KING OF PRUSSIA, Pa.,
June 1, 2015 /PRNewswire/ -- Findings
from CSL Behring's RAPID study, the largest placebo-controlled
trial ever conducted in patients with alpha-1 antitrypsin
deficiency (AATD), demonstrate that the use of
Alpha1-Proteinase Inhibitor therapy may slow the
progressive loss of lung tissue experienced by these critically ill
patients. According to findings of RAPID (Randomized,
Placebo-controlled Trial of Augmentation Therapy in Alpha-1
Proteinase Inhibitor Deficiency), published by The Lancet,
patients with AATD treated with Alpha1-Proteinase
Inhibitor therapy exhibited a lower annual rate of lung density
decline compared to placebo, when measured using chest computed
tomography (CT), at full inspiration.
"RAPID is regarded as a landmark study validating almost two
decades of focus on the lung-density endpoint as the most sensitive
way to track lung tissue decline and the seven-year collaboration
of an international team of investigators," said Kenneth R. Chapman, MD, Director of the Asthma
& Airway Centre at the University Health Network in
Toronto, and lead author of the
paper. "Our findings provide additional evidence that treatment
with an Alpha1-Proteinase Inhibitor may slow the
accelerated loss of lung tissue that is a characteristic of this
potentially debilitating disease."
AATD is a hereditary condition that can severely affect a
patient's lung function. The condition is marked by a low level or
absence of alpha-1-proteinase inhibitor (A1-PI), a
natural protein that inhibits neutrophil elastase, thereby
preventing destruction of lung tissue. Severe deficiency of
A1-PI is associated with a strong tendency for the
development of emphysema, a form of chronic obstructive pulmonary
disease (COPD), and can significantly impact everyday life and life
expectancy. According to a recent registry, emphysema affects
54 percent of diagnosed Alpha-1-deficient patients.
On average, it takes over seven years from the time a patient's
lung symptoms first appear until a proper diagnosis is made. And
nearly half (43 percent) of patients see at least three physicians
before being diagnosed. Approximately 90 percent of individuals
with AATD are not diagnosed or are thought to have other
conditions, such as asthma or smoking-related COPD.
"We are excited that the results of this important study in
Alpha-1 have been published in the highly-respected journal The
Lancet," said John Walsh,
co-founder, President and CEO of the Alpha-1 Foundation. "We
commend CSL Behring for their outstanding commitment to the Alpha-1
community and advancing the understanding and treatment of the
disease. These results further support the use of augmentation
therapy in the treatment of Alpha-1, and we hope they bolster
efforts of Alpha-1 communities around the world to win access to
therapy."
Study Design and Findings
According to study protocol,
the effect of CSL Behring's Alpha1-Proteinase Inhibitor
therapy on the progression of emphysema, the primary endpoint of
the study, was measured by CT scan over 24 months. The primary
statistical endpoint was assessed by the rate of lung density loss
at total lung capacity (TLC) and functional residual capacity (FRC)
combined. This combined measure was not statistically different
between the treatment and placebo group. However, when
measured at TLC (or full inspiration), there was a 34 percent
reduction in the annual rate of lung density decline compared with
placebo.
Serious adverse events were not statistically different between
groups, with one death in the A1-PI group and three
deaths in the placebo group.
RAPID was a multicenter, double-blind, randomized, parallel
group, placebo-controlled study comparing the efficacy and safety
of CSL Behring's Alpha1-Proteinase Inhibitor therapy
with placebo in patients with emphysema due to AATD. The
participants, 180 non-smokers aged 18-65, were randomly assigned to
receive A1-PI intravenously 60 mg/kg weekly or
placebo.
About Zemaira
Zemaira is a highly-purified form of Alpha1
Proteinase Inhibitor (human) currently approved in Brazil, New
Zealand, and the US, where it is indicated for chronic
augmentation and maintenance therapy in adults with
Alpha1 deficiency and clinical evidence of
emphysema.
CSL Behring is seeking additional approvals around the world for
this A1-PI therapy, which is currently under review with
the European Medicines Agency.
Important Safety Information
Alpha1-Proteinase
Inhibitor (Human), Zemaira® is indicated to raise the plasma level
of alpha1-proteinase inhibitor (A1-PI) in patients with
A1-PI deficiency and related emphysema. The effect of
this raised level on the frequency of pulmonary exacerbations and
the progression of emphysema have not been established in clinical
trials.
Zemaira may not be suitable for everyone; for example, people
with known hypersensitivity to components used to make Zemaira,
those with a history of anaphylaxis or severe systemic response to
A1-PI products, and those with certain IgA deficiencies.
If you think any of these may apply to you, ask your doctor.
Early signs of hypersensitivity reactions to Zemaira include
hives, rash, tightness of the chest, unusual breathing difficulty,
wheezing, and feeling faint. Immediately discontinue use and
consult with physician if such symptoms occur.
In clinical studies, the following adverse reactions were
reported in at least 5% of subjects receiving Zemaira: headache,
sinusitis, upper respiratory infection, bronchitis, fatigue,
increased cough, fever, injection-site bleeding, nasal symptoms,
sore throat, and swelled blood vessels.
Because Zemaira is made from human blood, the risk of
transmitting infectious agents, including viruses and,
theoretically, the Creutzfeldt-Jakob disease (CJD) agent, cannot be
completely eliminated.
Please see full prescribing information for Zemaira.
You are encouraged to report negative side effects of
prescription drugs to the FDA. Visit http://www.fda.gov/medwatch,
or call1-800-FDA-1088.
About CSL Behring
CSL Behring is a leader in the
plasma protein therapeutics industry. Committed to saving lives and
improving the quality of life for people with rare and serious
diseases, the company manufactures and markets a range of
plasma-derived and recombinant therapies worldwide.
CSL Behring therapies are used around the world to treat
coagulation disorders including hemophilia and von Willebrand
disease, primary immune deficiencies, hereditary angioedema and
inherited respiratory disease, and neurological disorders in
certain markets. The company's products are also used in cardiac
surgery, organ transplantation, burn treatment and to prevent
hemolytic disease of the newborn.
CSL Behring operates one of the world's largest plasma
collection networks, CSL Plasma. CSL Behring is a global
biopharmaceutical company and a member of the CSL Group of
companies. The parent company, CSL Limited (ASX:CSL), is
headquartered in Melbourne,
Australia. For more information, visit
http://www.cslbehring.com/.
Contact:
Jennifer Seiler
CSL Behring
O: 610-878-4802
jennifer.seiler@cslbehring.com
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