FDA Grants MeiraGTx Rare Pediatric Disease Designation for Experimental Gene Therapy to Treat Achromatopsia

Rare Daily Staff

The U.S. Food and Drug Administration granted MeiraGTx Rare Pediatric Disease designation for the company’s experimental gene therapy AAV-CNGA3 for the treatment of patients with achromatopsia (ACHM) due to mutations in the CNGA3 gene.

ACHM is an inherited retinal disease that severely limits a person’s sight by preventing cone photoreceptors in the eye from functioning. Individuals with ACHM are often legally blind from birth, have extreme sensitivity to light, and experience involuntary eye movements.

AAV-CNGA3 is an experimental gene therapy treatment designed to restore cone function, delivered to the cone receptors at the back of the eye via subretinal injection.

In 2018, the FDA granted AAV-CNGA3 Orphan Drug designation and received a positive opinion from the European Medicines Agency’s Committee for Orphan Medicinal Products recommending orphan medicinal product designation.

“We are very pleased that the FDA and EMA continue to recognize the important research MeiraGTx is doing for people living with rare inherited disorders such as ACHM,” said Zandy Forbes, CEO of MeiraGTx. “Our focus remains on patient benefit and developing products that offer a cure to people living with serious diseases.”

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 AAV-CNGA3 eligible for a Rare Pediatric Disease Priority Review Voucher upon approval of the therapy by the FDA.

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, Ultragenyx and Kyowa Hakko Kirin sold their Priority Review Voucher for $80.6 million.

August 27, 2018
Photo: Zandy Forbes, CEO of MeiraGTx