Target Health Blog

Orphan Drug Development at Target Health Inc.

January 30, 2017


Target Health is very pleased that it has prepared, submitted and obtained over 20 orphan drug designations, with 3 of these products reaching the market. There were 2 additional orphan programs in which Target Health played a major role.

One program was in cystic fibrosis, on behalf of our good friend and colleague, Dr. Tibor Sipos President and CEO of Digestive Care Inc, which was approved by FDA in 2012, without an ODD. The second program was for Regeneron, where Target e*CRF was used in the development program of Arcalyst, for the treatment of Cryopyrin-Associated Periodic Syndromes which was approved in 2008.

Ongoing clinical development programs treating orphan diseases include Dravet syndrome, Fabry disease, Growth Hormone, and Huntington’s disease.

The following is the list of orphan diseases for which Target Health obtained ODD on behalf of its clients:

Alagille syndrome; Aplastic anemia; Behcet’s disease; Burn progression in hospitalized patients; Caries prevention, head and neck cancer; Cushing’s syndrome secondary to ectopic ACTH secretion; Debridement in hospitalized patients with 3rd degree burns (EMA Approved 2012); Edema-related effects in hospitalized patients with 3rd degree burns; Fibrolamellar carcinoma (FLC); Fungal Infections; Gaucher Disease (NDA Approved 2012); Growth Hormone (Licensed to Big Pharma); Hereditary angioedema (NDA Approved 2011); Hodgkins Lymphoma; Intradialytic Hypotension (symptomatic hypotension in the immediate post-dialytic period); Invasive aspergillus infections; Multiple myeloma; Osteonecrosis of the jaw; Ovarian cancer; Rabies; Scleroderma.

For more information about Target Health contact Warren Pearlson (212-681-2100 ext. 165). For additional information about software tools for paperless clinical trials, please also feel free to contact Dr. Jules T. Mitchel or Ms. Joyce Hays. The Target Health software tools are designed to partner with both CROs and Sponsors. Please visit the Target Health Website.

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