In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, diagnose, or prevent rare diseases or conditions. For the purpose of this status, “rare” is considered any condition affecting under 200,000 Americans. According to Healio, the U.S. Food and Drug Administration (FDA) recently granted this status to efzofitimod, developed as a potential treatment option for patients with systemic sclerosis. 

Drug developer aTyr Pharma describes efzofitimod as: 

a potential first-in-class, disease modifying therapy for patients with severe inflammatory lung diseases with high unmet medical need. Efzofitimod works by selectively modulating Neuropilin-2 (NRP2) to downregulate the innate and adaptive immune responses in uncontrolled inflammatory disease states to resolve inflammation and prevent subsequent fibrosis.

Outside of systemic sclerosis, aTyr Pharma is also evaluating efzofitimod for conditions such as pulmonary sarcoidosis, COVID-19, interstitial lung disease (ILD), and idiopathic pulmonary fibrosis (IPF). For example, in studies of IPF, efzofitimod showed anti-fibrotic properties – meaning it helps reduce scarring (fibrosis) associated with the condition. 

Because efzofitimod received Orphan Drug designation, aTyr Pharma also receives a variety of benefits. These include fee waivers, tax credits, increased regulatory assistance, and seven years of market exclusivity upon drug approval.

About Systemic Sclerosis

Also known as systemic scleroderma, systemic sclerosis is an autoimmune disorder which causes skin and organ fibrosis (scarring and skin thickening). An autoimmune disorder means that the body’s immune system mistakenly attacks parts of the body – in this case, skin and other organs. While sclerosis can be localized (affecting only a specific area or part of the body), systemic sclerosis means that the condition affects multiple parts of the body. The condition causes the creation of excess collagen, which leads to a buildup of scar tissue and thickened skin. Systemic sclerosis is 4x more common in females than males. Symptoms include:

• Open sores on the fingers OR painful bumps or deposits under the skin
• Raynaud’s phenomenon
• Hardened or tightened skin
• Joint pain
• Heartburn
• Difficulty swallowing
• Unintended weight loss
• Swollen fingers and hands
• High blood pressure
• Gastrointestinal distress
• Shortness of breath
• Impaired esophageal, lung, kidney, or heart function