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Melphalan – New 505(b)(2) Orphan Designation

Delcath Systems Inc. announced that the FDA has granted an additional orphan designation for melphalan for the treatment of neuroendocrine tumours metastatic to the liver.  Delcath uses a proprietary system they call the Percutaneous Hepatic Perfusion (PHP) System to deliver high doses of existing drugs directly to the site of the tumor.  Melphalan was first approved in 1964 in a tablet form under the brand name Alkeran by GSK for the palliative treatment of multiple myeloma and for the palliation of non-resectable epithelial carcinoma of the ovary.  It was subsequently developed as an injection and  approved in 1992  with an orphan indication for systemic administration for palliative treatment of patients with multiple myeloma for whom oral therapy is not appropriate.

This is a great example of taking an older drug, improving it and even getting an orphan indication.  This gets 7 years of data exclusivity upon approval.