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FDA Grants Breakthrough Therapy Designation to cPMP Replacement Therapy for Patients with Molybdenum Cofactor Deficiency (MoCD)

  FDA Grants Breakthrough Therapy Designation to cPMP Replacement Therapy for
  Patients with Molybdenum Cofactor Deficiency (MoCD) Type A

Business Wire

LAUSANNE, Switzerland -- October 24, 2013

Alexion Pharma International Sàrl, a subsidiary of  Alexion Pharmaceuticals,
Inc. (Nasdaq:ALXN), today announced that the U.S. Food and Drug Administration
(FDA) has granted a Breakthrough Therapy designation to cyclic pyranopterin
monophosphate (cPMP, or ALXN1101), an enzyme co-factor replacement therapy for
the treatment of patients with molybdenum cofactor deficiency (MoCD) type A, a
severe and life-threatening, ultra-rare, genetic metabolic disorder that
causes catastrophic and irreversible neurologic damage within the first weeks
of life.

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.^1

“In designating cPMP as a Breakthrough Therapy, the FDA recognizes the
life-threatening nature of MoCD Type A, the positive early clinical results
associated with cPMP, and the lack of any effective treatment options for
infants born with this devastating disease,” said Martin Mackay, Ph.D.,
Executive Vice President, Global Head of R&D at Alexion. “ALXN1101 is an
innovative approach to the treatment of MoCD Type A, as it targets an
essential step in the pathophysiology of the disease and the underlying cause
of the disease by replacing the naturally occurring cPMP molecule, which is
lacking in patients with MoCD Type A.”

Alexion looks forward to working closely with the FDA and obtaining FDA
guidance on the subsequent development of ALXN1101 for the treatment of
patients with MoCD Type A, including obtaining advice on generating evidence
needed to support approval of the drug in an efficient manner. Alexion has
initiated a natural history study in patients with MoCD Type A and has also
completed dosing with the synthetic cPMP in a study in healthy volunteers.

About Molybdenum Cofactor Deficiency (MoCD) Type A

MoCD Type A is a severe, ultra-rare and genetic metabolic disease affecting
newborns in which a genetic deficiency of cPMP results in the inability of the
body to form an essential cofactor called molybdenum cofactor, resulting in
its absence. This cofactor is essential for the appropriate functioning of
several critical metabolic enzymes. A deficiency or absence of this cofactor
leads to accumulation of toxic molecules, including the neurotoxin sulfite.
Clinically, the absence of this cofactor and the resulting build-up of sulfite
in the brain lead to damage and destruction of nerve cells, brain swelling,
uncontrollable seizures, catastrophic and irreversible brain damage, and
ultimately, death.^2 There are currently no treatment options for patients
with MoCD Type A.

About cPMP/ALXN1101

ALXN1101 is a synthetic version of cPMP and is designed to replace the
naturally occurring cPMP lacking in infants with MoCD Type A. ALXN1101
restores the deficient enzyme activity which then causes clearance of the
toxic metabolite sulfite thereby preventing the irreversible neurologic damage
observed in untreated patients with MoCD Type A. Encouraging early clinical
experience with an earlier form of cPMP replacement therapy has been reported
by independent investigators in Germany and Australia.^3

About Alexion

Alexion Pharmaceuticals, Inc. is a biopharmaceutical company focused on
serving patients with severe and ultra-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 Pharmaceuticals, Inc. can be
found at: www.alexionpharma.com.

[ALXN-G]

Safe Harbor Statement

This news release contains forward-looking statements, including statements
related to potential medical benefits of cPMP/ALXN1101 for molybdenum cofactor
deficiency (MoCD) Type A. 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 cPMP/ALXN1101
for MoCD Type A, delays in arranging satisfactory manufacturing capabilities
and establishing commercial infrastructure for cPMP/ALXN1101 for MoCD Type A,
the possibility that results of clinical trials are not predictive of safety
and efficacy results of cPMP/ALXN1101 in broader or different patient
populations, the risk that third party payors (including governmental
agencies) will not reimburse for the use of cPMP/ALXN1101 (if approved) at
acceptable rates or at all, the risk that estimates regarding the number of
patients with cPMP/ALXN1101 and observations regarding the natural history of
patients with cPMP/ALXN1101 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, 2013. 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. Public Law 112-144. U.S. Government Printing Office, July 9, 2012.
http://www.gpo.gov/fdsys/pkg/PLAW-112publ144/pdf/PLAW-112publ144.pdf.

2. Vijayakumar_Pediatric Neurology_45_2011; Per, Brain Dev.2005; Serrano,
Pediatr Radiol, 2007

3. Veldman A, Santamaria-Araujo JA, Sollazzo S, et al. Successful treatment of
molybdenum cofactor deficiency type A with cPMP. Pediatrics.
2010;125(5):e1249-54.

Contact:

Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Exec. Director, Corporate Communications
or
Kim Diamond, 203-439-9600
Senior Director, Corporate Communications
or
Investors:
Rx Communications
Rhonda Chiger, 917-322-2569
 
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