Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
the Company has submitted marketing applications to the U.S. Food
and Drug Administration (FDA) and the European Medicines Agency
(EMA) to extend the indication for Soliris® (eculizumab) as a
treatment for patients with refractory generalized myasthenia
gravis (gMG) who are anti-acetylcholine receptor (AChR)
antibody-positive. The European submission has been validated by
the European Medicines Agency (EMA), marking the beginning of the
review process in Europe for this potential new indication for
Soliris. Both the U.S. and EU submissions are supported by the
comprehensive data from the Phase 3 REGAIN study.
If approved, Soliris would address a significant unmet need for
patients with refractory gMG who have largely exhausted
conventional therapy. Refractory gMG patients who are anti-AChR
antibody-positive are an ultra-rare segment of MG, a debilitating,
complement-mediated neuromuscular disease in which patients suffer
profound muscle weakness throughout the body, resulting in slurred
speech, impaired swallowing and choking, double vision, disabling
fatigue, shortness of breath due to respiratory muscle weakness,
frequent hospital and ICU visits with prolonged stays, and episodes
of respiratory failure.1-6
“Today there is an urgent need among patients suffering with
refractory gMG, as there are no effective therapies for this
ultra-rare and devastating disease population, causing patients to
face disabling limitations in their daily lives, including
difficulty walking, talking, swallowing, and breathing normally,”
said Martin Mackay, Ph.D., Executive Vice President and Global Head
of R&D at Alexion. “The U.S. and EU regulatory submissions put
us one step closer to accomplishing our goal of transforming the
lives of patients suffering with refractory gMG with anti-AChR
antibodies. We look forward to working with regulatory authorities
as they review our applications.”
Soliris has received Orphan Drug Designation (ODD) for the
treatment of patients with MG in the U.S. and EU. Soliris is not
approved in any country for the treatment of patients with
refractory gMG.
About Refractory Generalized Myasthenia Gravis
Refractory generalized myasthenia gravis (gMG) patients who are
anti-acetylcholine receptor (AChR) antibody-positive represent an
ultra-rare segment of patients with MG—a debilitating,
complement-mediated neuromuscular disease—who experience severe
morbidities despite currently available MG therapies.1,2,3
MG typically begins with weakness in the ocular muscles and
often progresses to the more severe and generalized form, known as
gMG, to include weakness of the head, neck, trunk, limb and
respiratory muscles.7 While most symptoms in gMG patients are
managed with conventional therapies, 10% to 15% of patients are
considered refractory—meaning they do not respond to multiple
conventional therapies and continue to suffer profound muscle
weakness throughout the body, resulting in slurred speech, impaired
swallowing and choking, double vision, disabling fatigue, shortness
of breath due to respiratory muscle weakness, frequent hospital and
ICU visits with prolonged stays, and episodes of respiratory
failure.4,5,6,8
Today, there are no therapies that are effective in this
ultra-rare population of patients suffering from refractory
gMG.
About Soliris® (eculizumab)
Soliris is a first-in-class terminal complement inhibitor
developed from the laboratory through regulatory approval and
commercialization by Alexion. Soliris is approved in the U.S.
(2007), European Union (2007), Japan (2010) and other countries as
the first and only treatment for patients with paroxysmal nocturnal
hemoglobinuria (PNH) to reduce hemolysis. PNH is a debilitating,
ultra-rare and life-threatening blood disorder, characterized by
complement-mediated hemolysis (destruction of red blood cells).
Soliris is also approved in the U.S. (2011), European Union (2011),
Japan (2013) and other countries as the first and only treatment
for patients with atypical hemolytic uremic syndrome (aHUS) to
inhibit complement-mediated thrombotic microangiopathy, or TMA
(blood clots in small vessels). aHUS is a debilitating, ultra-rare
and life-threatening genetic disorder characterized by
complement-mediated TMA. Soliris is not indicated for the treatment
of patients with Shiga-toxin E. coli-related hemolytic uremic
syndrome (STEC-HUS). For the breakthrough medical innovation in
complement inhibition, Alexion and Soliris have received some of
the pharmaceutical industry's highest honors: the Prix Galien USA
(2008, Best Biotechnology Product) and France (2009, Rare Disease
Treatment).
More information on Soliris, including the full U.S. prescribing
information, is available at www.soliris.net.
Important Safety Information
The U.S. product label for Soliris includes a boxed warning:
“Life-threatening and fatal meningococcal infections have occurred
in patients treated with Soliris. Meningococcal infection may
become rapidly life-threatening or fatal if not recognized and
treated early [see Warnings and Precautions (5.1)]. Comply with the
most current Advisory Committee on Immunization Practices (ACIP)
recommendations for meningococcal vaccination in patients with
complement deficiencies. Immunize patients with a meningococcal
vaccine at least two weeks prior to administering the first dose of
Soliris, unless the risks of delaying Soliris therapy outweigh the
risk of developing a meningococcal infection. [See Warnings and
Precautions (5.1) for additional guidance on the management of the
risk of meningococcal infection]. Monitor patients for early signs
of meningococcal infections and evaluate immediately if infection
is suspected. Soliris is available only through a restricted
program under a Risk Evaluation and Mitigation Strategy (REMS).
Under the Soliris REMS, prescribers must enroll in the program [see
Warnings and Precautions (5.2)]. Enrollment in the Soliris REMS
program and additional information are available by telephone:
1-888-SOLIRIS (1-888-765-4747) or at www.solirisrems.com.”
In patients with PNH, the most frequently reported adverse
events observed with Soliris treatment in clinical studies were
headache, nasopharyngitis (runny nose), back pain and nausea.
Soliris treatment of patients with PNH should not alter
anticoagulant management because the effect of withdrawal of
anticoagulant therapy during Soliris treatment has not been
established. In patients with aHUS, the most frequently reported
adverse events observed with Soliris treatment in clinical studies
were headache, diarrhea, hypertension, upper respiratory infection,
abdominal pain, vomiting, nasopharyngitis, anemia, cough,
peripheral edema, nausea, urinary tract infections, and pyrexia.
Soliris is not indicated for the treatment of patients with
Shiga-toxin E. coli-related hemolytic uremic syndrome
(STEC-HUS). Please see full prescribing information for Soliris,
including BOXED WARNING regarding risk of serious meningococcal
infection.
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 the potential medical benefits of Soliris®
(eculizumab) for the treatment of myasthenia gravis, and Alexion's
future clinical, regulatory and commercial plans for Soliris for
the treatment of myasthenia gravis. 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, 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, 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. Suh J, Goldstein JM, Nowak RJ. Clinical characteristics of
refractory myasthenia gravis patients. Yale J Biol
Med. 2013; 86:255-60.
2. Gilhus NE, Verschuuren JJ. Myasthenia gravis: subgroup
classification and therapeutic strategies. Lancet
Neurol. 2015;14:1023-36.
3. Drachman D, Adams R, Hu R, Jones R, Brodsky R. Rebooting the
immune system with high-dose cyclophosphamide for treatment of
refractory myasthenia gravis. Ann NY Acad Sci.
2008;1132:305-314.
4. Sathasivam S. Diagnosis and management of myasthenia
gravis. Prog Neurol Psychiatry. 2014;18(1):6-14.
5. Howard JF, ed. Myasthenia Gravis: A Manual for the
Health Care Provider. St. Paul, MN: Myasthenia Gravis Foundation of
America, Inc.; 2008.
6. Safety and efficacy of eculizumab in refractory generalized
myasthenia gravis (REGAIN study). Clinicaltrials.gov identifier
NCT01997229.
7. Melzer N, Ruck T, Fuhr P, et al. Clinical features,
pathogenesis, and treatment of myasthenia gravis: a supplement to
the Guidelines of the German Neurological Society. J
Neurol. 2016 Feb 17. [Epub ahead of print]
8. Targeting the Complement System in Refractory Myasthenia
Gravis. Supplement to Neurology Reviews. February 2016.
View source
version on businesswire.com: http://www.businesswire.com/news/home/20170109005263/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
Alexion Pharmaceuticals (NASDAQ:ALXN)
Historical Stock Chart
From Aug 2024 to Sep 2024
Alexion Pharmaceuticals (NASDAQ:ALXN)
Historical Stock Chart
From Sep 2023 to Sep 2024