Biotech Values

[DewDiligence]

VPHM – FDA Requires New Study for Cinryze in Acute HAE

[Cinryze was approved by the FDA for prophylactic use in Oct 2008 (#msg-32795311).]

http://phx.corporate-ir.net/phoenix.zhtml?c=92320&p=irol-newsArticle&ID=1296156

›ViroPharma Receives Complete Response Letter for Cinryze Supplemental Biologics License Application for Acute Treatment of Hereditary Angioedema

- Company Also Granted Expansion of Prophylaxis Therapy Label to Include Patient Labeling for Self-Administration -

EXTON, Pa., June 4 /PRNewswire-FirstCall/ -- ViroPharma Incorporated (Nasdaq: VPHM) today announced that it has received a Complete Response letter from the U.S. Food and Drug Administration (FDA) related to its supplemental Biologics License Application (sBLA) for Cinryze (C1 esterase inhibitor [human]) as a treatment for acute attacks of hereditary angioedema (HAE). The FDA has requested an additional clinical study, due to their opinion that the placebo controlled study submitted in support of the sBLA lacked robustness. In the Complete Response letter, the FDA cited no safety concerns related to acute treatment with Cinryze in the clinical studies.

In addition, ViroPharma announced that FDA has approved the patient labeling for Cinryze to include self-administration for routine prophylaxis, once patients are properly trained by their healthcare provider.

"Though Cinryze is already successfully preventing attacks of HAE in many patients, there remains an unmet medical need for patients suffering from acute laryngeal attacks," said Vincent Milano, ViroPharma's president and chief executive officer. "Despite having a statistically significant result using the most conservative intent-to-treat (ITT) analysis, the FDA feels that the data are not robust enough to support approval at this time. We intend to respond to the FDA about our plans, and we will provide an update on the second quarter financial call."

In October 2008, Cinryze was approved as the first and only targeted therapy for routine prophylaxis against angioedema attacks. In clinical studies and in commercial use, Cinryze has been generally well-tolerated. In the controlled clinical trials of Cinryze, there were no deaths or serious adverse reactions related to Cinryze administration, or discontinuations due to treatment-emergent adverse events.

"Patients in the United States with HAE have been waiting for a safe and effective emergency treatment for a long time," said Anthony Castaldo, president of the U.S. and International Hereditary Angioedema Association. "Cinryze as a prophylactic treatment has changed the lives of many people living with this debilitating disease; we look forward to a day when this important therapy will be approved in the acute setting."

Patient Labeling for Self Administration

Self-administration of Cinryze provides patients with the ability to administer Cinryze themselves, when properly trained, in the comfort of their own home. This is an important option for some patients because they may no longer need to travel to their doctor's office - sometimes hours away - every three to four days for in-office infusions. Cinryze is administered intravenously though a small needle.

"We are pleased that the FDA has recognized the importance of self-administration for patients," continued Milano. "Empowering patients to administer Cinryze themselves allows them greater flexibility in managing their disease."‹