Alkeus Pharmaceuticals Secures FDA Rare Pediatric Disease Designation: Impact on Pediatric Treatments

Alkeus Pharmaceuticals Secures FDA Rare Pediatric Disease Designation: Impact on Pediatric Treatments

November 18, 2024 Catherine Williams - Chief Editor Health

Alkeus Pharmaceuticals Announces Designations for Gildeuretinol in Stargardt Disease

Alkeus Pharmaceuticals has received Rare Pediatric Disease and Fast Track designations from the FDA for gildeuretinol (ALK-001). This oral therapy aims to treat Stargardt disease, a serious and progressive condition that causes severe vision loss in children and adults. There is currently no approved treatment for this disease.

Michel Dahan, President and CEO of Alkeus, noted the importance of these designations. They highlight the potential for gildeuretinol to become a significant therapy for patients. The drug also holds Breakthrough Therapy and Orphan Drug designations.

The FDA’s Rare Pediatric Disease designation applies to therapeutics for serious rare diseases affecting individuals from birth to 18 years. This designation may lead to a priority review voucher upon approval. Fast Track designation expedites the development and review of drugs addressing serious conditions with significant unmet needs.

Recent data from Alkeus’ TEASE program were presented at the American Academy of Ophthalmology’s 2024 annual meeting. In a 24-month study (TEASE-1), gildeuretinol slowed the growth of atrophic retinal lesions by 21.6% compared to untreated patients. A subsequent analysis showed a 29.5% reduction in lesion growth rates.

Dr. Christine Nichols Kay also shared findings from TEASE-3, which indicated no disease progression in early-stage Stargardt patients who received gildeuretinol for two to six years. These patients also maintained stable visual acuity.

Stargardt disease affects an estimated 30,000 to 87,000 people in the U.S. It is caused by a defect in the ABCA4 protein, leading to toxic by-products that damage the retina.

The TEASE program includes four clinical studies examining the effects of gildeuretinol in Stargardt disease. TEASE-1 specifically demonstrated a significant reduction in the growth rate of retinal lesions.

For more information, visit Alkeus Pharmaceuticals.