At the end of June 2022, biopharmaceutical company InflaRx N.V. (“InflaRx”) shared via press release that its product vilobelimab recently earned Orphan Drug designation in both the United States and the European Union. Currently, vilobelimab is being developed as a therapeutic for both severe COVID-19 infection and pyoderma gangrenosum (PG). However, the Orphan Drug designation was granted solely for pyoderma gangrenosum.
About Orphan Drug Designation
In the United States, Orphan Drug designation is granted to drugs or biologics intended to treat, prevent, or diagnose rare conditions. Rare conditions are those affecting fewer than 200,000 Americans. As an incentive, drug developers – in this case, InflaRx – receive benefits such as fee waivers, tax credits, increased regulatory assistance, and seven years of market exclusivity upon approval.
Alternately, in Europe, the EMA grants Orphan Drug designation to therapies intending to treat, diagnose, or prevent rare, debilitating, or life-threatening illnesses. To be eligible for this designation, the illness or condition must not affect more than 5 in every 10,000 citizens. Incentives include protocol assistance and ten years of market exclusivity upon approval.
What is Vilobelimab?
Developed by InflaRx, vilobelimab is a monoclonal anti-C5a antibody. Since C5a is part of the complement system, it can contribute to severe and sometimes harmful inflammation within the body. By blocking C5a, vilobelimab works to reduce tissue and organ inflammation, as well as related damage. So far, clinical studies have found vilobelimab to be relatively safe and well-tolerated. It has also been relatively effective in treating pyoderma gangrenosum, according to clinical trial results. In the future, InflaRx hopes to complete another Phase 3 clinical study on pyoderma gangrenosum, gain emergency use authorization for vilobelimab for ventilated individuals with COVID-19, and develop the treatment further for cutaneous squamous cell carcinoma (cSCC).
About Pyoderma Gangrenosum (PG)
Pyoderma gangrenosum is a rare ulcerating inflammatory skin condition. PG is considered to be idiopathic, or without a known cause. However, some researchers believe that PG can be considered an autoimmune disorder. According to the National Organization for Rare Disorders (NORD), PG:
often begins as small, quick-spreading reddish or purple-colored bumps or blisters. These small growths eventually develop into swollen, open sores with a well-defined blue or violet-colored border.
PG is slightly more common in females than in males. It can affect people of all ages, but most commonly occurs between ages 20-50. PG has been associated with other conditions such as inflammatory bowel disease, rheumatoid arthritis, and granulomatosis with polyangiitis (among others). Sores often affect the legs.
