Abeona Therapeutics, a start-up company created around intellectual property licensed from Nationwide Children's Hospital to develop treatments for Sanfilippo Syndrome Types A and B, has been granted Orphan Drug Designations for its lead investigational therapies by the U.S. Food and Drug Administration (FDA) Office of Orphan Products Development. Following the successful close of seed financing in late 2013, Abeona is raising funds to advance its gene therapy-based clinical programs for both Sanfilippo Syndrome type A and B. Phase I/II clinical trials for both diseases are anticipated to begin in 2014.
Sanfilippo Syndrome is a deadly genetic disease resulting from the body's inability to properly break down certain sugars. Symptoms often appear in the first year of life, and the disease causes progressive muscular and cognitive decline in children after the age of two. There is no cure and currently no approved treatments for Sanfilippo Syndrome. As such, children afflicted with Sanfilippo Syndrome experience progressive loss of speech as well as the inability to eat or walk, and rarely live past their second decade of life.
"It is so exciting to see this groundbreaking science take another critical step forward towards bringing this treatment to children," said Cara O'Neill MD, whose SavingEliza.com campaign has gathered international attention in the past month for this disease and their patient advocate group, Cure Sanfilippo Foundation (USA).
"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," explained Tim Miller, Ph.D, Abeona's President/CEO. "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."
"We are encouraged by the FDA's continued recognition of the need for new treatments for rare and orphan diseases like Sanfilippo Syndrome and the designation of these gene therapies as potential therapeutic options," said Kevin Flanigan, MD, Professor of Pediatrics at Nationwide Children's hospital and principal investigator of a Sanfilippo Natural History study underway at the institution.
"There are no FDA-approved treatments for this deadly disease, and we're thrilled this designation may help accelerate the path towards approval for these much needed therapies," commented Kathleen Buckley, President of Team Sanfilippo, whose fourteen-year old son also suffers from the disease.