aTyr Pharma announcse EC granted ODD for efzofitimod

aTyr Pharma announced that the European Commission, EC, granted orphan drug designation, ODD, for the company’s lead therapeutic candidate, efzofitimod, for the treatment of systemic sclerosis based on the opinion of the European Medicines Agency, EMA, Committee for Orphan Medicinal Products, COMP. Efzofitimod is currently being investigated in a global pivotal Phase 3 clinical trial in patients with pulmonary sarcoidosis, a major form of interstitial lung disease, and a Phase 2 proof-of-concept study in patients with SSc-ILD is expected to begin in the third quarter of 2023. “We are pleased that the EC recognizes the need for new and impactful treatments for the nearly 100,000 people living with systemic sclerosis in the European Union,” said Sanjay S. Shukla, M.D., M.S., President and CEO of aTyr. “This orphan drug designation takes into account more than just the rarity of the disease. In addition to the clinical proof-of-concept data generated for efzofitimod in pulmonary sarcoidosis, the COMP considered that the non-clinical in vivo data for efzofitimod in systemic sclerosis, which demonstrated a reduction in the decline of lung function and skin fibrosis not achieved with an authorized medicine in the EU, constitutes a potentially clinically relevant advantage over existing therapies. We believe efzofitimod presents a much-needed opportunity to meaningfully advance treatment options for those whose lungs are affected by systemic sclerosis, and we look forward to the expected initiation of our Phase 2 clinical trial of efzofitimod in patients with SSc-ILD in the third quarter of this year.”