The US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to AceLink Therapeutics’ AL01211 to treat Fabry disease. 

A glucosylceramide synthase (GCS) inhibitor, AL01211 has high potency, increased selectivity and other beneficial drug properties that support once-a-day oral dosing.

Additionally, it provides a much-required oral small molecule treatment as a substitute to existing therapies for Fabry disease, which need repeated intravenous dosing. 

AceLink plans to begin Phase II clinical trials of AL01211 in Fabry disease patients next year.

This trial will enrol such patients who are either naive to enzyme replacement treatment or are looking for a substitute for enzyme replacement therapy. 

AceLink Therapeutics chief medical officer Marvin Garovoy said: “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. 

“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.”

The company has commenced talks with possible commercial partners who share its commitment to guarantee that patients with Fabry disease globally obtain access to a new, once-daily oral treatment, which can boost the quality of life.

Established in 2018, AceLink focuses on the development of therapies for genetic ailments with unmet needs. 

In March, the company received clearance from the Chinese National Medical Products Administration (NMPA) and Center for Drug Evaluation (CDE) for the Investigational New Drug (IND) application of AL01211.