Intraocular Injection Granted Orphan Drug Designation for Retinoblastoma

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Icon Bioscience announced that melphalan intraocular injection, the company's investigational product for the treatment of retinoblastoma, has been granted Orphan Drug Designation by the FDA. Icon's melphalan intraocular injection is formulated using its proprietary Verisome drug delivery technology, designed to deliver therapeutic levels of the drug for an extended period of time via a single intravitreal injection. Melphalan is a nitrogen mustard derivative.

Orphan Drug Designation is generally granted to drugs or biologics intended for treatment of rare diseases and disorders, ie, those affecting <200,000 people in the U.S. This designation conveys special incentives to the sponsor, including tax credit for fifty percent of the cost of clinical trials, prescription drug user fee waiver, and seven years of U.S. market exclusivity for the drug or biologic upon FDA approval.

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