Several orphan drugs have exciting catalysts in the first quarter of 2011. These include Prescription Drug User Fee Act (PDUFA) action dates for ipilimumab for pretreated metastatic melanoma and vandetanib for medullary thyroid cancer (MTC) as well as results from the second Phase III trial of teduglutide for short bowel syndrome (SBS).

A decision by the US Food and Drug Administration (FDA) on the approval of ipilimumab — a fully human monoclonal antibody developed by Medarex (now part of Bristol-Myers Squibb) that targets the immune-cell receptor CTLA4 (cytotoxic T lymphocyte-associated antigen 4) to block the T-cell inhibitory pathway — is expected by 26 March. Ipilimumab was developed as a monotherapy for advanced melanoma in patients who have received prior therapy, for whom the median overall survival is 6 to 9 months. The drug failed to meet the end point of an objective response rate of more than 10% in a pivotal study. Medarex subsequently met with the FDA and the agency requested additional overall survival data to further determine the benefit of ipilimumab.

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