FDA Grants Rare Pediatric Disease Designation to Taysha for GM2 Gangliosidosis Gene Therapy

Rare Daily Staff

The U.S. Food and Drug Administration granted Rare Pediatric Disease designation to Taysha Gene Therapies’ TSHA-101, an experimental AAV9-based gene therapy for the treatment of GM2 Gangliosidosis.

GM2 Gangliosidosis, also known as Tay-Sachs disease and Sandhoff disease, is a rare, neurodegenerative disease that causes progressive dysfunction of the central nervous system. There are no therapies for the treatment of the disease, and current treatment is limited to supportive care. Patients with GM2 Gangliosidosis typically succumb to disease in early childhood.

The FDA also granted TSHA-101 Orphan Drug designation. Taysha expects to initiate a clinical study of TSHA-101 by the end of the year.

Dallas, Texas-based Taysha reunites former investors and executives from gene therapy pioneer AveXis, which also got its start in Texas. TSHA-101 is the first of a pipeline of 17 gene therapy product candidates that Taysha is developing in partnership with the University of Texas Southwestern Medical Center for the treatment of monogenic diseases of the CNS in both rare and large patient populations.

“Receiving both Orphan Drug designation and Rare Pediatric Disease designation by the FDA speaks to the strength of the translational data package supporting TSHA-101 for GM2 Gangliosidosis,” said RA Session II, president, CEO, and founder of Taysha. “Furthermore, these designations also highlight the FDA’s recognition that GM2 Gangliosidosis is a devastating rare disease, and we believe this is an important milestone for the GM2 Gangliosidosis community.”

The FDA grants Rare Pediatric Disease designation for serious or life-threatening diseases with manifestations in individuals aged from birth to 18 years, including access to the FDA’s expedited review and approval process. The designation makes TSHA-101 eligible for a Rare Pediatric Disease Priority Review voucher upon approval of the therapy by the FDA. There are no approved therapies for the condition.

The vouchers can be used to reduce the time of an FDA new drug approval review to six months from ten months. The vouchers are potentially lucrative because they are transferable. Most recently, Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.

Photo: RA Session II, president, CEO, and founder of Taysha

Editor’s note: This story was updated to correct the most recent sales of a priority review voucher.