FDA Grants Abeona Therapeutics Orphan Drug Designation For Sanfilippo Syndrome Treatments

The FDA has announced that it has granted Abeona Therapeutics orphan drug designation for its Sanfilippo Syndromes A and B treatments. Abeona spent 2013 raising seed funds, and the company plans to start Phase I/II clinical trials for its gene based therapies later this year.

Sanfilippo Syndrome is a fatal genetic disease that stops the body from being able to break down certain sugars, which cause progressive muscular and cognitive declines beginning early on in life. Currently, there is no cure for the disease, and children who are afflicted progressively lose the ability to speak, eat, or walk.

Tim Miller, Abeona’s President and CEO, released a statement regarding the FDA’s approval. “The FDA's approval of our request for orphan drug designation is an important regulatory milestone for Abeona that supports our strategy for treating these devastating diseases,” said Miller. “The benefits include seven years of market exclusivity from product launch in the United States, tax credits for clinical research costs, and waiver of Prescription Drug User Fee Act (PDUFA) filing fees.”

There are currently no treatments for Sanfilippo Syndrome that are approved by the FDA. The orphan drug designation can only be applied to diseases or conditions which afflict fewer than 200,000 patients in the US. The designation gives drug producers a seven year exclusive marketing period in the US market.

Abeona Therapeutics is a startup company which was launched at the Nationwide Children’s Hospital, with the specific goal of developing drugs for Sanfilippo Syndromes Types A and B. The company has collaborated with numerous children’s and Sanfilippo advocacy groups, including the Children’s Medical Research Foundation Inc., Team Sanfilippo, Foundation Sanfilippo, and the Sanfilippo Children’s Research Foundation.