Neurotrope, Inc.  announced that Neurotrope BioScience, Inc., its wholly-owned operating subsidiary, has been granted orphan drug designation by the US Food and Drug Administration (FDA) 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. Neurotrope 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 S. Ramat, president and chief executive officer of Neurotrope, Inc. "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.”

Orphan drug designation is granted by the US FDA Office of Orphan Products Development (OOPD) to novel drugs or biologics that treat a rare disease or condition affecting fewer than 200,000 patients in the U.S. The designation provides the drug developer with a seven-year period of US marketing exclusivity, as well as tax credits for clinical research costs, the ability to apply for annual grant funding, clinical research trial design assistance and waiver of Prescription Drug User Fee Act (PDUFA) filing fees. The OOPD also works on rare disease issues with the medical and research communities, professional organizations, academia, governmental agencies, industry, and rare disease patient groups.

Also commenting on the announcement, Dr. Daniel Alkon, scientific director of BRNI and chief scientific officer of Neurotrope BioScience, Inc., stated, “We are very encouraged by the pre-clinical data we acquired in a Fragile X mouse study that suggests treatment with bryostatin can mature synapses and increase the number of synaptic connections resulting in improved learning and memory.”

FXS is the most common cause of inherited intellectual disability and the most common known genetic cause of autism or autism spectrum disorders. There is currently no FDA approved treatment for FXS available on the market today. Symptoms of FXS include moderate to severe learning disabilities, behavioral disorders, seizures and cognitive impairment. FXS is caused by a partial or a full mutation of the FMR1 gene.