FDA Grants Rare Pediatric Disease Status to EyeDNA's Gene Therapy HORA-PDE6b for Retinal Dystrophy

eyeDNA Therapeutics, a company specializing in gene therapy for inherited retinal disorders, has secured a Rare Pediatric Disease Designation (RPDD) from the U.S. Food & Drug Administration (FDA) for its gene therapy, HORA-PDE6b. This therapy aims to treat PDE6b-related Retinitis Pigmentosa (RP), a rare retinal disorder affecting up to 3,000 individuals in the United States and 4,000 in Europe. PDE6b RP is a hereditary condition caused by mutations in the PDE6b gene, leading to dysfunctional proteins that hinder the conversion of light into electrical signals in the retina, eventually resulting in vision loss. Symptoms usually manifest during childhood and can lead to blindness by midlife. Currently, there are no approved therapies for this disease.

HORA-PDE6b is an innovative gene therapy using an AAV5 vector to deliver a healthy copy of the PDE6b gene directly into the subretinal space. This approach facilitates rapid expression of the functional protein in photoreceptor cells, potentially slowing or halting the degeneration process in affected patients. Promising outcomes from a Phase I/II trial demonstrated significant improvement in visual function and a favorable safety profile at a 24-month follow-up.

The CEO of eyeDNA Therapeutics, Rodolphe Clerval, highlighted the significance of the RPDD from the FDA, which underscores the urgent need for effective treatments for this progressive and irreversible disease. The designation potentially accelerates the availability of HORA-PDE6b to patients in the U.S. and Europe, a crucial step given that early intervention could substantially alter the disease's trajectory and improve quality of life.

Rare pediatric diseases, as defined by the FDA, are serious conditions primarily affecting children under 18, with a prevalence of fewer than 200,000 cases in the U.S. The RPDD comes with additional incentives, including a Priority Review Voucher (PRV), which the FDA awards once the designated drug gains approval for pediatric use. The PRV can expedite the review process for a different drug or indication, shaving several months off the standard timeline. Moreover, these vouchers are valuable assets, with transactions exceeding $100 million, allowing the holder to sell or apply them to another project.

eyeDNA Therapeutics aims to make HORA-PDE6b accessible through an accelerated approval process, reflecting their commitment to addressing unmet medical needs in genetic retinal disorders. The company, a subsidiary of Coave Therapeutics, continues to develop its gene therapy programs, leveraging Coave's innovative ALIGATER™ platform to enhance the delivery and efficacy of genetic medicines.

In its ongoing mission, eyeDNA Therapeutics is concentrating on advancing treatments that bring meaningful change to patients suffering from inherited retinal dystrophies. The HORA-PDE6b program represents a significant step forward for those affected by PDE6b RP, opening the door to potential therapeutic breakthroughs and offering hope where previously there was none. The company's focus on precision, safety, and efficacy in genetic medicine positions it at the forefront of addressing complex challenges in gene therapy, particularly for non-hepatic tissues.

Further data from the ongoing Phase I/II trial is anticipated, with particular interest in results from younger patients who may derive the most significant clinical benefit from early intervention with HORA-PDE6b. As eyeDNA continues its research, it remains committed to transforming the landscape for those affected by debilitating retinal diseases, working towards a future where genetic conditions no longer dictate the limits of vision and life quality.