated commercial and drug development operations with a primary focus in hematology and oncology, received approval from the US Food and Drug Administration (FDA) of Evomela (melphalan) for use in two indications: 1) 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. Spectrum plans to launch Evomela with its existing sales force.

“We extend congratulations to our partner Spectrum Pharmaceuticals on achieving FDA approval for Evomela in these two indications and look forward to the commercial launch of this product,” said John Higgins, chief executive officer of Ligand. “Through partnerships such as this one, Ligand is able to share in the economic success of important, innovative new drugs across a range of indications and patient populations.”

Spectrum Pharmaceuticals licensed global development and commercialisation rights to Evomela from Ligand Pharmaceuticals in March 2013. Spectrum assumed responsibility for completing the pivotal phase 2 clinical trial, and was responsible for filing the New Drug Application (NDA). Under the license agreement, Ligand received an upfront fee and earned a $6 million milestone payment on Evomela approval, as well as royalties on net sales.

Multiple myeloma is a systemic malignancy of plasma cells that accumulate in the bone marrow, usually associated with monoclonal antibody secretion, and results in bone marrow failure and bone destruction. It is the second most common hematologic disease with nearly 30,000 new cases projected in the US in 2016 and more than 11,000 deaths annually (American Cancer Society Stats, 2016). The rate of ASCT for patients with MM is growing by approximately 3.3 per cent annually.

Melphalan is the most commonly used IV agent for high-dose conditioning for patients undergoing ASCT for MM. The current IV melphalan market is approximately $100 million annually, with predominant use in ASCT; Evomela is the only intravenous melphalan product that is approved for use in the high-dose conditioning indication.

Evomela was approved by the FDA based on its bioequivalence to the standard melphalan formulation (Alkeran) in a phase 2 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.

Evomela’s new melphalan formulation does not contain propylene glycol. The use of the Captisol technology to reformulate also contributes to the 4-hour admixture stability of Evomela at room temperature. This is in addition to the 1-hour stability of reconstituted Evomela drug product at room temperature and 24-hour stability at stability at refrigerated temperature (5°C).