Captisol-Enabled Melphalan Meets Primary Endpoint in Melanoma Trial
Product does not contain propylene glycol
A pivotal phase II study of Captisol-enabled (CE) (propylene glycol-free) melphalan has met its primary endpoint.
The study’s main objective was to determine the treatment’s overall safety and toxicity profile in patients with multiple myeloma receiving 200 mg/m2 of CE melphalan as myeloablative therapy prior to autologous stem cell transplantation. The secondary objectives evaluated the efficacy of CE melphalan in this patient population, as measured by the multiple myeloma response rate (according to International Myeloma Working Group criteria), and the rates of myeloablation and engraftment.
A new drug application is expected to be submitted to the FDA in the third quarter of 2014.
In a previous clinical study, CE melphalan met the requirements for establishment of bioequivalence to the current commercial intravenous (IV) formulation of melphalan.
CE (propylene glycol-free) melphalan is a new IV formulation of melphalan being investigated in the multiple myeloma transplant setting, for which it has been granted an "orphan drug" designation by the FDA. The new formulation eliminates the use of propylene glycol, which has been reported to cause renal and cardiac adverse effects that limit the ability to deliver higher doses of therapeutic compounds.
According to the product’s developer (Spectrum Pharmaceuticals), the use of Captisol technology to reformulate melphalan improves its stability and is expected to allow slower infusion rates and longer durations of administration, potentially enabling clinicians to achieve safely a higher dose intensity for pre-transplant chemotherapy.
Captisol is a patent-protected, chemically modified cyclodextrin with a structure designed to optimize the solubility and stability of drugs. The novel technology has enabled six FDA-approved products, including Kyprolis (carfilzomib, Onyx Pharmaceuticals), Nexterone (amiodarone, Baxter International), and Noxafil IV (posaconazole, Merck). More than 30 additional CE products are currently in clinical development.
Source: Spectrum Pharmaceuticals; April 23, 2014.