Spectrum Pharma Submits NDA For CE-Melphalan

(RTTNews) - Spectrum Pharmaceuticals, Inc. (SPPI) said Friday that it has submitted a New Drug Application or NDA to the U.S. Food and Drug Administration for approval of Captisol-Enabled Melphalan HCl for injection to be used as a high-dose conditioning treatment prior to stem cell transplantation in patients with multiple myeloma.

Henderson, Nevada-based Spectrum Pharma is also seeking approval for the palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate. The biotechnology company noted that the NDA submission represented an important step forward in bringing new treatment options to cancer patients.

Spectrum Pharma expects the FDA review to take about 10 months, and plans to launch the drug with its existing hematology and oncology sales force next year pending approval. The company noted that its formulation is free of propylene glycol and does not use a custom solvent for its reconstitution.

Spectrum Pharma gained global development and commercialization rights to CE-Melphalan from Ligand Pharmaceuticals Inc. (LGND) in March 2013.

The company assumed the responsibility for the pivotal clinical trial and was responsible for filing the NDA for CE-Melphalan. Under the license agreement, Ligand received a license fee and is eligible to receive milestone payments, as well as royalties following potential commercialization.

Rajesh Shrotriya, Chairman and Chief Executive Officer of Spectrum Pharmaceuticals said, "We believe the lack of propylene glycol in the preparation of CE-Melphalan eliminates the risk of the toxicities associated with that excipient. The improved solubility and stability of this novel melphalan formulation, CE-Melphalan, will make it an attractive treatment option for both transplant conditioning, and the palliative treatment of patients with multiple myeloma who cannot take oral melphalan."

Captisol-Enabled, propylene glycol-free Melphalan is an intravenous formulation of melphalan being investigated for the multiple myeloma transplant setting, for which it was earlier granted an Orphan Drug Designation by the FDA.

Spectrum Pharma noted that the Phase 2 pivotal trial evaluating CE-Melphalan was a multi-center trial evaluating safety and efficacy. The primary objective of the study was to determine the overall safety and toxicity profile in multiple myeloma patients receiving 200 mg/m2 of CE-Melphalan as myeloablative therapy prior to autologous stem cell transplantation or ASCT.

The secondary objectives evaluated the efficacy of CE-Melphalan in this patient population, as measured by Multiple Myeloma Response Rate, and the rates of myeloablation and engraftment.

The primary as well as secondary endpoints of this Phase 2 trial were met, and a comprehensive NDA was submitted to the FDA, Spectrum Pharma noted.

In a previous clinical study, CE-Melphalan met the requirements for establishment of bioequivalence to the currently approved commercial intravenous formulation of melphalan.

SPPI closed Wednesday's trading at $6.90, up $0.15 or 2.22 percent on a volume of 292,646 shares.