F2G announced 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 mold 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 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.
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.
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"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," Ian Nicholson, CEO of F2G, said.
The Phase 2b study for olorofim 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.
Olorofim has received orphan drug status from the European Medicines Agency for the treatment of invasive aspergillosis and invasive scedosporiosis.