Aridis Pharmaceuticals, Inc. (Nasdaq: ARDS) announced today that the European Medicines Agency (EMA) has granted Orphan Drug Designation to AR-501, the Company’s inhaled formulation of gallium citrate for treatment of lung infection in patients with cystic fibrosis. This past June, Aridis also received Orphan Drug Designation for AR-501 from the U.S. Food and Drug Administration (FDA) for the same condition.
“Receiving orphan designation from the EMA for AR-501 is an important step in ensuring the program is well positioned from a global regulatory development pathway standpoint as we continue to advance its ongoing Phase 1/2a clinical trial,” commented Vu Truong, PhD, Chief Executive Officer of Aridis Pharmaceuticals, Inc. “We remain on track to report data from the Phase 1 segment of the trial consisting of healthy subjects in Q1 2020 and the Phase 2a portion with cystic fibrosis subjects in Q2 2021.”
Orphan Drug Designation in Europe is available to companies developing products intended to treat a life-threatening or chronically debilitating condition that affects fewer than five in 10,000 persons in the European Union (EU). This designation allows for financial and regulatory incentives that include a 10-year period of marketing exclusivity in the EU after product approval, protocol assistance from the EMA at reduced fees during the product development phase, and access to centralized marketing authorization.
Cystic fibrosis patients often suffer from severe, persistent secondary bacterial lung infections due to their underlying lung disease which results in an immune-compromised state. AR-501 is a non-antibiotic, small molecule anti-infective in which gallium functions as an iron analog that antagonizes multiple iron-dependent pathways in microbes and thus, subverts multiple key functions in bacteria. Preclinical efficacy and safety data have demonstrated that AR-501 works synergistically with multiple antibiotics, is effective against antibiotic resistant strains, and has a low intrinsic resistance profile. AR-501 is being developed as a self-administered, weekly treatment which is being evaluated in an-going Phase 1/2a clinical trial funded by the Cystic Fibrosis Foundation (ClinicalTrials.gov Identifier: NCT03669614). – Bio Space