Endeavor BioMedicines receives Orphan Drug Designation for taladegib in the US and EU for idiopathic pulmonary fibrosis
Thursday, July 17, 2025
Endeavor BioMedicines, a clinical-stage biotechnology company focused on developing treatments for serious diseases, has received Orphan Drug Designation for its investigational therapy, taladegib (ENV-101), from both the U.S. Food and Drug Administration (FDA) and the European Commission (EC). The designation applies to the treatment of idiopathic pulmonary fibrosis (IPF), a chronic and progressive lung disease with limited therapeutic options.
The company is currently conducting a global Phase 2b clinical trial, known as WHISTLE-PF, to assess the effectiveness and safety of taladegib in individuals with IPF. This trial is evaluating different doses over a 24-week period, focusing on changes in lung function, lung capacity, fibrosis levels (measured by chest high-resolution CT scans), and patient-reported outcomes. Trial enrolment is ongoing and expected to be completed by 2026.
Orphan Drug Designation in the European Union is granted to therapies intended for rare, life-threatening conditions that affect fewer than five in 10,000 people. The designation provides incentives such as protocol assistance, reduced regulatory fees, and ten years of market exclusivity if approved.
In the United States, the FDA grants Orphan Drug Designation to therapies targeting rare diseases that affect fewer than 200,000 people. Benefits of this designation include financial support for development, tax credits, and up to seven years of market exclusivity if the drug receives approval for the designated use.
Taladegib is being developed as a potential treatment to slow or reverse disease progression in IPF by targeting disease-related signalling pathways, offering a possible advancement beyond current standard-of-care therapies.
Source: businesswire.com
