EC grants aTyr Pharma ODA to efzofitimod to treat sarcoidosis

aTyr Pharma announced that the European Commission granted orphan drug designation for the company’s lead therapeutic candidate, efzofitimod, for the treatment of sarcoidosis based on the opinion of the European Medicines Agency Committee for Orphan Medicinal Products . Efzofitimod is a first-in-class immunomodulator that downregulates innate and adaptive immune responses in uncontrolled inflammatory disease states via selective modulation of neuropilin-2 and is currently being investigated in a global pivotal Phase 3 study in patients with pulmonary sarcoidosis, the main form of the disease. "The EC’s decision to grant orphan drug designation to efzofitimod acknowledges the unmet medical need in sarcoidosis for the approximately 150,000 people in the European Union (EU) living with this chronic, debilitating disease," said Sanjay S. Shukla, M.D., M.S., President and CEO of aTyr. "The criteria for orphan status in the EU go beyond the rarity of the disease. This designation takes into account the preclinical and clinical proof-of-concept data generated for efzofitimod, including data from a Phase 1b/2a study showing efzofitimod’s ability to improve lung function and quality of life measures in the context of a steroid taper, and recognizes the potential benefit of this novel immunomodulator to be a transformative, disease modifying therapy with clinically meaningful outcomes for patients."