Date: 2012-06-18
Type of information:
phase: 3
Announcement: presentation of clinical safety and efficacy data for Ruconest at the annual meeting of the European Academy of Allergy and Clinical Immunology (EAACI), Geneva, Switzerland, 16 to 20 June 2012.
Company: Pharming (The Netherlands)
Product: Ruconest® (Rhucin® in non european countries - conestat alfa)
Action mechanism: Ruconest® 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.
Disease: acute angioedema attacks in patients with hereditary angioedema (HAE)
Therapeutic area:
Country:
Trial
details: Pharming is conducting a Phase III clinical study with Ruconest® under a Special Protocol Assessment (SPA) that is intended to support the submission of a Biologics License Application (BLA) to the FDA. The drug is being evaluated for the treatment of acute attacks of angioedema in patients with HAE in an international, multicenter, randomized, placebo-controlled Phase III study at a dosage of 50 U/kg with a primary endpoint of time to beginning of relief of symptoms. The study is expected to be completed in Q3 2012.
Latest
news: The new data covers a number of aspects that are relevant to the increasing number of physicians that use Ruconest® in the day-to-day treatment of HAE patients in the European Union: Efficacy and safety data for Ruconest® in the treatment of adolescents suffering from acute attacks of HAE underpinning a potential extension of the European labeling of Ruconest® and “real- life” experience by French physicians; building confidence in Ruconest® by successf ul treatment with Ruconest of a HAE patient that previously failed various other treatments. In addition an analysis of a previously reported open- label study to explore potential benefits of Ruconest® in prophylaxis of HAE, potentially an additional indication that could be explored. The headlines and authors of the poster presentations are: Toubi et al: “Safety and Efficacy evaluation of rhC1INH for the treatment of HAE attacks in adolescent patients”. This analysis reviewed data from 16 adolescent HAE patients who were treated for a total of 50 angioedema attacks with Ruconest®. Patients were treated up to 7 times for HAE attacks at all locations. Median times to onset of symptom relief for successive attacks ranged from 19 to 123 minutes. Median times to minimal symptoms ranged from 120 to 650 minutes. Ninety percent of the attacks responded within 4 hours after treatment, and none of the attacks relapsed. The most frequentl y reported adverse event was headache. No hypersensitivity reactions and no drug-related serious adverse events were observed. No treatment-emergent antibodies developed in these patients. Bouillet et al: The case of a type III HAE patient who failed several therapies and was successfully treated with Ruconest. Reshef et al: “Safety and Efficacy of a weekly infusion of Recombinant Human C1 Inhibitor (rhC1INH) for Prophylaxis of Hereditary Angioedema (HAE) attacks”. This was an open label study on the prophylactic effect of once-weekly administration of Ruconest® in 25 HAE patients. Patients included in this study had a history of frequent HAE attacks, with a significant impact on their quality of life. The frequency of HAE attacks during the 8 week treatment period was reduced by approximately 50 percent, from a median of 0.6 attacks per week to 0.3. The repeated administrations were generally safe and well-tolerated.
