FDA Grants Abeona’s Gene Therapy for Sanfilippo Syndrome RMAT Designation

Rare Daily Staff

The U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy to Abeona Therapeutics’ experimental gene therapy for Sanfilippo syndrome, a rare lysosomal storage disease.

Sanfilippo syndromes, also known as mucopolysaccharidosis or MPS type III, is a group of four inherited genetic diseases each caused by a single gene defect. The four forms of MPS III are identified as type A, B, C or D. Each causes enzyme deficiencies that result in the abnormal accumulation of metabolic waste in cells from improperly broken down carbohydrates.

The waste fragments accumulate in the cells throughout the body causing progressive damage. In MPS III, the predominant symptoms occur due to accumulation within the central nervous system, including the brain and spinal cord, resulting in cognitive decline, motor dysfunction, and eventual death. There is no cure for MPS III and treatments are largely supportive care.

Established under the 21st Century Cures Act, the RMAT designation is an expedited program for the advancement and approval of regenerative medicine products where preliminary clinical evidence indicates the potential to address unmet medical needs for life-threatening diseases or conditions.

Similar to Breakthrough Therapy designation, the RMAT allows companies developing regenerative medicine therapies to work more closely and frequently with the FDA, and RMAT-designated products may be eligible for priority review and accelerated approval. In November 2017, the FDA expanded the RMAT designation for gene therapies.

“We are encouraged to have received the first gene therapy RMAT designation in MPS IIIA and look forward to further collaborating with the FDA to determine next steps in the development pathway for ABO-102,” said Carsten Thiel, CEO of Abeona Therapeutics. “This action further reinforces the clinical significance in the data observed in the ongoing phase 1/2 trial and the high unmet need for effective treatment options for patients suffering from MPS IIIA.”

In the ongoing phase 1/2 trial, subjects receive a single intravenous injection of ABO-102 to facilitate systemic delivery of a functional copy of the gene associated with onset and progression of MPS IIIA. Subjects are evaluated at multiple time points post-injection for safety, tolerability and efficacy. To date, 11 subjects have been enrolled.

An update on results from the trial will be presented at the American Society for Gene and Cell Therapy in May this year.

ABO-102 has been granted Rare Pediatric Disease Designation, Fast Track Designation. and Orphan Product Designation in the U.S. and Orphan Drug Designation in the European Union.

April 24, 2018
Photo: Carsten Thiel, CEO of Abeona Therapeutics