FDA grants rare pediatric disease, fast track designations to Stargardt disease therapy
Click Here to Manage Email Alerts
Key takeaways:
- Gildeuretinol, an oral therapy for Stargardt disease, received rare pediatric disease and fast track designations from the FDA.
- The designations are backed by positive safety and efficacy findings.
The FDA granted rare pediatric disease and fast track designations to gildeuretinol, an investigational oral therapy for the treatment of Stargardt disease, according to a press release from Alkeus Pharmaceuticals.
“There is enormous unmet need in Stargardt disease because there is currently no approved treatment available to patients,” Seemi Khan, MD, MPH, MBA, chief medical officer of Alkeus Pharmaceuticals, told Healio. “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.”
The designations are supported by data from the TEASE-1 and TEASE-3 clinical studies. In TEASE-1, a placebo-controlled, double-masked, randomized 24-month study, gildeuretinol, also known as ALK-001, demonstrated a 21.6% slowed growth rate of atrophic retinal lesions area compared with untreated patients. It also showed a 29.5% growth rate reduction of atrophic lesions in a sensitivity analysis using non-transformed values, according to the release.
In TEASE-3, patients with early-stage Stargardt disease treated with gildeuretinol showed no disease progression and were asymptomatic while being treated for between 2 and 6 years, with “relatively stable visual acuity,” the release said.
Gildeuretinol had a favorable safety profile in both studies.
“The FDA grants rare pediatric disease designation to therapeutics intended to treat serious or life-threatening rare diseases, and Alkeus may be eligible to receive a priority review voucher, which can significantly speed up the approval process for any future drug application. Fast track designation signals that the drug is a high priority to address an unmet medical need,” Khan said. “Our top priority is to prepare [a new drug application] submission for Stargardt disease as soon as possible given the urgency for the patients and their families.”
The FDA previously granted breakthrough therapy and orphan drug designations to gildeuretinol.
Collapse
Read more about
Click Here to Manage Email Alerts