– Strensiq is the First Approved Treatment in
Europe for Patients Suffering from HPP, a Life-Threatening
Ultra-Rare Metabolic Disorder –
Alexion Pharmaceuticals, Inc. (NASDAQ: ALXN) announced today
that the European Commission has approved Strensiq™ (asfotase alfa)
for long-term enzyme replacement therapy in patients with
pediatric-onset hypophosphatasia (HPP) to treat the bone
manifestations of the disease. The Summary of Product
Characteristics (SmPC) states that HPP is associated with multiple
bone manifestations including rickets/osteomalacia, altered calcium
and phosphate metabolism, impaired growth and mobility, respiratory
compromise that may require ventilation, and vitamin B6-responsive
seizures. Strensiq is the first therapy approved in the European
Union (EU) for the treatment of patients with HPP, a
life-threatening, ultra-rare metabolic disorder. 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.
“Hypophosphatasia is an extremely rare disorder that can have
devastating consequences for patients and families. Without
treatment, patients may face significant challenges related to
development, growth, and mobility, with an extremely high risk of
mortality in infants,” said PD Dr. med Christine Hofmann,
Children’s Hospital, University of Würzburg, Pediatric Rheumatology
and Osteology Section, Würzburg, Germany. “I am very pleased that
patients with pediatric-onset HPP in Europe now have an approved
treatment that addresses the underlying cause of their genetic,
lifelong metabolic disease by replacing tissue non-specific
alkaline phosphatase.”
HPP is a genetic and progressive metabolic disease in which
patients experience devastating effects on multiple systems of the
body, leading to debilitating or life-threatening complications. It
is an ultra-rare disease, which is defined as a disease that
affects fewer than 20 patients per one million in the general
population.1 HPP is characterized by defective bone mineralization
that can lead to deformity of bones and other skeletal
abnormalities, as well as systemic complications such as profound
muscle weakness, seizures, pain, and respiratory failure leading to
premature death in infants.2-6
"As the first approved treatment for pediatric-onset HPP in
Europe, Strensiq is an innovative therapy for patients suffering
from this devastating and life-threatening ultra-rare disease. We
are pleased that the EU label will allow any patient who had
symptoms of HPP prior to the age of 18 to be eligible for
treatment,” said David Hallal, Chief Executive Officer of Alexion.
“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 pediatric-onset HPP have access to Strensiq, a
life-transforming treatment, as quickly as possible.”
The EC has granted marketing authorization for Strensiq for
long-term enzyme replacement therapy in patients with
pediatric-onset HPP to treat the bone manifestations of the
disease. The SmPC states that HPP is associated with multiple bone
manifestations including rickets/osteomalacia, altered calcium and
phosphate metabolism, impaired growth and mobility, respiratory
compromise that may require ventilation, and vitamin B6-responsive
seizures. The natural history of untreated infant hypophosphatasia
patients suggests high mortality if ventilation is required. The
SmPC also indicates that 71% of infant patients treated with
Strensiq who required ventilation support remain alive and continue
on treatment.
The EC approval of Strensiq applies to all 28 EU member states
as well as Iceland, Norway, and Lichtenstein and follows the June
2015 positive opinion granted by the Committee for Medicinal
Products for Human Use (CHMP). Strensiq has also been approved for
the treatment of HPP by the Japanese Ministry of Health, Labour and
Welfare and by Health Canada. The FDA granted Breakthrough Therapy
designation for Strensiq and accepted Alexion’s Biologics License
Application (BLA) for Priority Review.
Clinical Data
The approval of Strensiq in the EU was based on clinical data
from four pivotal prospective studies and their extensions,
comprising 68 patients with pediatric-onset HPP (ranging from
newborns to 66 years of age). Study results showed that patients
with pediatric-onset HPP treated with Strensiq demonstrated rapid
and sustained improvements in bone mineralization, as measured by
the Radiographic Global Impression of Change (RGI-C) scale, which
evaluates the severity of rickets based on X-ray images. Patients
in the clinical studies also had improvements in skeletal
structure, as demonstrated by x-ray appearance of joints, by
histological appearance of bone biopsy material, and by apparent
catch-up height-gain.
The most common adverse reactions observed in clinical studies
were injection site reactions and injection-associated adverse
reactions. Most of these reactions were non-serious and mild to
moderate in intensity.
About Hypophosphatasia (HPP)
HPP is a genetic, chronic and progressive ultra-rare metabolic
disease characterized by defective bone mineralization that can
lead to destruction and deformity of bones, profound muscle
weakness, seizures, respiratory failure and premature death.2-6
HPP is caused by mutations in the gene encoding an enzyme known
as tissue non-specific alkaline phosphatase (TNSALP).2,3 The
genetic deficiency in HPP can affect people of all ages.2 HPP
is traditionally classified by the age of the patient at the onset
of symptoms of the disease, with infantile- and juvenile-onset HPP
defined as manifestation of the first symptom prior to 18 years of
age.
HPP can have devastating consequences for patients at any stage
of life.2 In a natural history study, infants who had their
first symptom of HPP within the first 6 months of life had high
mortality, with an overall mortality rate of 73% at 5
years.7 In these patients, mortality is primarily due to
respiratory failure.2,6,8 In patients surviving to adolescence
and adulthood, long-term clinical sequelae include recurrent and
non-healing fractures, profound muscle weakness, debilitating pain
and the requirement for ambulatory assistive devices such as
wheelchairs, wheeled walkers and canes.2,5
About Strensiq™ (asfotase alfa)
Strensiq™ (asfotase alfa) is an innovative enzyme
replacement therapy designed to address the underlying cause of
HPP—a deficiency of TNSALP activity. By replacing the defective
enzyme, treatment with Strensiq aims to prevent or reverse the
mineralization defects of the skeleton, thereby preventing serious
skeletal and systemic morbidity and premature death.
Strensiq is approved in Japan and Canada as a treatment for
patients with HPP, and a Biologics License Application for Strensiq
has been accepted for priority review by the U.S. Food and Drug
Administration (FDA).
Important Safety Information
Severe allergic-type hypersensitivity reactions are possible in
patients treated with Strensiq, including urticaria, difficulty
breathing and/or cardiovascular collapse. Administration of
Strensiq may result in local injection site reactions.
Craniosynostosis have been reported in patients less than
5 years of age. Ophthalmic (conjunctival and corneal)
calcification and nephrocalcinosis have been reported in patients
treated with Strensiq. There are insufficient data to establish a
causal relationship between exposure to Strensiq and progression of
craniosynostosis or between exposure to Strensiq and ectopic
calcification.
Serum parathyroid hormone concentration may increase in patients
administered Strensiq. Patients taking Strensiq may display
disproportionate weight increase.
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 Statement
This news release contains forward-looking statements, including
statements related to potential medical benefits of
Strensiq™(asfotase alfa) for hypophosphatasia (HPP).
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 Strensiq for HPP, delays in arranging satisfactory
manufacturing capabilities and establishing commercial
infrastructure for Strensiq for HPP, the possibility that results
of clinical trials are not predictive of safety and efficacy
results of Strensiq in broader or different patient populations,
the risk that third party payors (including governmental agencies)
will not reimburse for the use of Strensiq at acceptable rates or
at all, the risk that estimates regarding the number of patients
with Strensiq and observations regarding the natural history of
patients with Strensiq 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. Rockman-Greenberg C. Hypophosphatasia. Pediatr Endocrinol Rev.
2013; 10(suppl 2):380-388. 3. Whyte MP. Hypophosphatasia: nature’s
window on alkaline phosphatase function in humans. In: Bilezikian
JP, Raisz LG, Martin TJ, eds. Principles of Bone Biology. Vol 1.
3rd ed. San Diego, CA: Academic Press; 2008:1573-1598. 4. Whyte MP,
Greenberg CR, Salman N, et al. Enzyme-replacement therapy in
life-threatening hypophosphatasia. N Engl J Med. 2012;
366(10):904-913. 5. Seshia SS, Derbyshire G, Haworth JC,
Hoogstraten J. Myopathy with hypophosphatasia. Arch Dis Child.
1990; 65(1):130-131. 6. Baumgartner-Sigl S, Haberlandt E, Mumm S,
et al. Pyridoxine-responsive seizures as the first symptom of
infantile hypophosphatasia caused by two novel missense mutations
(c.677T>C, p.M226T; c.1112C>T, p.T371I) of the
tissue-nonspecific alkaline phosphatase gene. Bone. 2007;
40(6):1655-1661. 7. Whyte MP, Leung E, Wilcox W, et al.
Hypophosphatasia: a retrospective natural history study of the
severe perinatal and infantile forms. Poster presented at the 2014
Pediatric Academic Societies and Asian Society for Pediatric
Research Joint Meeting, Vancouver, B.C., Canada, May 5, 2014.
Abstract 752416. 8. Whyte MP, Rockman-Greenberg C, Hofmann C, et
al. Improved survival with asfotase alfa treatment in pediatric
patients with hypophosphatasia at high risk of death. Poster
presented at the American Society for Bone and Mineral Research
(ASBMR) 2014 Annual Meeting, Houston, September 14, 2014. Abstract
1097.
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version on businesswire.com: http://www.businesswire.com/news/home/20150831006265/en/
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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