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  • FDA approves Vitamin A...

FDA approves Vitamin A as breakthrough therapy for Stargardt disease and AMD

Written By : Dr. Nandita Mohan |Medically Reviewed By : Dr. Kamal Kant Kohli Published On 2021-07-16T10:15:38+05:30  |  Updated On 16 July 2021 11:14 AM IST
FDA approves Vitamin A as breakthrough therapy for Stargardt disease and AMD
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ALK-001 is a chemically-modified vitamin A which has been granted a breakthrough by the Food and Drug Administration (FDA) for the prevention and treatment of Stargardt disease and age-related macular degeneration (AMD).

Stargardt disease is the most common inherited macular dystrophy affecting both adults and children, and having a prevalence of 1 in 8000–10 000. STGD1 has an autosomal recessive mode of inheritance associated with disease-causing mutations in the ABCA4 gene. It is both clinically and genetically highly heterogeneous.

However, toxic vitamin A aggregates ("dimers") have been implicated in the pathophysiology of Stargardt and dry-AMD. Vitamin A dimers form when two molecules of vitamin A are combined. Specifically, ALK-001 is vitamin A modified by replacing hydrogen with deuterium, a stable, non-radioactive isotope of hydrogen. Substituting vitamin A with ALK-001 safely slows the rate of formation of vitamin A dimers and prevents the disease in mice.

ALK-001 is a form of vitamin A that has been chemically-modified to slow the formation of toxic vitamin A dimers in the eye and is usually consumed once a day orally.

The data has been collated from a double-masked, randomized, placebo-controlled phase 2 TEASE trial. The research assessed the safety and tolerability of ALK-001, as well as its effects on the progression of Stargardt disease.

In humans, a Phase 2 clinical trial of ALK-001 in patients with Stargardt disease has shown statistically and clinically-significant efficacy. However, the results of the study will be presented and published later.

The results from our phase 2 trial provide a strong basis for regulatory filing and approval of ALK-001 for the treatment of Stargardt disease, revealed Leonide Saad, PhD, CEO of Alkeus Pharmaceuticals. He also added that the team look forward to working with the FDA and other regulatory agencies so that can bring ALK-001 to patients as quickly as possible.

Furthermore, the Company is also investigating ALK-001 for the treatment of geographic atrophy secondary to dry age-related macular degeneration in a phase 3 study, he added.


vitamin ainvestigationpreventiontreatmentStargardt diseaseFood and drug administration
Source : FDA grants Alkeus Pharmaceuticals Breakthrough Therapy designation for ALK-001 (C20-D3-vitamin A) for the treatment of Stargardt disease. [press release]. Somerville, MA: Alkeus Pharmaceuticals, Inc.; July 14, 2021.
Dr. Nandita Mohan
Dr. Nandita Mohan

    Dr. Nandita Mohan is a practicing pediatric dentist with more than 5 years of clinical work experience. Along with this, she is equally interested in keeping herself up to date about the latest developments in the field of medicine and dentistry which is the driving force for her to be in association with Medical Dialogues. She also has her name attached with many publications; both national and international. She has pursued her BDS from Rajiv Gandhi University of Health Sciences, Bangalore and later went to enter her dream specialty (MDS) in the Department of Pedodontics and Preventive Dentistry from Pt. B.D. Sharma University of Health Sciences. Through all the years of experience, her core interest in learning something new has never stopped. She can be contacted at editorial@medicaldialogues.in. Contact no. 011-43720751

    Dr. Kamal Kant Kohli
    Dr. Kamal Kant Kohli

    Dr Kamal Kant Kohli-MBBS, DTCD- a chest specialist with more than 30 years of practice and a flair for writing clinical articles, Dr Kamal Kant Kohli joined Medical Dialogues as a Chief Editor of Medical News. Besides writing articles, as an editor, he proofreads and verifies all the medical content published on Medical Dialogues including those coming from journals, studies,medical conferences,guidelines etc. Email: drkohli@medicaldialogues.in. Contact no. 011-43720751

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