ALK-001 for Stargardt Disease Granted Breakthrough Therapy Designation

On July 14, 2021, biopharmaceutical company Alkeus Pharmaceuticals, Inc. (“Alkeus”) shared via press release that its therapy ALK-001 (C20-D3-vitamin A) received Breakthrough Therapy designation from the FDA for the treatment of Stargardt disease.


According to Alkeus, ALK-001 is:

a chemically-modified vitamin A [where hydrogen is replaced with deuterium…to slow the rate of formation of toxic vitamin A aggregates] being investigated for the prevention and treatment of Stargardt disease and age-related macular degeneration. ALK-001 is taken once a day by mouth.

Currently, there are no FDA-approved treatment options for patients with Stargardt disease. Thus, ALK-001 has the potential to fulfill an unmet need within this patient community. The Breakthrough Therapy designation was granted based on Phase 2 clinical trial data. During the study, researchers determined that ALK-001 was able to halt the formation of vitamin A aggregates in photoreceptors and protect against vision loss. More data will be reported near the end of 2021.

Overall, this designation is intended to accelerate drug development and review for drugs or biologics intending to treat serious and life-threatening conditions. Additionally, the designation is granted to products which show significant benefits over currently available treatments. As of right now, ALK-001 is the first and only therapy to have received this designation for Stargardt disease.

Stargardt Disease

Typically, ABCA4 gene mutations are considered the most common cause of Stargardt disease. This gene is usually responsible for the transportation of vitamin A. Regardless, patients with Stargardt disease experience cone and rod death within the eye. As these die, patients progressively lose vision. In most cases, vision loss is noticed during childhood, although it can also occur in adulthood. Patients often experience vision loss until their vision is 20/200 or worse. Symptoms include:

  • Color blindness
  • Bright light sensitivity
  • Loss of peripheral vision
  • Gray, black, or hazy spots in the center of vision
  • Difficulty adjusting when moving from light to dark environments
  • Blurry or distorted vision
  • Difficulty with facial recognition

Learn more about Stargardt disease.

Jessica Lynn

Jessica Lynn

Jessica Lynn has an educational background in writing and marketing. She firmly believes in the power of writing in amplifying voices, and looks forward to doing so for the rare disease community.

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