Alkeus Pharmaceuticals Announces FDA Breakthrough Therapy Designation for ALK-001 (C20-D3-Vitamin A) in Stargardt Disease

Stargardt Disease (STGD, Image source: aao.org)

July 14, 2021 /BioonBIOON/ --Alkeus Pharmaceuticals recently announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation (BTD) to ALK-001 (C20-D3-vitamin A) for the treatment of Stargardt's disease (STGD). Notably,ALK-001 is the only drug granted Breakthrough Therapy Designation (BTD) for the treatment of Stargardt disease.Currently,There is currently no approved treatment for Stargardt disease.. Previously,FDAALK-001 has been granted Orphan Drug Designation (ODD).

BTD is an FDA new drug review pathway designed to expedite the development and review of new drugs intended to treat serious or life-threatening conditions, for which preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapies. Drugs granted BTD are eligible to receive, during development, benefits includingFDAWith closer guidance, including from senior officials, it is eligible for rolling review and potential priority review during the evaluation process, to ensure that new treatment options are made available to patients in the shortest possible time.

ALK-001 (C20-D3-vitamin A) is a chemically modified vitamin A., for the treatment of various retinal degenerative diseases. The drug is administered once daily, and clinical data indicate that,ALK-001 safely slows the progression of Stargardt disease while preserving the normal visual cycle.。

Stargardt disease is a progressiveGeneticsA degenerative retinal disease that can cause irreversible vision loss, leading to blindness. This is a rare and severe condition, with symptoms typically beginning in childhood or adolescence. Almost every patient diagnosed with this disease will become legally blind.

ALK-001 Mechanism of Action

FDAThe granting of BTD to ALK-001 is based on a Phase 2, double-blind, randomized, placebo-controlledClinical TrialData review. Data from this trial and other ongoing studies are expected to be released later this year.

In addition to Stargardt disease, ALK-001 for the treatment of patients with dry age-related macular degeneration (AMD)Clinical Trialsis also underway. In the United States, AMD is the leading cause of irreversible blindness.

Dr. Leonide Saad, Chief Executive Officer of Alkeus Pharmaceuticals, Inc., stated: "Receiving Breakthrough Therapy Designation in the field of Stargardt disease is a transformative milestone. Our Phase 2Clinical TrialThe results provide a strong rationale for the regulatory submission and approval of ALK-001 for the treatment of Stargardt disease. We look forward toFDAcollaborate with other regulatory authorities so that we can bring ALK-001 to patients as soon as possible.” (Bioon.com)

Original Source:FDA Grants Alkeus Pharmaceuticals Breakthrough Therapy Designation for ALK-001 (C20-D3-vitamin A) for the Treatment of Stargardt Disease