FDA dishes out Orphan designations for bryostatin and ADS-5102

US orphan disease specialist Neurotrope (OTC: NTRP) saw its shares rise 8.9% to $1.22 on Thursday, after it revealed that it had received Orphan Drug designation by the US Food and Drug Administration for its lead proprietary drug candidate, bryostatin, in the treatment of Fragile X syndrome (FXS).

Bryostatin is a potent activator of Protein Kinase C (PKC), which the company believes is a viable therapeutic approach for the treatment of Fragile X syndrome. Operating subsidiary Neurotrope BioScience is developing bryostatin under a licensing agreement with the Blanchette Rockefeller Neuroscience Institute (BRNI).

"We are pleased to have received orphan drug designation for bryostatin for the treatment of FXS which represents an area of significant unmet need for patients and families who live with the consequences of this genetic disorder," said Charles Ramat, president and chief executive of Neurotrope, adding: "One of the key strategies for our company's future is the licensing of novel therapeutics to develop treatments for orphan diseases such as FXS. In keeping with this strategic imperative, we have also initiated preclinical work with bryostatin for the treatment of Niemann-Pick type C, a rare devastating genetic disorder in children. As we progress our clinical development programs for Fragile X and NPC we continue to work towards building a robust portfolio of drug development candidates in orphan disease indications."