eyeDNA Therapeutics receives FDA Rare Pediatric Disease Designation for HORA-PDE6b, a novel gene therapy for PDE6b retinitis pigmentosa
The company is advancing its Phase I/II trial and exploring accelerated approval pathways in the US and Europe.
(Image credit: AdobeStock/digitalmio13)
eyeDNA Therapeutics announced it has been granted a Rare Pediatric Disease Designation (RPDD) by the FDA for HORA-PDE6b, its novel gene therapy for patients with inherited retinal dystrophy (IRD) caused by mutations of the PDE6b gene.
PDE6b Retinitis Pigmentosa (PDE6b RP) is a rare inherited retinal dystrophy that affects an estimated 3000 individuals in the United States and 4000 in Europe, accounting for 1% to 3% of all cases of retinitis pigmentosa (RP). The disease is caused by mutations in the PDE6B gene, resulting in a dysfunctional PDE6b protein. This disruption impairs the conversion of light into electrical signals, leading to the progressive loss of photoreceptors and vision. Symptoms typically begin in childhood and often progress to blindness by midlife. At present, no approved treatments are available for PDE6b RP.¹
HORA-PDE6b is an AAV5-based gene therapy designed to deliver a functional, non-mutated copy of the human PDE6B gene into the subretinal space. This approach facilitates rapid transgene expression and the synthesis of functional PDE6b protein in photoreceptor cells. By providing these cells with a functional protein, HORA-PDE6b has the potential to significantly delay or even halt retinal degeneration in patients with PDE6b deficiency.
eyeDNA presented positive 24-month follow-up results1 from its Phase I/II study (NCT03328130) evaluating the safety and efficacy of HORA-PDE6b at the Association for Research in Vision and Ophthalmology (ARVO) 2024 meeting in Seattle, WA, USA in May.2
According to the company, it is exploring the optimal paths to making HORA-PDE6b available to PDE6b RP patients in the US and in Europe via an accelerated approval process.
Rodolphe Clerval, CEO of eyeDNA, noted that PDE6b retinitis pigmentosa is a progressive and irreversible inherited degenerative disease that leads to significant visual impairment and blindness with currently no treatments available.
“This Rare Pediatric Disease Designation (RPDD) for HORA-PDE6b from the FDA recognizes the urgent need for effective treatments for this devastating disease where early treatment could slow or halt disease progression and have a profound impact on patients’ quality of life,” Clerval said in a statement. “We look forward to providing further data updates from our ongoing Phase I/II trial with HORA-PDE6b, and we are particularly excited to see initial results in younger patients where HORA-PDE6b could produce the greatest clinical benefit.”
Rare pediatric diseases are defined by the FDA as serious or life-threatening conditions that primarily affect children under 18 years of age and have fewer than 200,000 cases in the United States. Companies that receive a Rare Pediatric Disease Designation (RPDD) are eligible for a Rare Pediatric Disease Priority Review Voucher (PRV) from the FDA when their designated drug is approved for the associated indication in the pediatric population.1
According to the company, the PRV allows the recipient company to request FDA priority review for a New Drug Application (NDA) or Biologics License Application (BLA) for a different product or indication, reducing review time and accelerating approval and market entry by at least four months. The voucher can be used by the original recipient or sold to another company, with PRVs recently fetching prices exceeding $100 million.
