Spectrum Pharmaceuticals, Inc. An International Commercial-Stage Biotechnology Company
Spectrum Pharmaceuticals, Inc. An International Commercial-Stage Biotechnology Company
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Captisol-Enabled MelphalanSpectrum Pharmaceuticals, Inc. An International Commercial-Stage Biotechnology Company

Target Indications

Conditioning treatment prior to hematopoietic stem cell transplant (HSCT) for patients with multiple myeloma

Route of Administration

Intravenous (IV)

Mode of Action

Melphalan inhibits DNA replication and transcription causing cytotoxicity in dividing and non-dividing cells including multiple myeloma. Captisol-enabled™ melphalan is a new propylene glycol (PG)-free IV formulation of melphalan developed for patients with multiple myeloma undergoing HSCT. The formulation completely avoids the use of PG, which is used as a co-solvent in the current formulation of melphalan. The use of Captisol technology increases stability of reformulated melphalan potentially enabling clinicians to achieve a higher dose intensity of pre-transplant chemotherapy.

Status

  • Captisol-enabled PG-free melphalan was granted Orphan Drug status by the FDA for use as a high-dose conditioning regimen prior to HSCT.
  • Phase 2a clinical study results demonstrated that Captisol-enabled melphalan conditioning allows successful engraftment following autologous HSCT without unexpected adverse events. The new formulation met the requirements for establishment of bioequivalence to the current commercial intravenous formulation of melphalan.1
  • The Phase 2b pivotal trial has been completed for use as a conditioning treatment prior to autologous HSCT for patients with multiple myeloma. Captisol-enabled melphalan met the primary endpoint in this trial. More information about the clinical data is available here.
  • Spectrum Pharmaceuticals filed an NDA in December, 2014.
  • Reference:
  • 1. Aljitawi O.S., Ganguly S., Abhyankar S.H., Ferree M., Marks R., Pipkin J.D., McGuirk J.P. Phase IIa cross-over study of propylene glycol-free melphalan (LGD-353) and alkeran in multiple myeloma autologous transplantation. Bone Marrow Transplantation 2014 49:8 (1042-1045).
Spectrum Pharmaceuticals, Inc. An International Commercial-Stage Biotechnology Company

About Multiple Myeloma and Melphalan

Myeloma staging

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 abnormally high levels, which can overcrowd bone marrow and interfere with the production of normal blood cells. Myeloma cells also produce abnormally high levels of proteins that may cause multiple organ failure.

Multiple myeloma mechanism of action

There were an estimated 22,000 new cases of multiple myeloma in 2013 alone (per NCI and ACS), with the incidence of new cases continuing to increase by approximately 1.7% per year.

The current intravenous melphalan market is approximately $100 million annually, with predominant use in HSCT. The rate of autologous HSCT for patients with multiple myeloma is growing by approximately 3.3% annually.