ViroPharma Incorporated announced that the European Medicines Agency (EMA) has accepted the filing of its Marketing Authorization Application (MAA) for Cinryze for acute treatment and prophylaxis against hereditary angioedema (HAE), which was submitted through the centralized procedure in March 2010. ViroPharma is seeking European approval for both prevention and treatment of acute attacks of hereditary angioedema (HAE).

Cinryze was approved by the US Food and Drug Administration in October 2008 for routine prophylaxis against angioedema attacks in adolescent and adult patients with HAE. It is currently not approved for use in acute attacks of HAE in the US.

"Hereditary angioedema is a genetic inflammatory disease suffered by patients across the globe, and the acceptance of this application by the EMA is an important step in the process towards bringing this important therapy to HAE patients in Europe suffering from this debilitating disease," commented Vincent Milano, ViroPharma's president and chief executive officer. "We are looking forward to the day when we may be in position to provide this therapeutic alternative to this additional group of HAE patients."

The MAA is based on comprehensive clinical data including analyses of the results of ViroPharma's phase 3 studies of acute and prophylactic use of Cinryze in patients with HAE, plus recent data from the company's longer-term open label studies of the drug. The MAA acceptance triggers the initiation of EMA's regulatory review process.

In 2009 Cinryze received orphan medicinal product designation in the European Union. In the EU, orphan medicinal product designation is conferred upon investigational products for diseases that affect fewer than five in 10,000 patients. Products with orphan designation that are the first to be approved for a specific indication receive ten years of market exclusivity in the European Union plus two additional years with approval of a Paediatric Investigation Plan. In March of 2010, ViroPharma announced the adoption of a positive outcome to the compliance check for its Paediatric Investigation Plan for Cinryze.

Cinryze is a highly purified, pasteurized and nanofiltered plasma-derived C1 esterase inhibitor product that has been approved by U.S. FDA for routine prophylaxis against angioedema attacks in adolescent and adult patients with HAE. C1 inhibitor therapy has been used acutely for more than 35 years in Europe to treat patients with C1 inhibitor deficiency. Cinryze is not currently approved in the European Union or any of its member states.

The most common adverse reactions observed in clinical trials have been upper respiratory infection, sinusitis, rash and headache. No drug-related serious adverse events (SAEs) have been observed in clinical trials. Severe hypersensitivity reactions may occur. Thrombotic events have occurred in patients receiving high dose off-label C1 inhibitor therapy well above the approved treatment dosage regimen. With any blood or plasma derived product, there may be a risk of transmission of infectious agents, e.g. viruses and, theoretically, the CJD agent. The risk has been reduced by screening patients for prior exposure to certain virus infections and by manufacturing steps to reduce the risk of viral transmission including pasteurization and nanofiltration.

Cinryze is for intravenous use only. A dose of 1000 Units of Cinryze can be administered every three or four days for routine prophylaxis against angioedema attacks in HAE patients. Cinryze is administered at an injection rate of 1 mL per minute.

HAE is a rare, severely debilitating, life-threatening genetic disorder caused by a deficiency of C1 inhibitor, a human plasma protein. This condition is the result of a defect in the gene controlling the synthesis of C1 inhibitor. C1 inhibitor maintains the natural regulation of the contact, complement, and fibrinolytic systems, that when left unregulated, can initiate or perpetuate an attack by consuming the already low levels of endogenous C1 inhibitor in HAE patients. Patients with C1 inhibitor deficiency experience recurrent, unpredictable, debilitating, and potentially life threatening attacks of inflammation affecting the larynx, abdomen, face, extremities and urogenital tract. Patients with HAE experience approximately 20 to 100 days of incapacitation per year. There are estimated to be at least 6,500 people with HAE in the United States and approximately similar demographics in the European Union.

ViroPharma Incorporated is an international biopharmaceutical company committed to developing and commercializing innovative products for physician specialists to enable the support of patients with serious diseases for which there is an unmet medical need, and providing rewarding careers to employees.