AceLink Therapeutics, a biopharmaceutical company developing therapies for genetic diseases, has received Orphan Drug Designation (ODD) from the U.S. Food and Drug Administration (FDA) for AL01211 as a treatment for Fabry disease.
“Receiving an FDA Orphan Drug Designation further validates AL01211 as a novel, once-daily orally administered GCS inhibitor as a potential treatment to improve the quality of life of patients suffering from Fabry disease,” said Marvin Garovoy, M.D., chief medical officer of AceLink Therapeutics.
“AL01211 was selected based on its unique properties, including high potency and inability to cross the blood-brain barrier, to more effectively treat the peripheral organs affected by Fabry disease.”
In an effort to expand future access to AL01211, AceLink Therapeutics plans to conduct Phase II clinical studies involving Fabry disease patients who are either naive to enzyme replacement therapy or are seeking an alternative to enzyme replacement therapy.
“Our objective is to rapidly advance AL01211, so we have initiated discussions with potential commercial partners that are equally committed to ensuring that Fabry disease patients around the world have access to a novel, once-a-day oral therapy with the potential to dramatically improve their quality of life,” said Jerry Shen, Ph.D., Chief Executive Officer of AceLink Therapeutics.
