FDA Grants Fast Track Designation to MeiraGTx Gene Therapy for Rare Eye Disease

Rare Daily Staff

The U.S. Food and Drug Administration granted MeiraGTx Fast Track designation for its experimental gene therapy AAV-RPGR for the treatment of X-linked retinitis pigmentosa due to defects in the retinitis pigmentosa RPGR gene, an inherited eye disease that causes blindness.

XLRP represents the most severe forms of retinitis pigmentosa, a group of inherited retinal diseases characterized by progressive retinal degeneration and vision loss. The most frequent mutation causing the condition is in the RPGR gene accounting for more than 70 percent of the cases of X-linked retinitis pigmentosa and 20 percent of all cases of retinitis pigmentosis.

“XLRP is a devastating condition that causes rapid progression to blindness and currently has no approved treatment options,” said Alexandria Forbes, president and CEO of MeiraGTx. “This Fast Track designation is an important milestone for both patients living with XLRP and MeiraGTx, allowing our team to communicate closely and often with the FDA as we work to bring a much-needed therapy to patients.”

The FDA’s Fast Track process is designed to expedite the development and review of drugs used to treat serious conditions and fill an unmet medical need. Fast Track designation enables the company to have early and frequent communication with the FDA throughout the drug development and review process, which may facilitate faster drug approval and patient access.

MeiraGTx is currently conducting an open label, phase 1/2 dose escalation clinical trial of AAV-RPGR in adult and pediatric patients.

The FDA has granted AAV-RPGR Orphan Drug designation and the European Medicines Agency granted Orphan Medicinal Product designation.

April 23, 2018
Photo: Alexandria Forbes, president and CEO of MeiraGTx