– Kanuma is the First Approved Treatment for Patients Suffering from
LAL-D, a Life-threatening Ultra-rare Metabolic Disorder –
CHESHIRE, Conn. -- (Business Wire)
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.
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“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.
[ALXN-G]
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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Contacts:
Alexion Pharmaceuticals, Inc.
Media:
Stephanie Fagan,
203-271-8223
Senior Vice President, Corporate Communications
or
Kim
Diamond, 203-439-9600
Executive Director, Corporate Communications
or
Investors:
Elena
Ridloff, CFA, 203-699-7722
Executive Director, Investor Relations
Source: Alexion Pharmaceuticals, Inc.
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