Conditioning treatment prior to autologous stem cell transplant for patients with multiple myeloma
Route of Administration
Mode of Action
Captisol-enabled™ melphalan is a new IV formulation of melphalan that has the potential to offer multiple advantages for clinicians and patients in the multiple myeloma transplant setting. The formulation completely avoids the use of propylene glycol, which is used as a co-solvent in the current formulation of melphalan and has been reported to cause renal and cardiac side-effects that limit the ability to deliver higher quantities of intended therapeutic compounds. The use of Captisol technology to reformulate melphalan is anticipated to allow for longer administration durations and slower infusion rates, potentially enabling clinicians to avoid reductions and safely achieve a higher dose intensity of pre-transplant chemotherapy.
Captisol-enabled melphalan was granted Orphan Drug status by the FDA for use as a high-dose conditioning regimen prior to hematopoietic progenitor (stem) cell transplantion. If approved, the propylene glycol-free formulation of melphalan will be the first product approved for this indication.
Phase 2B (Pivotal Trial)
Currently melphalan has met the endpoints in a trial for use as a conditioning treatment prior to autologous stem cell transplant for patients with multiple myeloma. The Company plans to file an NDA in 2014.
Spectrum has assumed responsibility for this ongoing pivotal clinical trial and will be responsible for filing an NDA, which is anticipated in 2014.
In a previous Phase 2 study, Captisol-enabled melphalan seemed to demonstrate an acceptable safety profile, and it met the requirements for establishment of bioequivalence to the current commercial intravenous formulation of melphalan.
About Multiple Myeloma and Melphalan
Multiple myeloma is a cancer of plasma cells, a type of white blood cell present in the bone marrow that produces antibodies. In multiple myeloma, a group of plasma cells (myeloma cells) becomes cancerous and multiplies, raising the number of plasma cells to a higher-than-normal level, which can crowd out normal blood cells and lead to abnormally high proteins in the blood or urine.
There are an estimated 22,000 new cases in 2013 (per NCI and ACS) of multiple myeloma in the United States each year, with the incidence of new cases increasing by approximately 1.7% per year.
The current intravenous melphalan market is approximately $100 million annually, with predominant use in stem cell transplants. The rate of autologous stem cell transplants for patients with multiple myeloma is growing by approximately 3.3% annually.