FDA Gives “Breakthrough” Status to Potential New Treatment for Vision Disease

Clarksburg MD – Rooted in key, early funding from BrightFocus, a proposed treatment for Stargardt disease – an inherited blinding retinal degenerative condition beginning in childhood – has received “Breakthrough Therapy Designation” from the U.S. Food and Drug Administration, allowing for expedited testing and federal review towards possible patient use.

The proposed drug, a chemically modified oral form of Vitamin A, is also being tested for use in age-related macular degeneration (AMD), including an advanced form known as geographic atrophy whose current lack of effective treatment is a major unmet need in vision health. AMD, a non-inherited disease characterized similarly to Stargardt and marked by a progressive loss of central vision, is the leading form of blindness among older adults, currently impacting the sight of 11 million Americans.

Known in scientific terms as ALK-001 (C20-D3-vitamin A), the proposed therapy is based on foundational work done by Ilyas Washington, PhD, a former Columbia University chemist and co-founder of Alkeus Pharmaceuticals, the developer of this potential drug. BrightFocus awarded Dr. Washington with one of the first research grants of his scientific career, allowing him to begin exploring a possible role for enriched Vitamin A in treating vision disease.

“BrightFocus was the first major funder of my academic lab. They gave me the opportunity – and the confidence – to believe that I could someday stop someone from losing their sight,” Dr. Washington said.

“This is an exciting, hopeful time for vision research. The work of Dr. Washington and his team shows us the power of science to save sight and the critical importance of key, early funding for bold new ideas,” said BrightFocus Foundation President and CEO Stacy Pagos Haller.