Nuformix plc (LON:NFX), a pharmaceutical development company targeting unmet medical needs in fibrosis and oncology via drug repurposing, has announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation (ODD) in Idiopathic Pulmonary Fibrosis (IPF) for tranilast lystate, the active drug substance enabled for inhaled delivery in Nuformix’s NXP002 lead programme.
The FDA grants ODD for investigational treatments for rare diseases, such as IPF, defined as affecting fewer than 200,000 people in the United States. ODD qualifies the developer for certain incentives with the goal of accelerating drug development for patients, including tax credits for clinical trials or qualified clinical testing costs, a waiver of the Prescription Drug User Fee Act application fee when a marketing application is submitted, and the potential to receive seven years of marketing exclusivity upon product approval.
Dr Dan Gooding, Executive Director, Nuformix, said: “We are delighted to receive confirmation that Orphan Drug Designation has been granted by the FDA for our NXP002 programme in IPF, a high-mortality rare disease, in urgent need of new treatments. The granting of ODD in the United States, alongside the European EMA ODD granted in April 2025, will be valuable for the potential future licensing partners we are in discussions with and I look forward to providing further updates in due course.”
