Ligand Pharmaceuticals Incorporated announces that its partner Spectrum Pharmaceuticals has submitted a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for Captisol-enabled Melphalan (CE-Melphalan) HCl for injection (propylene glycol-free) for use as a high-dose conditioning treatment prior to hematopoietic progenitor (stem) cell transplantation in patients with multiple myeloma. Spectrum is also seeking approval for the palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate.
According to Spectrum Pharmaceuticals, the Captisol-enabled Melphalan NDA was filed under a 505(b) application and Spectrum expects FDA review to take approximately 10 months. Spectrum plans to launch this drug with its existing hematology/oncology sales force next year pending approval.
“We congratulate the team at Spectrum Pharmaceuticals on their development progress,” said John Higgins, Ligand’s President and Chief Executive Officer. “With a specialty sales force already in place, we look forward to Spectrum’s potential launch and commercial success with this valuable asset.”
Ligand licensed global development and commercialization rights to CE-Melphalan to Spectrum in 2013. Spectrum assumed the responsibility for a pivotal clinical trial and was responsible for filing the NDA. Under the license agreement, Ligand received a license fee and is eligible to receive revenue from Captisol material sales, milestone payments, as well as royalties on net sales following potential commercialization.
According to previous announcements by Spectrum, the Phase 2 pivotal trial evaluating CE-Melphalan was a multicenter 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/m 2 of CE-Melphalan as myeloablative therapy prior to autologous stem cell transplantation (ASCT). The secondary objectives evaluated the efficacy of CE-Melphalan in this patient population as measured by Multiple Myeloma Response Rate (according to International Myeloma Working Group [IMWG] criteria), and the rates of myeloablation and engraftment. The primary as well as secondary endpoints of this Phase 2 trial were met.
In a previous clinical study, CE- Melphalan met the requirements for establishment of bioequivalence to the currently approved commercial intravenous formulation of melphalan. This demonstrated bioequivalence of CE-Melphalan may facilitate the use of this new more stable, propylene glycol-free Captisol formulation of melphalan following the potential approval of the NDA.
