Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
the U.S. Food and Drug Administration (FDA) has granted orphan
drug designation (ODD) to ALXN1007, a novel anti-inflammatory
monoclonal antibody targeting complement protein C5a, for the
treatment of acute graft-versus-host disease (GVHD). Alexion is
currently investigating ALXN1007 in patients with acute GVHD of the
lower gastrointestinal tract (GI-GVHD), a severe and
life-threatening rare autoimmune disease that can occur as a
complication of stem cell or bone marrow transplantation.1,2,3
“There is an urgent need for an effective treatment for GI-GVHD,
which causes severe symptoms and often leads to mortality following
allogeneic hematopoietic stem cell transplantation. Currently, even
with best available care, about one-third of severely affected
patients die from this devastating complication within six months
of onset,” said Martin Mackay, Ph.D., Executive Vice President and
Global Head of R&D at Alexion. “Alexion is pleased that the FDA
has granted orphan drug designation to ALXN1007, recognizing its
potential to improve response rates in patients facing acute
GI-GVHD.”
ALXN1007 is currently being evaluated in a Phase 2 study in
patients with newly diagnosed acute GI-GVHD. More information on
this trial is available at www.clinicaltrials.gov under the
identifier NCT02245412. In August of 2016, ALXN1007 was granted ODD
by the European Commission. ALXN1007 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 Graft-Versus-Host Disease of the Lower GI tract
(GI-GVHD)
GI-GVHD is an immune-mediated disease that affects approximately
10 percent of patients who receive an allogeneic hematopoietic stem
cell transplant or bone marrow transplant.1,2,4 Patients with
severe, acute GI-GVHD have a 30 to 40 percent mortality rate within
the first six months post-transplant.5 There are currently
limited treatment options in acute GI-GVHD.
About ALXN1007
ALXN1007 is a novel anti-inflammatory monoclonal antibody
targeting complement protein C5a being evaluated in a Phase 2 trial
for patients with acute GI-GVHD.
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 news release contains forward-looking statements, including
statements related to potential medical benefits of ALXN1007 for
the treatment of graft-versus-host disease (GVHD). 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 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, 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 the SEC and DOJ investigations, 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 June 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.
Jagasia M, Arora M, Flowers MED, et al.
Risk factors for acute GVHD and survival after hematopoietic cell
transplantation. Blood. 2012;119(1):296-307.
2.
MacMillan ML, DeFor TE, Weisdorf DJ. What
predicts high risk acute graft-versus-host disease (GVHD) at
onset?: identification of those at highest risk by a novel acute
GVHD risk score. Br J Haematol. 2012;157:732-741.
3.
Iqbal N, Salzman D, Lazenby AJ, Wilcox CM.
Diagnosis of gastrointestinal graft-versus-host disease. Am J
Gastroenterol. 2000 Nov;95(11):3034-8.
4.
Weisdorf DJ, Snover DC, Haake R, et al.
Acute upper gastrointestinal graft-versus-host disease: clinical
significance and response to immunosuppressive therapy. Blood.
1990;76(3):624-629.
5.
Bolaños-Meade J, Logan BR, Alousi AM, et
al. Phase 3 clinical trial of steroids/mycophenolate mofetil vs
steroids/placebo as therapy for acute GVHD: BMT CTN 0802. Blood.
2014;124(22):3221–3227.
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version on businesswire.com: http://www.businesswire.com/news/home/20161019006469/en/
Alexion Pharmaceuticals, Inc.Media:Stephanie Fagan,
475-230-3777Senior Vice President, Corporate CommunicationsorKim
Diamond, 475-230-3775Executive Director, Corporate
CommunicationsorInvestors:Elena Ridloff, CFA, 475-230-3601Vice
President, Investor Relations
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