FDA RMAT Designation Granted to Sanfilippo Syndrome Therapy

ABO-102, which has previously received rare pediatric disease designation, fast track designation, and orphan drug designation from the U.S. Food and Drug Administration (FDA), was the recipient of another acknowledgement this morning when it was granted regenerative medicine advanced therapy (RMAT) designation.

Abeona Therapeutics, Inc. announced the news about its adeno associated virus (AAV)-mediated gene therapy for the treatment of mucopolysaccharidosis type IIIA (MPS IIIA, Sanfilippo syndrome).

In November, Rare Disease Report sat down with Timothy Miller, Ph.D., President and former CEO of Abeona, to discuss the excitement surrounding the new therapeutic option for this rare disease. “These kids present with 100% enlarged livers and spleens, and a year out, we’re able to reduce that liver volume by about 85%,” he said. “That then starts to translate into, ‘Okay, well what else do you see?’ We see changes in systemic effects leading to reductions in the urinary heparin sulfate and sugars. Then, because we’ve been able to remove that underlying disease pathology, we’re able to change the organ structure and improvements can be seen in more important areas, like the brain.”

Abeona continues to engage the FDA on its ongoing Phase 1/2 trial, in which enrolled subjects are administered a single intravenous injection of ABO-102 to facilitate systemic delivery of a functional copy of the SGSH gene associated with the onset and progression of the disease. To date, 11 subjects have been enrolled.

"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, Ph.D., CEO of Abeona Therapeutics in a press release. “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 study, subjects are evaluated at multiple time points post-injection for safety, tolerability, and efficacy. The current cohorts are 1x, 2x, and 6x dose escalations.

The RMAT designation was established under the 21st Century Cures Act and 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. With it, Abeona is permitted to work more closely and frequently with the FDA, and AB-102 may now be eligible for priority review and accelerated approval.

Abeona has stated its intentions to present an update on results from the trial at the American Society for Gene and Cell Therapy (ASGCT) in Chicago in May.

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