F2G Ltd, a UK- and Austria-based biotech developing novel therapies for life-threatening systemic fungal infections, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead first-in-class candidate, olorofim (formerly F901318) for “treatment of invasive aspergillosis” and for “treatment of lomentospora/scedosporium infections”1, which are invasive mould infections, for which there is significant unmet medical need. This development follows F2G’s announcement in November 2019 of olorofim as the first antifungal agent to be granted Breakthrough Therapy designation by the FDA.
Olorofim is currently being investigated in an open-label single-arm Phase 2b study (ClinicalTrials.gov Identifier: NCT03583164) in patients with proven invasive fungal disease (IFD) or probable invasive aspergillosis (IA) with limited treatment options (refractory disease, resistance, or intolerance to available agents). Olorofim has been well tolerated across more than 17 years of patient dosing days with a median therapy duration of 12 weeks. Preliminary efficacy and safety data from this study were provided to the FDA as part of the ODD submission.
Orphan Drug status is intended to advance drug development for rare diseases. The FDA provides Orphan Drug Designation to drugs and biologics that demonstrate promise for the diagnosis and/or treatment of rare diseases or conditions that affect fewer than 200,000 people in the U.S. The designation can provide development and commercial incentives for designated compounds and medicines, including eligibility for a seven-year period of market exclusivity in the U.S. after product approval, FDA assistance in clinical trial design and an exemption from FDA user fees.
Commenting on the news, Ian Nicholson, CEO of F2G Ltd, said: “As we continue to work towards our goal of rapidly developing olorofim as a novel treatment for patients suffering from serious and life-threatening fungal infections, we are delighted to have been granted Orphan Drug Designation for the treatment of two specific fungal infections. Alongside the previously granted Breakthrough Therapy designation, this represents an important milestone and we look forward to continuing to work with the FDA to accelerate the development of this potentially life-saving therapy.”