Qurient said Thursday that the U.S. Food and Drug Administration has granted orphan drug designation (ODD) to Telacebec, a Buruli ulcer treatment.

Qurient’s Buruli ulcer treatment has won an ODD status from the U.S. FDA. (Qurient)
Qurient’s Buruli ulcer treatment has won an ODD status from the U.S. FDA. (Qurient)

Orphan drugs show promise in the treatment, prevention, or diagnosis of orphan diseases. The designation provides tax and license application fee exemption and grants a monopoly for seven years if it becomes the first drug recognized in its field.

The designation comes after the company has been developing the candidate as a treatment for resistant tuberculosis. However, the company decided to expand its indication as they confirmed Telacebec’s therapeutic effects in treating Buruli ulcers through a researcher-led clinical trial.

“We published the results of the trial in renowned international medical journals, such as the Nature Communication and Antimicrobial Agents and Chemotherapy, in 2019 and 2020,” the company said. With the ODD designation, Qurient plans to launch global phase 2 trials this year through a collaboration with the World Health Organization (WHO).

“As Burly ulcer is a rare disease with a small number of patients, the company expects that it will be able to receive approval for a small-scale clinical trial,” a company official said. “Therefore, we will promote the clinical trials separately from the ongoing trials for resistant tuberculosis.”

Telacebec is a candidate substance that Qurient licensed from Institut Pasteur Korea as a treatment for resistant tuberculosis.

Buruli ulcer is a skin necrosis disease caused by infection with mycobacterium ulcerans.

The disease mainly occurs in Africa, Australia and South America, and is a refractory disease with no suitable treatment options.

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