– Kanuma is the First Approved Treatment for
Patients Suffering from LAL-D, a Life-threatening Ultra-rare
Metabolic Disorder –
Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) announced today
that the European Commission (EC) has approved Kanuma™ (sebelipase
alfa) for long-term enzyme replacement therapy (ERT) in patients of
all ages with lysosomal acid lipase deficiency (LAL-D). Kanuma, an
innovative ERT, is the first approved treatment in the European
Union for patients with LAL-D, a genetic and progressive ultra-rare
metabolic disease in which patients suffer multi-organ damage and
premature death. Alexion expects to begin serving patients in
Germany in October and is now commencing reimbursement processes
with healthcare authorities in each of the major European
countries.
“Today’s approval is a crucial milestone for patients with
LAL-D, a grave condition that can have devastating consequences for
patients of all ages,” said Vassili Valayannopoulos, M.D., Ph.D.,
investigator in the Kanuma pivotal studies, Hôpital Necker-Enfants
Malades and IMAGINE Institute, Paris. “In clinical studies, 67% of
infants treated with Kanuma survived beyond 12 months of age,
whereas without treatment, these patients would have faced a
near-certain fatal outcome. In pediatric and adult patients, Kanuma
was also shown to reduce the markers of liver injury and lipid
accumulation, which can lead to serious and life-threatening
complications.”
LAL-D is a genetic, chronic and progressive metabolic disease in
which infants, children and adults suffer multi-organ damage and
premature death. It is an ultra-rare disease, which is defined as a
disease that affects fewer than 20 patients per one million of the
general population.1 Patients with LAL-D often experience a rapid
onset of life-threatening disease manifestations, and similar to
other liver diseases, many patients may be asymptomatic until they
experience a severe consequence of the disease. LAL-D is caused by
genetic mutations that result in a marked decrease or loss in LAL
enzyme activity in the lysosomes across multiple body tissues,
leading to the chronic build-up of cholesteryl esters and
triglycerides in the liver, blood vessel walls and other
tissues.2,3
“We are pleased that the European Commission has approved Kanuma
for patients of all ages with LAL-D, enabling us to serve infants,
children and adults in Europe with the first approved treatment for
this ultra-rare, severe and life-threatening disease,” said David
Hallal, Chief Executive Officer of Alexion. “In the absence of any
effective therapy, patients with LAL-D face devastating morbidities
including liver failure and premature mortality. We are grateful to
the investigators, patients, and their families who participated in
the clinical trials that made this approval possible and we are now
commencing reimbursement processes with healthcare authorities
throughout Europe to ensure that patients with LAL-D have access to
Kanuma, a life-transforming treatment, as quickly as possible.”
Kanuma is a highly innovative enzyme replacement therapy (ERT)
designed to address the underlying cause of LAL-D. The approval of
Kanuma applies to all 28 EU member states as well as Iceland,
Norway, and Lichtenstein and was granted under the accelerated
assessment procedure. The decision follows the June 2015 positive
opinion granted by the Committee for Medicinal Products for Human
Use (CHMP). In addition, the U.S. Food and Drug Administration
granted Breakthrough Therapy designation for Kanuma for LAL
Deficiency presenting in infants and accepted the Kanuma BLA
(Biologics License Application) for Priority Review.
Clinical Data
The approval of Kanuma in the EU was based on data from two
clinical studies and a supporting open-label extension study
comprising infant, pediatric, and adult patients with LAL-D. Study
results showed significant benefit in terms of survival (67%, or 6
out of 9) in patients with the infant form of LAL-D beyond 12
months, compared with 0 out of 21 patients in an untreated
historical cohort. Infant patients treated with Kanuma also had
improvements in liver parameters, including ALT and AST, as well as
weight gain within the first several weeks of treatment. In
pediatric and adult patients with LAL-D, treatment with Kanuma
resulted in normalization of ALT, reduction in liver fat content
and other markers of liver injury compared to placebo, as well as
significant improvements in lipid accumulation as measured by LDL-C
and HDL-C. In patients who received Kanuma during the double-blind
period and subsequently entered the open-label extension period,
reductions in ALT levels were maintained and further improvements
were seen in LDL-C and HDL-C.
The most serious adverse reactions experienced by 3% of patients
in clinical trials were signs and symptoms consistent with
anaphylaxis. Signs and symptoms included chest discomfort,
conjunctival injection, dyspnea, generalized and itchy rash,
hyperemia, mild eyelid edema, rhinorrhea, severe respiratory
distress, tachycardia, tachypnea and urticaria.
About Lysosomal Acid Lipase Deficiency (LAL-D)
LAL-D is a genetic, chronic and progressive ultra-rare metabolic
disease associated with devastating morbidities and premature
mortality. In patients with LAL-D, genetic mutations result in
decreased activity of the LAL enzyme. This leads to marked
accumulation of cholesteryl esters and triglycerides in vital
organs, blood vessels, and other tissues, resulting in progressive
and multi-organ damage including fibrosis, cirrhosis, liver
failure, accelerated atherosclerosis, cardiovascular disease, and
other devastating consequences.2,3
LAL-D affects patients of all ages with clinical manifestations
from infancy through adulthood and may have sudden and
unpredictable clinical complications. Infants experience profound
growth failure, liver fibrosis, and cirrhosis with a median age of
death at 3.7 months.4 In an observational study, approximately 50%
of children and adults with LAL-D progressed to fibrosis,
cirrhosis, or liver transplant in 3 years.5 The median age of onset
of LAL-D is 5.8 years and the disease can be diagnosed with a
simple blood test.6,7
About Kanuma™ (sebelipase alfa)
Kanuma™ (sebelipase alfa) is an innovative enzyme replacement
therapy designed to address the underlying cause of lysosomal acid
lipase deficiency (LAL-D) by aiming to reduce substrate
accumulation in the lysosomes of cells throughout the body,
including the liver, to prevent vital organ damage and premature
death.
The FDA granted Breakthrough Therapy designation for Kanuma for
LAL Deficiency presenting in infants and accepted the Kanuma BLA
for Priority Review. In addition, a New Drug Application for Kanuma
has been submitted to Japan’s Ministry of Health, Labour and
Welfare.
Important Safety Information
Hypersensitivity reactions, including anaphylaxis, have been
reported in patients treated with sebelipase alfa therefore,
appropriate medical support must be readily available when
sebelipase alfa is administered. If severe reactions occur, the
sebelipase alfa infusion should be immediately stopped and
appropriate medical treatment should be initiated. For patients who
have experienced allergic reactions during infusion, caution should
be exercised upon re-administration.
The most serious adverse reactions experienced by 3% of patients
in clinical studies were signs and symptoms consistent with
anaphylaxis. Signs and symptoms included chest discomfort,
conjunctival injection, dyspnoea, generalised and itchy rash,
hyperaemia, mild eyelid oedema, rhinorrhoea, severe respiratory
distress, tachycardia, tachypnoea and urticaria.
About Alexion
Alexion is a global biopharmaceutical company focused on
developing and delivering life-transforming therapies for patients
with devastating and rare disorders. Alexion developed and
commercializes Soliris® (eculizumab), the first and only approved
complement inhibitor to treat patients with paroxysmal nocturnal
hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS),
two life-threatening ultra-rare disorders. Alexion is also
establishing a premier global metabolic rare disease franchise,
which includes Kanuma™ (sebelipase alfa) for patients with
lysosomal acid lipase deficiency (LAL-D), and Strensiq™ (asfotase
alfa) for patients with hypophosphatasia (HPP). In addition,
Alexion is advancing the most robust rare disease pipeline in the
biotech industry, with highly innovative product candidates in
multiple therapeutic areas. As the global leader in complement
inhibition, the Company is strengthening and broadening its
portfolio of complement inhibitors across diverse platforms,
including evaluating potential indications for Soliris in
additional severe and ultra-rare disorders. This press release and
further information about Alexion can be found at:
www.alexion.com.
Forward-Looking Statements
This news release contains forward-looking statements, including
statements related to potential medical benefits of Kanuma™
(sebelipase alfa) for lysosomal acid lipase deficiency (LAL-D).
Forward-looking statements are subject to factors that may cause
Alexion’s results and plans to differ from those expected,
including, for example, decisions of regulatory authorities
regarding marketing approval or material limitations on the
marketing of Kanuma for LAL-D, delays in arranging satisfactory
manufacturing capabilities and establishing commercial
infrastructure for Kanuma for LAL-D, the possibility that results
of clinical trials are not predictive of safety and efficacy
results of Kanuma in broader or different patient populations, the
risk that third party payors (including governmental agencies) will
not reimburse for the use of Kanuma at acceptable rates or at all,
the risk that estimates regarding the number of patients with
Kanuma and observations regarding the natural history of patients
with Kanuma are inaccurate, and a variety of other risks set forth
from time to time in Alexion's filings with the Securities and
Exchange Commission, including but not limited to the risks
discussed in Alexion's Quarterly Report on Form 10-Q for the period
ended June 30, 2015. Alexion does not intend to update any of these
forward-looking statements to reflect events or circumstances after
the date hereof, except when a duty arises under law.
References
1.
REGULATION (EU) No 536/2014 OF THE
EUROPEAN PARLIAMENT AND OF THE COUNCIL of 16 April 2014 on clinical
trials on medicinal products for human use, and repealing Directive
2001/20/EC.
http://eur-lex.europa.eu/legal-content/EN/TXT/PDF/?uri=CELEX:32014R0536&qid=1421232837997&from=EN
2. Bernstein DL, et al. Chloesteryl ester storage disease: review
of the findings in 135 reported patients with an underdiagnosed
disease. J Hepatol. 2013;58:1230-43.
doi:10.1016/j.jhep.2013.02.014. 3. Reiner Z, et al. Lysosomal acid
lipase deficiency – an under-recognized cause of dyslipidemia and
liver dysfunction. Atherosclerosis. 2014;235:21-30.
doi:10.1016/j.atherosclerosis.2014.04.003. 4. Jones S et al. Severe
and rapid disease course in the natural history of infants with
lysosomal acid lipase deficiency. Mol Genet Metab. 2014
Feb;111(2):S57-58. 5. Data on file, Alexion. 6.
Burton et al. Clinical Features of
Lysosomal Acid Lipase Deficiency - a Longitudinal Assessment of 48
Children and Adults. J Pediatr Gastroenterol Nutr. 2015 August 6.
doi: 10.1097/MPG.0000000000000935
7. Hamilton J, et al. A new method for the measurement of lysosomal
acid lipase in dried blood spots using the inhibitor Lalistat 2.
Clin Chim Acta. 2012;413:1207-10. doi:10.1016/j.cca.2012.03.019.
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version on businesswire.com: http://www.businesswire.com/news/home/20150831006304/en/
For Alexion Pharmaceuticals, Inc.MediaStephanie Fagan,
203-271-8223Senior Vice President, Corporate CommunicationsorKim
Diamond, 203-439-9600Executive Director, Corporate
CommunicationsorInvestorsElena Ridloff, CFA, 203-699-7722Executive
Director, Investor Relations
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