Efzofitimod, potential SSc treatment, named an orphan drug in EU

Phase 3 trial of efzofitimod is enrolling adults with pulmonary sarcoidosis

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

Share this article:

Share article via email
A doctor spotlighting the words

The European Commission (EC) has given orphan drug designation to efzofitimod as a potential treatment of systemic sclerosis, also known as SSc or scleroderma, aTyr Pharma, the therapy’s developer, announced in a press release.

The EC’s decision provides the company with certain incentives, such as fee reductions and a 10-year protection from competition should efzofitimod be approved. It followed an opinion by the Committee for Orphan Medicinal Products (COMP), an arm of the European Medicines Agency, favoring the designation.

To qualify as an orphan drug, a medication must be of significant benefit for a rare disease — defined as one affecting not more than 5 in 10,000 people in the European Union — or a treatment that, when marketed, would be unlikely to generate enough returns to justify the investment needed for its development.

Recommended Reading
A person speaks before a group at a conference.

High CCL24 levels in SSc associated with more severe disease

Estimated 100,000 people in Europe are living with systemic sclerosis

“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 (EU),” said Sanjay S. Shukla, MD, president and CEO of aTyr Pharma.

“This orphan drug designation takes into account more than just the rarity of the disease,” Shukla added.

The company announced plans to launch a Phase 2 trial in people with interstitial lung disease (ILD) associated with SSc this year. Efzofitimod holds orphan drug and fast track designations in the U.S.

“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,” Shukla said.

In SSc, the immune system mistakenly attacks the body’s tissues, causing them to become inflamed and scarred. When scarring (fibrosis) affects the lungs, it can cause their tissues to become thicker and stiffer.

Current disease treatments for SSc-ILD are limited, aiming mainly to slow progression, the company noted.

Efzofitimod is designed to bind to neuropilin-2 (NRP2), a protein present on the surface of immune cells. By binding to NRP2, efzofitimod is expected to dampen inflammation, helping to ease fibrosis.

Its lead indication is pulmonary sarcoidosis, a type of ILD that occurs when small clumps of inflammatory cells known as granulomas grow in the lungs. A placebo-controlled Phase 3 trial called EFZO-FIT (NCT05415137) is now testing efzofitimod in up to 264 adults with pulmonary sarcoidosis, and eligible patients are being enrolled at sites across the U.S., Europe and Japan.

A previous Phase 1/2 trial (NCT03824392), found efzofitimod to be safe and effective for pulmonary sarcoidosis, improving patients’ lung function and easing symptoms such as shortness of breath, cough, and fatigue.

“In addition to the clinical [trial] proof-of-concept data generated for efzofitimod in pulmonary sarcoidosis, the COMP considered that the non-clinical in vivo [in lab animals] 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,” Shukla said.