MeiraGTx (MGTX) said Wednesday that the US Food and Drug Administration has granted its AAV8-RK-RetGC program rare pediatric disease designation for treating Leber congenital amaurosis caused by GUCY2D mutations (LCA1).
The company said it has received this fourth rare pediatric disease designation in the past three months, following similar designations for treatments targeting LCA4 retinal dystrophy, Bardet-Biedl syndrome, and RDH12-associated retinal dystrophy.
The designation is given to drugs for rare diseases affecting fewer than 200,000 patients, primarily those under 18. If a company gains approval for a treatment for a rare pediatric disease, it may receive a voucher for priority review of a future application, the company added.
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