Analysis of Pivotal Study Data Showed Routine Prevention of Hereditary Angioedema (HAE) Attacks
Analysis of Pivotal Study Data Showed Routine Prevention of Hereditary Angioedema (HAE) Attacks with Cinryze® (C1 esterase inhibitor [human]) Resulted in Improved Health-Related Quality of Life Outcomes Compared to Acute Therapy with C1 Inhibitor While On Placebo
- Data presented at the 2013 American College of Allergy, Asthma and Immunology (ACAAI) Annual Scientific Meeting -
EXTON, Pa., Nov. 12, 2013 /PRNewswire/ -- ViroPharma Incorporated (Nasdaq: VPHM), an international biopharmaceutical company committed to developing and commercializing innovative products that address unmet medical needs and rare diseases, today announced results of a new quality of life data analysis from the randomized, placebo-controlled pivotal prophylaxis study of Cinryze® (C1 esterase inhibitor [human]), the first and only C1 esterase inhibitor therapy approved for routine prevention of angioedema attacks in patients with HAE. The release of these data marks the first prospective evaluation of the impact of routine preventative treatment on the quality of life of patients with HAE. Quality of life data were specified in the protocol to be collected at the pre-treatment baseline and at the end of each of the two treatment periods but is not included in the prescribing information for Cinryze.
These data were presented in a poster entitled, Quality of Life in Patients with Hereditary Angioedema Receiving Nanofiltered C1 Inhibitor for Prophylaxis: Results of a Randomized, Placebo-Controlled, Crossover Study, by William Lumry, MD, FAAAI, FACAAI, et al.
While the burden of HAE and the impact on patients' health-related quality of life (HRQoL) has been previously described, the effect of routine prophylaxis on quality of life has not been evaluated. The objective of this analysis was to evaluate the HRQoL of patients with HAE while they were receiving Cinryze either as routine prophylaxis or for the acute treatment of individual attacks in the absence of prophylaxis during a randomized, placebo-controlled, crossover study. HRQoL was measured by the Short Form-36 V 1.0 (SF-36) questionnaire, the standard patient-reported survey for patient health and quality of life. Cinryze is not approved by the FDA to treat HAE attacks.
"These data demonstrate that patients who received prophylaxis with C1 inhibitor had significantly better quality of life compared to acute therapy with C1 inhibitor while on placebo," commented Dr. William Lumry, lead author and Medical Director of Asthma and Allergy Research Associates in Dallas, TX. "The analysis underscores the importance of educating patients on the potential benefits of prevention, and that prevention should be considered by physicians among the therapeutic options for patients with hereditary angioedema."
In the randomized, placebo-controlled trial, patients received intravenous injections of 1,000 U Cinryze or placebo every 3 to 4 days for 12 weeks and then crossed over to the other treatment for a second 12-week period. Patients could receive open-label Cinryze for acute treatment of breakthrough angioedema attacks during the Cinryze period, or for acute attacks in the placebo period. Sixteen of the 22 patients who were evaluated for efficacy completed SF-36 questionnaires for both treatment periods.
Results of the study included the following:
The baseline mean physical component summary score (36.4) was well below the mean for the US general population's 'normal' HRQoL score (50), highlighting the physical toll of experiencing frequent and unpredictable angioedema attacks;
Mean SF-36 scores at the end of the placebo period (plus acute therapy with C1 inhibitor when angioedema attacks occurred) were generally lower than or equal to baseline, indicating worsening of, or no improvement in, HRQoL;
Mean SF-36 scores at the end of the Cinryze prophylaxis period were generally greater than placebo (plus acute therapy with C1 inhibitor) and baseline, indicating improvement in HRQoL over both;
Least-square mean differences between Cinryze prophylaxis and placebo (plus acute therapy with C1 inhibitor) in norm-based SF-36 scores were 6.55 (p = .015) for the physical component summary score and 8.70 (p = .019) for the mental component summary score;
Statistically significant differences (p < .05) between Cinryze prophylaxis and placebo (plus acute therapy with C1 inhibitor) were also observed for all individual SF-36 domains (physical function, role physical, bodily pain, social function, mental health, role emotional, vitality, and general health); and
The magnitude of the difference between treatment groups was similar to or greater than those observed in other chronic diseases that are characterized by intermittent attacks/episodes, such as epilepsy, asthma, and migraine, when these are successfully managed.
About Cinryze® (C1 esterase inhibitor [human])
Cinryze is a highly purified, pasteurized and nanofiltered plasma-derived C1 esterase inhibitor product. In the U.S. and Canada, Cinryze is approved for routine prophylaxis (prevention) against angioedema attacks in adolescent and adult patients with HAE. In the EU, the product is approved for the treatment and pre-procedure prevention of angioedema attacks in adults and adolescents with hereditary angioedema (HAE), and routine prevention of angioedema attacks in adults and adolescents with severe and recurrent attacks of hereditary angioedema (HAE), who are intolerant to or insufficiently protected by oral prevention treatments or patients who are inadequately managed with repeated acute treatment. Cinryze is for intravenous use only.
Severe hypersensitivity reactions to Cinryze may occur. Thrombotic events have occurred in patients receiving Cinryze, and in patients receiving off-label high dose C1 inhibitor therapy. Monitor patients with known risk factors for thrombotic events. 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 donors for prior exposure to certain virus infections and by manufacturing steps to reduce the risk of viral transmission including pasteurization and nanofiltration.
The most common adverse reactions in clinical trials associated with Cinryze were rash, headache, nausea, erythema, phlebitis and local reactions at the injection site. Adverse events of sinusitis and upper respiratory infection also were observed in clinical trials. No drug-related serious adverse events (SAEs) were reported in clinical trials.
About Hereditary Angioedema (HAE)
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 at least 10,000 people in the European Union.
For more information on HAE, visit the U.S. HAE Association's website at www.haea.org and the HAEi's (International Patient Organization for C1 Inhibitor Deficiencies) website at www.haei.org.
About ViroPharma Incorporated
ViroPharma Incorporated is an international biopharmaceutical company committed to developing and commercializing novel solutions for physician specialists to address unmet medical needs of patients living with diseases that have few if any clinical therapeutic options. ViroPharma is developing a portfolio of therapeutics for rare and Orphan diseases including C1 esterase inhibitor deficiency, cytomegalovirus (CMV), Friedreich's Ataxia, eosinophilic esophagitis (EoE) and adrenal insufficiency. Our goal is to provide rewarding careers to employees, to create new standards of care in the way serious diseases are treated, and to build international partnerships with the patients, advocates, and health care professionals we serve. ViroPharma's commercial products address diseases including hereditary angioedema (HAE), seizures in children and adolescents, adrenal insufficiency and C. difficile-associated diarrhea (CDAD). For full U.S. prescribing information on our products, please download the package inserts at http://www.viropharma.com/Products.aspx; the prescribing information for other countries can be found at www.viropharma.com.
ViroPharma routinely posts information, including press releases, which may be important to investors in the investor relations and media sections of our company's web site, www.viropharma.com. The company encourages investors to consult these sections for more information on ViroPharma and our business.
Forward Looking Statements
Certain statements in this press release contain forward-looking statements that involve a number of risks and uncertainties. Forward-looking statements in this press release include statements regarding the quality of life in patients taking Cinryze. There can be no assurance that the data presented during the 70th American College of Allergy, Asthma and Immunology (ACAAI) Annual Scientific Meeting regarding Cinryze is predictive of how Cinryze will perform in commercial usage. In addition, the data that were discussed in this abstract and presentation are subject to different interpretations. The commercialization of pharmaceutical products is subject to risks and uncertainties. Our actual results could differ materially from those results expressed in, or implied by, these forward-looking statements. These factors, and other factors, including, but not limited to those described in our annual report on Form 10-K for the year ended December 31, 2012 and 10-Q for the quarter ended March 31, 2013, June 30, 2013, and September 30, 2013 filed with the Securities and Exchange Commission, could cause future results to differ materially from the expectations expressed in this press release. The forward-looking statements contained in this press release are made as of the date hereof and may become outdated over time. ViroPharma does not assume any responsibility for updating any forward-looking statements. These forward looking statements should not be relied upon as representing our assessments as of any date subsequent to the date of this press release.