RedHill Biopharma (RDHL) Granted EU Orphan Drug Designation for RHB-204

RedHill Biopharma Ltd. (Nasdaq: RDHL) ("RedHill" or the "Company"), a specialty biopharmaceutical company, today announced that the European Commission has granted Orphan Drug Designation to RHB-2041 for the treatment of nontuberculous mycobacteria (NTM) disease, following a positive opinion recommendation by the European Medicines Agency's (EMA) Committee for Orphan Medicinal Products (COMP).

The EMA grants Orphan status to treatments of a life-threatening or chronically debilitating condition that is rare (affecting not more than five in 10,000 people in the European Union)2. Orphan Designation provides for free protocol assistance (scientific advice from the EMA), potential reductions in fees, and eligibility for 10 years post-launch market exclusivity.

"NTM infections are increasing across the world and they are notoriously difficult to treat. There are no approved first-line stand-alone therapies in the U.S, EU, and Japan, highlighting the significant need for new options to treat this challenging and debilitating infectious disease," said Patricia Anderson, RedHill's SVP Regulatory Affairs. "The granting of EU Orphan Designation is important and brings significant developmental benefits, in addition to the provision for a potential 10 years of EU post-approval market exclusivity, which adds to the strong exclusivity secured in the U.S."

A U.S. Phase 3 study is ongoing to evaluate the efficacy and safety of RHB-204 in adults with pulmonary NTM disease caused by Mycobacterium avium complex (MAC) infection (NCT04616924). The study protocol provides for 6 months co-primary endpoint of sputum culture conversion (SCC) and clinical outcome (patient-reported outcomes - PRO) in a randomized placebo-controlled design, followed by open label active treatment with RHB-204 for 12 months from conversion.

The Company is advancing discussions with prospective partners for RHB-204 across multiple territories.

You May Also Be Interested In

• TG Therapeutics (TGTX) Announces Additional Data Presentations for BRIUMVI in Multiple Sclerosis
• Vertex Pharma (VRTX) Announces Advancements of Suzetrigine (VX-548) in Acute and Neuropathic Pain
• Flux Power (FLUX) Appoints Mark Leposky to its Board