C1-esterase inhibitor (C1-INH)
concentrate is a safe and effective therapy that rapidly relieves acute
abdominal and facial swelling attacks in patients with hereditary
angioedema (HAE), a rare and serious genetic disorder, according to data
presented at the American Academy of Allergy, Asthma & Immunology
(AAAAI) 64th Annual Meeting. Results from the prospective, double-blind
International Multi-center Prospective Angioedema C1-Inhibitor Trial
(I.M.P.A.C.T. 1), the largest HAE study ever conducted, showed patients
receiving C1-INH concentrate had a median time to symptom relief of 30
minutes, versus 90 minutes for patients receiving placebo. Additionally,
interim results from I.M.P.A.C.T. 2, also presented at AAAAI, demonstrated
for the first time C1-INH replacement therapy's effectiveness in rapidly
relieving HAE attacks in peripheral body locations, such as the hands and
feet.
HAE is a genetic disorder caused by a deficiency of C1-INH, which is
inherited in an autosomal dominant manner. Symptoms include episodes of
edema or swelling in the hands and feet, the face, the abdomen, and/or the
larynx. Patients who have abdominal attacks can experience episodes of
severe pain, diarrhea, nausea, and vomiting caused by swelling of the
intestinal wall. Attacks that involve the face and larynx can result in
airway closure, asphyxiation, and, if untreated, death. Diagnosis of HAE
requires a blood test to confirm low or abnormal levels of C1-INH. It is
estimated that 6,000 to 10,000 Americans -- and perhaps more -- suffer from
HAE.
"The findings of I.M.P.A.C.T. 1 confirm that C1-INH concentrate can
bring rapid relief compared to placebo in HAE patients who experience
facial and abdominal attacks," said Jonathan A. Bernstein, MD, FAAAAI,
FACP, CCI, Bernstein Clinical Research Center, Cincinnati, OH, and a lead
investigator of I.M.P.A.C.T 1. "While HAE attacks can occur all over the
body, facial and abdominal swelling can be the most painful and
life-threatening for patients and require treatment with a fast-acting
therapy."
Currently, there are no specifically-approved therapies for HAE in
North America. CSL Behring manufactures and sells C1-INH concentrate in
Germany, Austria, Switzerland, and several other countries.
About I.M.P.A.C.T. 1
Findings were based on a double-blind, placebo-controlled study of 124
HAE patients with acute, moderate, or severe abdominal or facial attacks.
C1-INH concentrate was administered at two different doses and compared to
placebo. The main study endpoints were: time to onset of symptom relief
from HAE attacks, and proportion of subjects with worsening clinical HAE
symptoms, and safety.
Key Findings
Subjects administered C1-INH 20 U/kg exhibited a highly significant
reduction in the median time to onset of HAE symptom relief (30 minutes)
compared to subjects receiving placebo (1.5 hours), (p=0.0025).
Additionally, the proportion of subjects with worsening HAE symptoms after
receiving treatment was significantly lower in the C1-INH P 20 U/kg treated
group (p= 0.001). No drug-related serious adverse events were reported.
About I.M.P.A.C.T. 2
In a separate open label study presented at the AAAAI 64th Annual
Meeting, Timothy J. Craig, D.O., Professor of Medicine and Pediatrics at
Penn State University in Hershey, PA and his colleagues found C1-INH to be
a safe and effective treatment for HAE attacks at any body site.
"This study demonstrates that C1-INH replacement therapy is highly
effective at rapidly relieving symptoms of peripheral attacks, along with
facial, abdominal, and laryngeal attacks," said Dr. Craig. "These findings
demonstrate the versatility of C1-INH and confirm the long-standing
observation that C1-INH replacement effectively treats HAE attacks at many
different areas of the body."
Study Design and Key Findings
Findings of I.M.P.A.C.T. 2 were based on treatment with 20 U/kg
bodyweight of C1-INH in 355 episodes of HAE attacks at any body location in
39 patients. The main study end-points were: time to onset of symptom
relief; complete resolution of all symptoms, and safety.
The median time to the onset of relief was less than 33 minutes for
abdominal, facial, laryngeal, and peripheral attacks. The median times to
complete resolution of symptoms were less than 2.5 hours for laryngeal
attacks, 8 hours for abdominal and 24 hours for facial and peripheral
attacks.
No drug-related serious adverse events have been reported to date.
About CSL Behring
CSL Behring is a global leader in the plasma protein biotherapeutics
industry. Passionate about improving the quality of patients' lives, CSL
Behring manufactures and markets a range of safe and effective
plasma-derived and recombinant products and related services. The company's
therapies are used in the treatment of immune deficiency disorders,
hemophilia, von Willebrand disease, other bleeding disorders and inherited
emphysema. Other products are used for the prevention of hemolytic diseases
in the newborn, in cardiac surgery, organ transplantation and in the
treatment of burns. The company also operates one of the world's largest
plasma collection networks, ZLB Plasma. CSL Behring is a subsidiary of CSL
Limited, a biopharmaceutical company with headquarters in Melbourne,
Australia. For more information, visit cslbehring.
CSL Behring
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