Æterna Zentaris Inc, a late-stage drug development company specialized in oncology and endocrinology, has received a positive opinion for orphan medicinal product designation for its compound perifosine from the Committee for Orphan Medicinal Products (COMP) of the European Medicines Agency, for the treatment of multiple myeloma. Keryx Biopharmaceuticals, Inc, Æterna Zentaris' partner and licensee for perifosine in the North American market, already announced in September 2009 that it has received Orphan Drug Designation for perifosine for the treatment of multiple myeloma from the US Food and Drug Administration (FDA).
Juergen Engel, president and CEO of Æterna Zentaris stated, "We are very pleased with the positive opinion which is summarized by the COMP as follows: 'The preclinical data and the preliminary clinical data obtained with perifosine justifies the claim on a clinically relevant advantage based in particular on the response obtained in relapsed and refractory multiple myeloma patients. Furthermore, perifosine is intended for oral administration, compared to the currently available parenteral treatments. This supports the assumption for a major contribution to patient care with regards to convenience and avoidance of a more aggressive administration route.' "
Perifosine is a novel oral anti-cancer agent that modulates several key signal transduction pathways, including Akt, MAPK, and JNK that have been shown to be critical for the survival of cancer cells.