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Pharming Announces Topline Study Results On Prophylactic Use Of Ruconest In Hereditary Angioedema
Leiden, The Netherlands, November 29, 2010. Pharming Group NV (“Pharming” or “the Company”) (NYSE Euronext: PHARM) today announced the results of its exploratory Phase II study on the safety and prophylactic effect of recombinant human C1 inhibitor (“rhC1INH”) in patients with Hereditary Angioedema (HAE).
The study is an open-label exploratory phase II study of the safety and prophylactic effect of a weekly 50 U/kg rhC1INH treatment in asymptomatic patients with hereditary C1INH deficiency (the “OPERA study”). In the study, 25 asymptomatic HAE patients with a history of frequent attacks received once weekly administrations of 50 U/kg of Ruconest for 8 weeks. Patients reported a median of 60 HAE attacks (range 39 to 467) over the past two years, corresponding to an averag e of 0.6 attacks per week (range 0.4 to 4.5). The breakthrough attack rate observed during the study was much lower with a median of 2 attacks over the 8 week period, corresponding to an average of 0.25 attacks per week (range 0 to 1.5). Weekly administrations of 50 U/kg rhC1INH was generally safe and well tolerated.
Dr. Rienk Pijpstra, Chief Medical Officer of Pharming: “The results of this exploratory study suggest that Ruconest, in addition to the approved indication in Europe for the acute treatment of angioedema attacks, could also offer significant value for long term prophylaxis in patients with frequent attacks. We plan further pharmacological investigations to optimize Ruconest dose and dosing interval for this indication.”
In October, the European Commission granted Pharming Marketing Authorization for its lead product Ruconest for the treatment of acute attacks of Hereditary Angioedema. Pharming’s marketing and distribution partner Swedish Orphan Biovitrum is preparing this year’s launch of Ruconest initially in Germany, the UK, Sweden, Finland and Denmark, followed by a rolling launch in each of the other European Area countries covered by the agreement.
About HAE and Ruconest™ (Rhucin® in non-European countries)
HAE is a human genetic disorder in which the patient is deficient in or lacks a functional plasma protein C1 inhibitor, resulting in an overreaction of the immune system. The disease is characterized by unpredictable and debilitating episodes of intense swelling of the extremities, face, trunk, genitals, abdomen and upper airway, which may last up to five days when untreated. In addition to the life-threatening nature of the disease in case of laryngeal attacks, quality of life for individuals with the disease may be seriously impaired. Approximately one in 30,000 individuals (1:10,000 ? 1:50,000) suffers from HAE with an average of ap proximately eight acute attacks per year.
Ruconest is a recombinant version of the human C1 inhibitor (C1INH) protein approved for the treatment of angioedema attacks in patients with HAE in all 27 EU countries plus Norway, Iceland and Liechtenstein. The product is produced through Pharming’s proprietary technology in milk of transgenic rabbits.
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