RedHill Biopharma Announces FDA Orphan Drug Designation for RHB-204 for the Treatment of NTM Infections

RHB-204 Phase 3 study in pulmonary NTM infections is planned to be initiated in the coming weeks
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Orphan Drug Designation, along with RHB-204’s FDA QIDP priority designation, will extend U.S. potential market exclusivity to a total of 12 years

TEL AVIV, Israel and RALEIGH, N.C., Oct. 14, 2020 (GLOBE NEWSWIRE) -- RedHill Biopharma Ltd. (Nasdaq: RDHL) (“RedHill” or the “Company”), a specialty biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to RHB-204 for the treatment of nontuberculous mycobacteria (NTM) disease.

“NTM infections are resistant to most antibiotics and are extremely challenging to treat. With no FDA-approved first-line therapy available to patients today, there is a tremendous urgent need for new treatments for this debilitating and potentially fatal infectious disease,” said Patricia Anderson, RedHill’s VP Regulatory Affairs. “Along with the QIDP designation already granted to RHB-204 by the FDA, the Orphan Drug Designation extends potential market exclusivity to a total of 12 years.”

A Phase 3 study to evaluate the safety and efficacy of RHB-204 in patients with pulmonary NTM infections caused by Mycobacterium avium Complex (MAC) is planned to be initiated in the coming weeks in the U.S.

The multi-center, randomized, double-blind, placebo-controlled, parallel-group Phase 3 study, under ongoing discussion with the FDA, is planned to be conducted at up to 40 sites across the U.S. and aims to enroll 125 patients, randomized at a 3:2 ratio to receive either RHB-204 or placebo. The study is planned to evaluate the safety and efficacy of RHB-204, evaluating patient-reported outcomes and sputum culture conversion (SCC) by Month 6 of treatment with RHB-204, compared to placebo, and patients will continue to receive treatment for 12 months from SCC.

The FDA grants orphan status to drugs intended to treat rare disorders that affect fewer than 200,000 people in the U.S. The designation provides various development incentives to the drug developer, including extended market exclusivity upon FDA approval, prescription drug user fee (PDUFA) waivers and tax credits for qualified clinical testing.

RHB-204 was previously granted a Qualified Infectious Disease Product (QIDP) designation by the FDA, providing for eligibility for Fast-Track development, NDA priority review and a five-year extension of U.S. market exclusivity, if approved. The Orphan Drug Designation extends U.S. market exclusivity for RHB-204 by an additional seven years, for a potential total of 12 years upon FDA approval.