AIM-listed drug developer wins US designation for inhaled fibrosis therapy, unlocking tax credits and potential seven-year market exclusivity
Shares in Nuformix PLC (LSE:NFX) jumped 12.5% to 0.29p after the US Food and Drug Administration (FDA) granted orphan drug designation (ODD) for its NXP002 programme targeting idiopathic pulmonary fibrosis (IPF), a rare and often fatal scarring of the lungs.
The designation covers tranilast lystate, the active compound in NXP002, which has been reformulated by Nuformix for inhaled delivery.
Orphan drug designation is granted by the FDA for treatments targeting rare diseases affecting fewer than 200,000 people in the United States, with IPF falling within that threshold.
The status unlocks a package of financial and regulatory incentives, including tax credits on clinical trial costs, a waiver of the Prescription Drug User Fee Act application fee when a marketing application is submitted, and the potential for seven years of marketing exclusivity if the product reaches approval.
The US designation follows orphan drug status granted by the European Medicines Agency (EMA) in April 2025, giving Nuformix recognised rare disease status on both sides of the Atlantic.
Dan Gooding, executive director of Nuformix, said the dual designations would strengthen the company's position in licensing discussions currently underway with potential partners.