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.
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