Company Developing Treatment for Rare Childhood Blindness Gets Special FDA Designation

The FDA has granted a special designation for a drug company developing a gene therapy for the treatment of leber congenital amaurosis (LCA).

MeiraGTx received rare pediatric disease designation for its gene therapy, A001. This designation provides incentives to advance the development of rare disease treatments, including access to the FDA’s expedited review and approval programs.

Designated companies may also qualify for a voucher that can be redeemed to receive a priority review of a subsequent marketing application for a different product.
This may help lead to a treatment development of another rare disease!

LCA is an eye disorder that primarily affects the retina, which is the specialized tissue at the back of the eye that detects light and color. People with this disorder typically have severe visual impairment beginning in infancy. The visual impairment tends to be stable, although it may worsen very slowly over time.

LCA occurs in approximately 2-to-3 per 100,000 newborns and is one of the most common causes of blindness in children.

A001 is an injectable treatment, which previously has received orphan drug designation from the FDA. It is intended specifically for patients with LCA due to mutations in the RPE65 gene.

For up to 16% of LCA cases, the underlying deficit is in the RPE65 gene.

“The FDA’s decision to award rare pediatric disease designation to A001, along with the previously received orphan drug designation, underscores the urgency of developing effective therapies for rare pediatric diseases like LCA2,” said Zandy Forbes, Ph.D., President and CEO of MeiraGTx. “We are pleased with the rapid progress in our LCA2 clinical study and we are excited to be including pediatric patients with this severe genetic disorder in both Europe and the U.S.”

This is promising progress for the development of such a rare condition that affects newborns, and puts families in challenging circumstances.


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