Nuformix plc (LON:NFX), a pharmaceutical development company targeting unmet medical needs in fibrosis and oncology via drug repurposing, has announced, further to the Company’s announcement on 30 April 2025, that the European Medicines Agency (EMA) has granted Orphan Drug Designation (ODD) in Idiopathic Pulmonary Fibrosis (IPF) for tranilast, the active drug substance enabled for inhaled delivery in Nuformix’s NXP002 lead programme.
ODD in the European Union (EU) is granted by the European Commission based on a positive opinion adopted by the EMA Committee for Orphan Medicinal Products that can demonstrate potential for significant advancement in treatment of rare and debilitating diseases affecting no more than five in 10,000 individuals in the EU. ODD provides incentives to developers of medicines for limited patient populations, including 10 years market exclusivity, protocol assistance (guidance on study design and scientific evaluation) and regulatory fee reductions.
Phil Molyneaux, MD, Professor of Pulmonary Medicine at the Royal Brompton Hospital, London, commented: “Patients with IPF currently lack treatment options that significantly alter the course of their disease. They also often face tolerability issues with existing therapies. Inhaled therapies like NXP002 offer the potential for improved efficacy by delivering the drug directly to the lungs, while avoiding the side effects associated with oral treatments. This approach could represent a meaningful step forward in addressing the urgent need for better treatment options for patients with IPF.”
Dr Dan Gooding, Executive Director, Nuformix, said: “We are delighted to receive confirmation that Orphan Drug Designation has been granted for our NXP002 programme in IPF. The EMA award recognises NXP002’s potential to significantly advance IPF treatment, thanks to its ability to modulate key targets in IPF progression, but also given its potential enhancement of current standard of care therapies. Orphan Drug Designation should significantly support our goal of progressing NXP002 through partnering and further development activities and, ultimately bringing solutions for fibrotic ILD indications to patients. We’re thrilled that like us, the EMA has recognised the promise of this potential breakthrough medicine. We will now submit an application for US FDA orphan drug designation and continue discussions with potential future licensing partners and will provide further updates in due course as appropriate.“
