The European Commission has granted Orphan Drug Designation to efzofitimod for the treatment of systemic sclerosis (SSc), aTyr Pharma announced.

This decision is based on the opinion of the European Medicines Agency Committee for Orphan Medicinal Products.

“In addition to the clinical proof-of-concept data generated for efzofitimod in pulmonary sarcoidosis, the Committee for Orphan Medicinal Products considered that the non-clinical in vivo data for efzofitimod in SSc, which demonstrated a reduction in the decline of lung function and skin fibrosis not achieved with an authorized medicine in the European Union, constitutes a potentially clinically relevant advantage over existing therapies,” said Sanjay S. Shukla, MD, MS, president and CEO of aTyr.

The phase 1b/2a multiple-ascending dose, placebo-controlled study in patients with pulmonary sarcoidosis provided the clinical proof-of-concept for efzofitimod, demonstrating the safety and consistent efficacy of the experimental drug compared with placebo. The key efficacy endpoints, including steroid reduction, lung function, clinical symptoms, and inflammatory biomarkers, were all met.

Read more about experimental therapies for SSc

The biotherapeutics company is now conducting a global pivotal EFZO-FIT phase 3 clinical trial evaluating efzofitimod in patients with pulmonary sarcoidosis; the phase 2 proof-of-concept study in patients with SSc is planned to start in the third quarter of 2023.

In 2022, the experimental drug received Orphan Drug and Fast Track designations for SSc from the US Food and Drug Administration (FDA).

Efzofitimod is a selective modulator of neuropilin-2 that downregulates the immune response in uncontrolled inflammatory diseases. Although the main indication for efzofitimod is pulmonary sarcoidosis, patients with SSc, who have limited treatment options associated with high toxicity and simply focus on slowing down disease progression, may also benefit from it.


European Commission grants orphan drug designation for aTyr Pharma’s efzofitimod for treatment of systemic sclerosis. News release. aTyr Pharma, Inc.; June 22, 2023.