Dutch Biotech Reports Positive Phase II Data on Rare Inflammatory Disease

Pharming has published Phase II data in The Lancet supporting its lead candidate as a treatment for the rare genetic disease hereditary angioedema. 

Pharming, one of the top biotechs in Leiden, specializes in recombinant protein technology. Its lead compound, ruconest, is a recombinant human C1 inhibitor protein. The results of a 32-patient Phase II study published today in The Lancet proved the drug candidate effective in reducing the frequency of skin-swelling edema attacks that characterize the rare condition of hereditary angioedema (HAE).

HAE is a genetic disorder caused by the deficiency of the C1 esterase inhibitor protein, which is involved in controlling inflammation processes induced by the immune system. Patients suffering from the disease, which affects around one in every 30,000 people worldwide, experience frequent swellings that cause disfiguration and pain, and can be deadly when affecting airway passages. By addressing the cause of the disease, Pharming aims to provide the first treatment to ever address such a debilitating disease.


Image via Syda Productions

Support good journalism, subscribe to Labiotech Insider

Labiotech Insider

If you liked this post and want to read more good journalism, support us by joining Labiotech Insider. Our Insider membership gives you access to exclusive content and other advantages.

We use cookies to give you the best experience. If you click on accept, we also use tracking cookies for analytics and advertising purposes. Check our privacy policy for more details. By accepting, you support our independent media and its’ freely accessible content. You may withdraw your consent at any time.