Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
the U.S. Food and Drug Administration (FDA) has granted orphan drug
designation (ODD) to ALXN1210, a highly innovative, longer-acting
anti-C5 antibody that inhibits terminal complement, which is being
evaluated for the treatment of patients with paroxysmal nocturnal
hemoglobinuria (PNH). PNH is a debilitating, ultra-rare,
life-threatening blood disorder in which uncontrolled activation of
complement, a component of the immune system, results in hemolysis
(destruction of a patient’s red blood cells).1
“Alexion is committed to achieving the highest levels of
innovation to address the needs of patients suffering from PNH, a
devastating ultra-rare disorder,” said Martin Mackay, Ph.D.,
Executive Vice President and Global Head of R&D at Alexion. “We
are pleased that the FDA has recognized the potential for ALXN1210
to offer a significant therapeutic advantage for patients with PNH.
Data from our ongoing clinical studies have shown rapid, complete,
and sustained complement inhibition in treated patients, and we
look forward to continuing to evaluate this highly innovative
molecule in our Phase 3 trial of ALXN1210 administered every eight
weeks.”
Alexion is currently enrolling patients in Phase 3 trials of
ALXN1210 in patients with PNH as well as in patients with atypical
hemolytic uremic syndrome (aHUS), another ultra-rare and
life-threatening disease caused by chronic uncontrolled complement
activation. More information on these clinical trials is available
at www.clinicaltrials.gov under the identifiers NCT02946463 and
NCT02949128. In June 2016, ALXN1210 was granted ODD by the European
Commission for the treatment of patients with PNH. ALXN1210 is
protected by a composition of matter patent in the U.S. and Europe
through 2035. ALXN1210 is not approved in any country.
The FDA, through its Office of Orphan Products Development
(OOPD), grants orphan status to drugs and biologic products that
are intended for the safe and effective treatment, diagnosis, or
prevention of rare diseases or disorders that affect fewer than
200,000 people in the United States. ODD provides a drug developer
with certain benefits and incentives, including a period of
marketing exclusivity if regulatory approval is ultimately received
for the designated indication.
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is an ultra-rare blood disorder in which chronic,
uncontrolled activation of complement, a component of the normal
immune system, results in hemolysis (destruction of the patient's
red blood cells). PNH strikes people of all ages, with an average
age of onset in the early 30s.1 Approximately 10 percent of
all patients first develop symptoms at 21 years of age or
younger.2 PNH develops without warning and can occur in men
and women of all races, backgrounds and ages. PNH often goes
unrecognized, with delays in diagnosis ranging from one to more
than 10 years.3 In the period of time before treatment was
available, it had been estimated that approximately one-third of
patients with PNH did not survive more than five years from the
time of diagnosis.4 PNH has been identified more commonly
among patients with disorders of the bone marrow, including
aplastic anemia (AA) and myelodysplastic syndromes
(MDS).5,6,7 In patients with thrombosis of unknown origin, PNH
may be an underlying cause.8
About ALXN1210
ALXN1210 is a highly innovative, longer-acting anti-C5 antibody
discovered and developed by Alexion that inhibits terminal
complement. In early studies, ALXN1210 demonstrated rapid,
complete, and sustained reduction of free C5 levels.9 Alexion
has completed enrollment in two ongoing clinical studies of
ALXN1210 in patients with PNH—a Phase 1/2 dose-escalating study and
an open-label, multi-dose Phase 2 study that is also evaluating
longer dosing intervals beyond 8 weeks.
ALXN1210 is currently in Phase 3 trials in patients with PNH and
aHUS. In addition, Alexion is conducting a Phase 1 study to
evaluate a new formulation of ALXN1210 administered subcutaneously
in healthy volunteers.
In June 2016, the European Commission granted Orphan Drug
Designation (ODD) to ALXN1210 for the treatment of patients with
PNH.
About Alexion
Alexion is a global biopharmaceutical company focused on
developing and delivering life-transforming therapies for patients
with devastating and rare disorders. Alexion is the global leader
in complement inhibition and has developed and commercializes 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.
In addition, Alexion’s metabolic franchise includes two highly
innovative enzyme replacement therapies for patients with
life-threatening and ultra-rare disorders, hypophosphatasia (HPP)
and lysosomal acid lipase deficiency (LAL-D). Alexion is advancing
the most robust rare disease pipeline in the biotech industry with
highly innovative product candidates in multiple therapeutic areas.
This press release and further information about Alexion can be
found at: www.alexion.com.
[ALXN-G]
Forward-Looking Statements
This press release contains forward-looking statements,
including statements related to Alexion's development plans for
ALXN1210, the medical benefits of ALXN1210 for the treatment of PNH
and aHUS, medical and commercial potential of ALXN1210, and plans
for regulatory filings for ALXN1210. Forward-looking statements are
subject to factors that may cause Alexion's results and plans to
materially differ from those expected, including for example,
decisions of regulatory authorities regarding marketing approval or
material limitations on the marketing of our products, delays,
interruptions or failures in the manufacture and supply of our
products and our product candidates, progress in establishing and
developing commercial infrastructure, failure to satisfactorily
address matters raised by the FDA and other regulatory agencies,
the possibility that results of clinical trials are not predictive
of safety and efficacy results of our products in broader patient
populations in the disease studied or other diseases, the risk that
strategic transactions will not result in short-term or long-term
benefits, the possibility that current results of commercialization
are not predictive of future rates of adoption of Soliris in PNH,
aHUS or other diseases, the possibility that clinical trials of our
product candidates could be delayed or that additional research and
testing is required by regulatory agencies, including for ALXN1210,
the adequacy of our pharmacovigilance and drug safety reporting
processes, the risk that third party payors (including governmental
agencies) will not reimburse or continue to reimburse for the use
of our products at acceptable rates or at all, risks regarding
government investigations, including investigations of Alexion by
the SEC and DOJ, the risk that anticipated regulatory filings are
delayed, including for ALXN1210, the risk that estimates regarding
the number of patients with PNH, aHUS, HPP and LAL-D are
inaccurate, the risks of shifting foreign exchange rates, and a
variety of other risks set forth from time to time in Alexion's
filings with the U.S. 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 September 30, 2016 and in
our other filings with the U.S. Securities and Exchange Commission.
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. Socié G, Mary JY, de Gramont A, et al. Paroxysmal
nocturnal haemoglobinuria: long-term follow-up and prognostic
factors. Lancet. 1996: 348:573-577. 2. Parker C, Omine M, Richards
S, et al. Diagnosis and management of paroxysmal nocturnal
hemoglobinuria. Blood. 2005;106(12):3699-3709. 3. Hillmen P, Lewis
SM, Bessler M, Luzzatto L, Dacie JV. Natural history of paroxysmal
nocturnal hemoglobinuria. N Engl J Med. 1995;333:1253-1258. 4.
Dacie JV, Lewis SM. Paroxysmal nocturnal haemoglobinuria: clinical
manifestations, haematology, and nature of the disease. Ser Haemat.
1972;5:3-23. 5. Wang H, Chuhjo T, Yasue S, Omine M, Naka S.
Clinical significance of a minor population of paroxysmal nocturnal
hemoglobinuria-type cells in bone marrow failure syndrome. Blood.
2002;100 (12):3897-3902. 6. Iwanga M, Furukawa K, Amenomori T, et
al. Paroxysmal nocturnal haemoglobinuria clones in patients with
myelodysplastic syndromes. Br J Haematol. 1998;102(2):465-474. 7.
Maciejewski JP, Rivera C, Kook H, Dunn D, Young NS. Relationship
between bone marrow failure syndromes and the presence of
glycophosphatidyl inositol-anchored protein-deficient clones. Br J
Haematol. 2001;115:1015-1022. 8. Hill A, Kelly RJ, Hillmen P.
Thrombosis in paroxysmal nocturnal hemoglobinuria. Blood.
2013;121:4985-4996. 9. Sahelijo L, Mujeebuddin A, Mitchell D, et
al. First in human single-ascending dose study: safety, biomarker,
pharmacokinetics and exposure-response relationships of ALXN1210, a
humanized monoclonal antibody to C5, with marked half-life
extension and potential for significantly longer dosing intervals.
Blood. 2015;126 (23):4777.
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version on businesswire.com: http://www.businesswire.com/news/home/20170106005146/en/
Alexion Pharmaceuticals, Inc.MediaStephanie Fagan,
475-230-3777Senior Vice President, Corporate CommunicationsorKim
Diamond, 475-230-3775Executive Director, Corporate
CommunicationsorInvestorsElena Ridloff, CFA, 475-230-3601Vice
President, Investor Relations
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