LCA10: Sepofarsen FDA Designation

15 October 2019: ProQR Therapeutics has today announced that it received Rare Pediatric Disease (RPD) designation from the  U.S. Food and Drug Administration (FDA) for sepofarsen for the treatment of Leber’s congenital amaurosis 10 (LCA10), the most common cause of blindness due to genetic disease in children.

This news follows on from their recent announcement regarding positive top-line results from the Phase 1/2 Study of Sepofarsen in LCA10 Patients, marking exciting times for ProQR and the Retina world.

The RPD designation provides priority review by the FDA to encourage treatments for rare pediatric diseases.

“We are pleased to have received rare pediatric designation from the U.S. FDA for our sepofarsen program for patients with LCA10, which is the most common cause of blindness due to genetic disease in children,” said Daniel de Boer, chief executive officer of ProQR. “This designation for sepofarsen underscores the significant unmet medical need for patients with this genetic disease causing blindness. Our goal is to advance a pipeline of programs that can treat inherited retinal diseases like LCA10 to bring medicines to patients as soon as possible.”

About sepofarsen

Sepofarsen (QR-110) is a first-in-class investigational RNA-based oligonucleotide designed to address the underlying cause of Leber’s congenital amaurosis 10 due to the p.Cys998X mutation (also known as the c.2991+1655A>G mutation) in the CEP290 gene. Sepofarsen is intended for administration via intravitreal injection. It has been granted orphan drug designation in the United States and the European Union and received fast-track designation from the FDA as well as access to the PRIME scheme by the EMA.

About Leber’s congenital amaurosis 10

Leber’s congenital amaurosis (LCA) is the most common cause of blindness due to genetic disease in children. It consists of a group of diseases of which LCA10 is the most frequent and one of the most severe forms. LCA10 is caused by mutations in the CEP290 gene, of which the p.Cys998X mutation has the highest prevalence. LCA10 leads to early loss of vision causing most people to lose their sight in the first few years of life. To date, there are no treatments approved or other products in clinical development that treat the underlying cause of the disease. Approximately 2,000 people in the Western world have LCA10 because of this mutation.

To read more about these positive top-line results of Sepofarsen from ProQR, check out the News Release on their website.

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