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 29 May 2025, that the European Medicines Agency had granted Orphan Drug Designation in Idiopathic Pulmonary Fibrosis (IPF) for tranilast, the active drug substance enabled for inhaled delivery in Nuformix’s NXP002 lead programme, that the Company has now submitted an application to the US Food and Drug Administration for ODD in the United States using FDA Form 4035.
The components required for the submission of Form 4035 included:
• information about Nuformix and the drug product;
• description of the rare disease or condition of interest, along with reasons why such therapy is needed;
• scientific rationale for the use of the drug for the rare disease or condition;
• a summary of the regulatory status and marketing history of the drug in the United States and in other countries; and
• documentation to demonstrate that the disease or condition meets qualifications to be a rare disease, defined as a disease or condition affecting fewer than 200,000 individuals in the United States.
The FDA will now review the application and, within 90 days of receipt, issue a designation letter, a request for more information or a denial.
If ODD is granted, Nuformix may be eligible for certain benefits such as 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.
The Company is also pleased to confirm that constructive discussions continue with a number of potential partners with a view to the Company securing an out-licence or option agreement for NXP002.
Dr Dan Gooding, Executive Director, Nuformix, said: “We are very pleased to have submitted the FDA ODD application following confirmation that Orphan Drug Designation has been granted for our NXP002 programme in IPF in the EU by the EMA. Whilst we await the FDA’s response we continue discussions with potential future licensing partners and will provide further updates in due course as appropriate.“
