|[April 11, 2014]
Researchers to Present New Data on Asfotase Alfa in Infants and Juveniles with Hypophosphatasia at the Joint Meeting of the Pediatric Academic Societies and the Asian Society for Pediatric Research
CHESHIRE, Conn. --(Business Wire)--
Alexion Pharmaceuticals, Inc. (Nasdaq: ALXN) today announced that
researchers are scheduled to present new data from the extension phase
of two clinical studies examining the long-term efficacy and safety of
asfotase alfa in infants and juveniles with hypophosphatasia (HPP) at
the joint meeting of the Pediatric Academic Societies (PAS) and the
Asian Society for Pediatric Research, which takes place May 3-6, 2014 in
Vancouver, B.C., Canada. The meeting will also feature the presentation
of results from a retrospective natural history study of patients with
perinatal and infantile HPP.
HPP is an inherited, ultra-rare metabolic disorder that can lead to
progressive damage to multiple vital organs, destruction and deformity
of bones, and death. The U.S. Food and Drug Administration (FDA) granted
Breakthrough Therapy designation for asfotase alfa in pediatric-onset
HPP, defined as patients whose first signs or symptoms of HPP occurred
prior to 18 years of age, including perinatal-, infantile-, and
juvenile-onset forms of the disease.
Abstracts summarizing the asfotase alfa studies and the HPP natural
history study were published today on the PAS website and are available
to conference registrants at: http://www.pas-meeting.org/.
The following abstract will be presented in a poster session on May 4,
2014 from 4:15pm - 7:30pm Pacific Daylight Time (PDT):
Abstract 752396: "Asfotase Alfa: Sustained Efficacy and Tolerability
in Infants and Young Children with Life-Threatening Hypophosphatasia,"
Whyte, et al.
The following abstracts will be presented in a poster session on May 5,
2014 from 4:15pm - 7:30pm PDT:
Abstract 752577: "Asfotase Alfa: Long-Term Safety and Efficacy in
Children with Hypophosphatasia," Madson, et al.
Abstract 752416: "Hypophosphatasia: A Retrospective Natural History
Study of the Severe Perinatal and Infantile Forms," Whyte, et al.
About Hypophosphatasia (HPP)
Hypophosphatasia (HPP) is a chronic, life-threatening, genetic, and
ultra-rare metabolic disease characterized by defective bone
mineralization that can lead to destruction and deformity of bones,
profound muscle weakness,seizures, and respiratory failure.1-4
HPP is caused by a genetic deficiency of an enzyme known as tissue
non-specific alkaline phosphatase (TNSALP).1
The genetic deficiency in HPP can affect people of all ages.1 HPP
is traditionally classified by the age of the patient at the onset of
symptoms of the disease. Patients with perinatal-onset HPP manifest
their first signs of disease in utero or at birth. This form of the
disease often leads to death at or soon after birth. Those patients who
survive birth often have severe rickets and severely compromised
Patients with infantile-onset HPP develop their first signs or symptoms
of HPP before 6 months of age. Individuals with this form of disease
develop rickets, skeletal abnormalities, fractures, failure to thrive
and respiratory failure. The prognosis of these patients may be poor
with mortality estimated to be as high as at 50%.1
Patients with juvenile-onset HPP exhibit their first signs or symptoms
of HPP after 6 months of age and before 18 years of age. Individuals
with this form of the disease are at risk for rickets, skeletal
complications including fractures, and can have delayed acquisition of
age-appropriate motor skills due to skeletal hypomineralization and
muscle weakness leading to the need for walking assistance; some may
About Asfotase Alfa
Asfotase alfa is an investigational, highly innovative, first-in-class
targeted enzyme replacement therapy. Asfotase alfa is designed to
address the underlying cause of HPP by normalizing the genetically
defective metabolic process, and preventing or reversing the severe and
potentially life-threatening complications of life-long dysregulated
According to the FDA, a Breakthrough Therapy designation is designed to
expedite the development of a drug to treat a serious or
life-threatening disease when preliminary clinical evidence indicates
that the drug may demonstrate substantial improvement over existing
therapies on one or more clinically significant endpoints. The
Breakthrough Therapy designation is part of the FDA Safety and
Innovation Act (FDASIA) of 2012.6
Alexion is a biopharmaceutical company focused on serving patients with
severe and rare disorders through the innovation, development and
commercialization of life-transforming therapeutic products. Alexion is
the global leader in complement inhibition and has developed and markets
Soliris® (eculizumab) as a treatment for patients with PNH and aHUS, two
debilitating, ultra-rare and life-threatening disorders caused by
chronic uncontrolled complement activation. Soliris is currently
approved in nearly 50 countries for the treatment of PNH, and in the
United States, European Union, Japan and other countries for the
treatment of aHUS. Alexion is evaluating other potential indications for
Soliris in additional severe and ultra-rare disorders beyond PNH and
aHUS, and is developing other highly innovative biotechnology product
candidates across multiple therapeutic areas. This press release and
further information about Alexion can be found at: www.alexionpharma.com.
1. Whyte MP. Hypophosphatasia. In: Glorieux FH, Jueppner H, Pettifor J,
eds. Pediatric bone: biology and diseases. 3rd ed. San Diego, CA (News - Alert):
Academic Press, 2012: 771-94.
2. Seshia SS, Derbyshire G, Haworth JC, Hoogstraten J. Myopathy with
Hypophosphatasia. Arch Dis Child. 1990; 65(1):130-1.
3. Whyte MP. Hypophosphatasia: Nature's window on alkaline phosphatase
function in humans. In: Principles of Bone Biology, 3rd Ed. Part II,
Chapter 73: Molecular Mechanisms of Metabolic Bone Disease, Academic
Press, 2008: 1573-98.
4. Silver MM, Vilos GA, Milne KJ. Pulmonary hypoplasia in neonatal
hypophosphatasia. Pediatr Pathol. 1998; 8:483-93.
5. Whyte MP. Hypophosphatasia and the extracellular metabolism of
inorganic pyrophosphate: Clinical and laboratory aspects. Crit Rev Clin
Lab Sci. 1991; 23:175-195.
6. Public Law 112-144. U.S. Government Printing Office, July 9, 2012. http://www.gpo.gov/fdsys/pkg/PLAW-112publ144/pdf/PLAW-112publ144.pdf.
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