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”, 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.”
About Olorofim / Clinical trial
The Phase 2b study for olorofim (ClinicalTrials.gov Identifier: NCT03583164) is a global open-label study in patients who have limited treatment options for difficult-to-treat invasive fungal mould infections such as azole-resistant aspergillosis, scedosporiosis, lomentosporiosis, and other rare mould infections. 34 centres are currently open in six countries (AU, BE, ES, NL, USA, IS) and a further 12 will open in 2020. Olorofim is being developed both as IV and oral formulations.
F2G is a world-leading UK- and Austria-based biotech company (F2G Ltd and F2G Biotech GmbH) focused on the discovery and development of novel therapies to treat life-threatening invasive fungal infections. F2G has discovered and developed a completely new class of antifungal agents called the orotomides. The orotomides target dihydroorotate dehydrogenase (DHODH), a key enzyme in the de novo pyrimidine biosynthesis pathway. This is a completely different mechanism from that of the currently marketed antifungal agents and gives the orotomides fungicidal activity against a broad range of rare and resistant fungal mould infections. Olorofim (formerly, F901318) is F2G’s leading candidate from this class and is in a Phase 2b open-label study focussing on rare and resistant invasive fungal infections such as aspergillosis (including azole-resistant strains), scedosporiosis, and lomentosporiosis. Olorofim has received orphan drug status from the European Medicines Agency for the treatment of invasive aspergillosis and invasive scedosporiosis. Olorofim is being developed both as IV and oral formulations. www.f2g.com