MeiraGTx Holdings Plc announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation (RPDD) to its AAV8-RK-RetGC program. This designation is for the treatment of patients with Leber congenital amaurosis due to GUCY2D mutations (LCA1), a severe retinal disease causing early-life blindness.
This marks the fourth RPDD the company has received in the last three months, following designations for AAV8-RK-AIPL1 for LCA4, AAV8-RK-BBS10 for Bardet-Biedl syndrome, and AAV5-RDH12 for RDH12-associated retinal dystrophy. RPDD is granted for therapies targeting orphan diseases affecting fewer than 200,000 patients in the U.S., with serious manifestations primarily in individuals aged 18 or younger.
The RPDD makes MeiraGTx eligible to receive a Priority Review Voucher (PRV) upon FDA approval of a Biologics License Application for AAV8-RK-RetGC. PRVs can be used by the sponsor or sold to another sponsor, with recent sales ranging from $100 million to $158 million, providing a potential source of non-dilutive capital for the company.
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