Date: 2015-05-18
Type of information: Collaboration agreement
Compound: Ruconest® (conestat alfa)
Company: Pharming (The Netherlands) Clinigen (UK)
Therapeutic area: Rare diseases - Genetic diseases - Hematological diseases
Type agreement: collaboration
Action mechanism: enzyme inhibitor/C1-esterase inhibitor. Ruconest® (conestat alfa) is a human recombinant C1-esterase inhibitor purified from the milk of transgenic rabbits. Hereditary angioedema, which is caused by having insufficient amounts of a C1-esterase inhibitor, affects approximately 6,000 to 10,000 people in the United States. People with HAE can develop rapid swelling of the hands, feet, limbs, face, intestinal tract, or airway. These acute attacks of swelling can occur spontaneously, or can be triggered by stress, surgery or infection. Swelling of the airway is potentially fatal without immediate treatment. Ruconest® is intended to restore the level of functional C1-esterase inhibitor in a patient’s plasma, thereby treating the acute attack of swelling. Ruconest® is the first and only plasma-free, recombinant C1-INH approval from the FDA and was approved in July 2014 and by the European Medicines Agency (EMA) in October 2010.
Disease: hereditary angioedema (HAE)
Details: * On May 18, 2015, Pharming and Clinigen have entered into an international global access collaboration for HAEi, the International Patient Organization for C1- Inhibitor Deficiencies. The “HAEi GAP” program will provide access to Ruconest® (conestat alfa) to eligible patients with hereditary angioedema (HAE), who currently do not have access to effective medication to treat acute attacks of the disease. HAEi GAP is the first global access program for HAE and the first to be driven by a patient organization. The program enables patients in countries where Ruconest® is not commercially available to gain access to the drug through an ethical and regulatory compliant “Named Patient Program” mechanism.
Financial terms:
Latest news: * On July 21, 2015, Pharming and Clinigen announced that their global collaboration to provide access to Pharming’s Ruconest® (conestat alfa) is now live. The unique access program was initiated by HAEi, the International Patient Organization for C1- Inhibitor Deficiencies.
