Receives orphan drug designation for its investigational treatment for Neurotrophic Keratitis Announced that the FDA has granted Orphan Drug Designation to Recordati Rare Disease’s investigational product REC 0559 for the treatment of neurotrophic keratitis.
REC 0559 is a low molecular weight non-peptidic human nerve growth factor (NGF) mimetic currently under global development by Recordati. REC 0559 was licensed in 2017 from MimeTech, an Italian-based company focused on the development of pharmaceutical applications for synthetic neurotrophin mimetics.
Neurotrophic keratitis is a rare degenerative corneal disease, which in its more severe forms affects less than one person out of 10,000 worldwide, and it results from deficiency of the trigeminal nerve or its branches, usually caused by surgery, neoplasia, aneurysm or facial trauma. Impairment or loss of corneal sensory innervation is responsible for corneal epithelial defects, ulcer and perforation, with progression of the disease leading to dramatic impairment to patients’ sight.Orphan drug designation applies to drugs that seek to treat rare diseases or conditions affecting fewerthan 200,000 patients in the U.S. while providing significant therapeutic advantage over existing therapies. The designation can provide development and commercial incentives for designated compounds and medicines, including eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design and an exemption from FDA user fees.