Date: 2012-10-31
Type of information:
phase:
Announcement: publication of Ruconest® HAE prophylaxis study in Allergy
Company: Pharming (The Netherlands)
Product: Ruconest® (Rhucin® in non-European territories - conestat alfa)
Action
mechanism: Ruconest® (conestat alfa) is a recombinant version of the human protein C1 inhibitor (C1INH). This protein naturally occurs in the human body. It belongs to the class of serine-protease inhibitors or serpins. It regulates several inflammatory pathways in the body by inhibiting certain proteins (proteases) that are part of the human defense system. Deficiency of functional C1 inhibitor leads to excessive activation of the complement system and other immunological and haemostatic pathways, giving cause to angioedema attacks. These attacks are characterized by acute and painful swellings of soft tissues. Administration of C1 inhibitor protein can normalize the immune response and stop the angioedemic attacks.
Ruconest® is produced through Pharming’s proprietary technology in milk of transgenic rabbits and is approved in Europe for the treatment of acute angioedema attacks in patients with HAE. Ruconest® has been granted orphan drug designation both for the treatment of acute attacks of HAE and prophylactic treatment of HAE.
Disease: acute attacks of angioedema in patients with Hereditary Angioedema (HAE)
Therapeutic area: Genetic diseases - Rare diseases
Country:
Trial details:
Latest
news: Pharming Group has announced that the positive results of a clinical trial with recombinant human C1 inhibitor (rhC1INH; Ruconest®) to prevent attacks of hereditary angioedema (HAE) have been accepted for publication in the international peer-reviewed journal, Allergy.
This was an open-label study to evaluate the prophylactic effect of once-weekly administration of Ruconest® in 25 HAE patients. The patients included in this study had a history of frequent HAE attacks (mean 0.9 attacks/week). During the 8 week Ruconest® treatment period, the mean frequency of HAE attacks was reduced by more than 50% to 0.4 attacks/ week. The repeated administrations were generally safe and well-tolerated. The full study results will be published online in November.
