Daily tablet shows promise as first treatment for Stargardt disease

A new drug for Stargardt disease, called gildeuretinol (ALK-001), has received special recognition from the Food and Drug Administration in the US, highlighting its potential to treat the condition.

The drug, taken as a daily tablet, could become the first treatment for Stargardt disease, after showing promising results in phase I and phase II clinical trials.

Alkeus Pharmaceuticals, which has developed gildeuretinol, has received two recognitions (Rare Paedriatic Disease and Fast Track) for the treatment from the FDA, to help speed up its approval process.

If gildeuretinol is approved, Alkeus could then receive a Priority Review Voucher, which would speed up the review process for the drug. 

How does the drug work?

Stargardt disease is usually caused by mutations in a gene called ABCA4. This gene is important for how the body uses vitamin A, which is essential for forming light-sensitive molecules in the retina (the light-sensitive layer of tissue at the back of the eye). These molecules enable vision, but their formation generates fatty by-products as waste.

The ABCA4 gene provides the code for a protein that cleans up these by-products to maintain retinal health. In people with Stargardt disease, mutations in the ABCA4 gene lead to a defective or missing protein, preventing this cleanup process from working properly. As a result, harmful waste accumulates in the retina, causing damage and vision loss over time.

Gildeuretinol helps by reducing the buildup of these harmful vitamin A by-products, which slows the damage and helps protect the retina from further damage, slowing the progression of disease.

Clinical trial results

In a two-year study, gildeuretinol slowed retinal damage by more than 20% in patients, compared to those who received no treatment. Another study with early-stage patients found that the drug appeared to stop the disease from worsening, with participants maintaining stable vision for up to six years.

The drug was also safe and well-tolerated, with no serious side effects reported.

Dr Christine Nichols Kay, a retinal specialist involved in the trials, said: “These results are a major step forward for patients with Stargardt disease.”

Gildeuretinol for late-stage dry age-related macular degeneration

Alkeus Pharmaceuticals has also reported promising results from its study of gildeuretinol for geographic atrophy (GA), a late-stage form of dry AMD.

Geographic atrophy is characterised by patches of retinal damage that appear like islands on an eye scan.

The study found a 15.3% reduction in the growth of GA lesions, areas where retinal cells have been damaged or died, over 24 months compared to a placebo. During the trial patients also lost 4.4 fewer letters on visual acuity tests. The drug shows potential to slow disease progression, and Alkeus is planning further trials. 

We will publish further updates when they become available.