Orphan Drug Strategy
A key element in Neurotrope’s strategy is to acquire drug product candidates for the treatment of orphan diseases that are neurodegenerative, developmental or inherited neurodegenerative disorders. The FDA designates a drug as orphan when it is used to treat a rare disease or condition having a prevalence of under 200,000 persons per year.
Neurotrope’s request for orphan designation of Bryostatin 1 for the treatment of patients with Fragile X Syndrome was granted by the FDA’s Office of Orphan Products Development in 2015.
A drug designated as an orphan must still undergo the regulatory review process to achieve marketing authorization, as with any other drug product in development. Sponsors of orphan drugs do, however, receive incentives from regulatory agencies and governments to encourage the development of such drugs.
Orphan Drug Incentives
A number of laws have been passed over the past 20 years to encourage Orphan Drug development. These have created many significant incentives for Orphan research.