FDA Grants Fast Track and Rare Pediatric Disease Designations to Gildeuretinol for Stargardt Disease

• The FDA has granted rare pediatric disease and fast track designations to gildeuretinol, an oral therapy being developed by Alkeus Pharmaceuticals for Stargardt disease.
• Clinical data from the TEASE-1 study showed gildeuretinol slowed the growth rate of atrophic retinal lesions by 21.6% compared to placebo over 24 months.
• In the TEASE-3 study, early-stage Stargardt disease patients treated with gildeuretinol showed no disease progression and maintained relatively stable visual acuity for 2-6 years.
• These designations may expedite the approval process for gildeuretinol, addressing a significant unmet need as there are currently no approved treatments for Stargardt disease.

The FDA has granted rare pediatric disease and fast track designations to gildeuretinol, an investigational oral therapy developed by Alkeus Pharmaceuticals for the treatment of Stargardt disease. These designations, announced in a press release by Alkeus Pharmaceuticals, highlight the potential of gildeuretinol to address a critical unmet need in Stargardt disease, a genetic eye disorder that causes progressive vision loss. There are currently no approved treatments for Stargardt disease, making the development of gildeuretinol a significant step forward.

Clinical Evidence Supporting the Designations

The FDA's decision is supported by data from the TEASE-1 and TEASE-3 clinical studies. The TEASE-1 study, a 24-month, placebo-controlled, double-masked, randomized trial, demonstrated that gildeuretinol slowed the growth rate of atrophic retinal lesions by 21.6% compared to untreated patients. A sensitivity analysis using non-transformed values showed a 29.5% reduction in the growth rate of atrophic lesions.

In the TEASE-3 study, patients with early-stage Stargardt disease treated with gildeuretinol showed no disease progression and remained asymptomatic for between 2 and 6 years, with relatively stable visual acuity. Both studies indicated a favorable safety profile for gildeuretinol.

Significance of the FDA Designations

According to Seemi Khan, MD, MPH, MBA, chief medical officer of Alkeus Pharmaceuticals, the rare pediatric disease designation may allow Alkeus to receive a priority review voucher, potentially accelerating the approval process for future drug applications. The fast track designation signifies that the drug is a high priority for addressing an unmet medical need.

"There is enormous unmet need in Stargardt disease because there is currently no approved treatment available to patients," said Dr. Khan. "We’re very encouraged about oral gildeuretinol’s potential to be the first therapy in Stargardt. We have generated compelling data in which gildeuretinol has shown significant efficacy and a favorable safety profile."

Gildeuretinol: Mechanism and Previous Designations

Gildeuretinol (ALK-001) is designed to reduce the dimerization of vitamin A, which is implicated in the pathogenesis of Stargardt disease. The FDA had previously granted breakthrough therapy and orphan drug designations to gildeuretinol.

Next Steps for Alkeus Pharmaceuticals

Alkeus Pharmaceuticals is prioritizing the preparation of a new drug application (NDA) submission for Stargardt disease. The company aims to expedite the availability of gildeuretinol to patients and their families, given the urgency of the condition.