SuppreMol Receives U.S. Orphan Drug Designation for SM101
4/23/2010 1:52:15 PM
Martinsried/Munich, Germany, April 19, 2010 -- SuppreMol GmbH, a privately held biopharmaceutical company developing novel therapeutics for the treatment of autoimmune diseases, today announced that the FDA's Office of Orphan Products Development has granted Orphan Drug Designation (ODD) for SuppreMol's lead product SM101, a recombinant human soluble Fc? receptor IIb, for the treatment of Idiopathic Thrombocytopenic Purpura (ITP). At present, SM101 is in a Phase Ib/IIa clinical study in ITP, with results expected in early 2011. For the same indication, the molecule has already been granted Orphan Medicinal Product Designation in Europe by the EU Commission in 2007.
"We are very pleased that the FDA, too, has granted orphan drug status for SM101 for the treatment of ITP," said Peter Buckel, CEO of SuppreMol. "The decision indicates the FDA's recognition that our drug candidate may address significant unmet medical needs in patients suffering from this autoimmune disease. SM101 has already demonstrated excellent tolerability and safety in Phase I trials and is currently in a clinical study to obtain proof-of-concept."
The US Orphan Drug Act provides incentives to encourage the development of drugs for rare disease conditions affecting fewer than 200,000 persons in the United States. Orphan drug designation offers a number of potential incentives, which may include waiver of FDA user fees, study design assistance, funding for clinical studies, tax credits for clinical research and a seven-year period of U.S. marketing exclusivity if the drug receives US marketing approval. It should be noted that orphan drug designation does not limit a drug to the particular orphan disease. The drug may be developed for more common diseases in parallel or afterwards.