– Kanuma is the First Approved Treatment for Patients Suffering from LAL-D, a Life-threatening Ultra-rare Metabolic Disorder –
CHESHIRE, Conn - Tuesday, September 1st 2015 [ME NewsWire]
(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.
“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.
Contacts
For Alexion Pharmaceuticals, Inc.
Media
Stephanie Fagan, 203-271-8223
Senior Vice President, Corporate Communications
Kim Diamond, 203-439-9600
Executive Director, Corporate Communications
Investors
Elena Ridloff, CFA, 203-699-7722
Executive Director, Investor Relations
Permalink: http://www.me-newswire.net/news/15584/en
No comments:
Post a Comment