- Final Evaluation Determination Confirms that Soliris is a “Very
Effective Treatment for aHUS with Significant Value to Patients” -
- Final Evaluation Determination Includes the Same Conditions
Discussed in Draft Recommendation, Including Establishment of an Expert
Center and a Robust System to Monitor Patients Receiving Treatment -
Company Website:
http://www.alexionpharma.com
CHESHIRE, Conn. -- (Business Wire)
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that the
National Institute for Health and Care Excellence (NICE) Highly
Specialised Technologies Evaluation Committee (EC) has recommended that
Soliris® (eculizumab) be commissioned for all patients in
England suffering from atypical hemolytic uremic syndrome (aHUS), a
severe and life-threatening ultra-rare disorder. With this final
evaluation determination, NICE has recommended Soliris for use within
the National Health Service (NHS) in England as the first and only
treatment for patients with aHUS.
“Today’s decision is an important victory for patients with aHUS and
physicians who now have assurance that they will have access to the
life-transforming efficacy of Soliris. We are pleased that Soliris will
be made available on the NHS for patients with aHUS and we commend NHS
England for its previous decision to provide interim funding to patients
for the extended period it took for NICE to make this final
determination,” said Leonard Bell, M.D., Chairman and Chief Executive
Officer of Alexion.
In the final evaluation determination published today, the NICE EC again
confirmed that Soliris represents an important treatment option of
significant value to patients with aHUS. The Committee recommended the
commissioning of Soliris, within its marketing authorization, for aHUS
subject to the conditions provided in the evaluation consultation
document released in September. Soliris was approved in November 2011 by
the European Commission for the treatment of patients with aHUS. The
Clinical Particulars section of the EU label states that “Soliris
treatment is recommended to continue for the patient's lifetime, unless
the discontinuation of Soliris is clinically indicated," as described in
the Special warnings and precautions for use subsection.1 As
noted by NICE in its final evaluation determination, after stopping
Soliris, severe TMA complications were reported including graft failure
needing hemodialysis, renal insufficiency, end-stage renal failure and
respiratory distress needing intubation.
“Patients with aHUS are at constant risk of sudden, progressive and
life-threatening damage to vital organs including the kidney and other
organs,” said Keith Woods, Vice President and General Manager of Alexion
Pharma UK. “Alexion supports the use of Soliris consistent with the
EMA-approved label and firmly believes that decisions regarding
continuation of Soliris should be made by the treating physician based
on best clinical judgment.”
NICE recommended that the following arrangements be in place as
conditions for the funding of Soliris for patients with aHUS:
-
Coordination of the use of eculizumab through an expert center;
-
Monitoring systems to record the number of people with a diagnosis of
atypical hemolytic uremic syndrome, the number of people who receive
eculizumab, and the dose and duration of treatment for these people;
-
A national protocol for starting and stopping eculizumab for clinical
reasons;
-
And a research programme with robust methods to evaluate when stopping
treatment or dose adjustment might occur.
About aHUS
aHUS is a chronic, ultra-rare, and life-threatening disease in which a
life-long and permanent genetic deficiency in one or more complement
regulatory genes causes chronic uncontrolled complement activation,
resulting in complement-mediated thrombotic microangiopathy (TMA), the
formation of blood clots in small blood vessels throughout the body.2,3
Permanent, uncontrolled complement activation in aHUS causes a life-long
risk for TMA, which leads to sudden, catastrophic, and life-threatening
damage to the kidney, brain, heart, and other vital organs, and
premature death.2,4 Seventy-nine percent of all patients with
aHUS die, require kidney dialysis or have permanent kidney damage within
three years after diagnosis despite plasma exchange or plasma infusion
(PE/PI).5 Moreover, 33 to 40 percent of patients die or
progress to end-stage renal disease with the first clinical
manifestation of aHUS despite PE/PI.5,6 The majority of
patients with aHUS who receive a kidney transplant commonly experience
subsequent systemic TMA, resulting in a 90 percent transplant failure
rate in these TMA patients.7
aHUS affects both children and adults. Complement-mediated TMA also
causes reduction in platelet count (thrombocytopenia) and red blood cell
destruction (hemolysis). While mutations have been identified in at
least ten different complement regulatory genes, mutations are not
identified in 30-50 percent of patients with a confirmed diagnosis of
aHUS.5
About Soliris®
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), the
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 TMA. aHUS is a debilitating,
ultra-rare and life-threatening genetic disorder characterized by
complement-mediated thrombotic microangiopathy, or TMA (blood clots in
small vessels). 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 including the full U.S. prescribing information on
Soliris 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)."
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 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 nearly 40 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, including asfotase alfa,
across multiple therapeutic areas. This press release and further
information about Alexion can be found at: www.alexionpharma.com.
[ALXN-G]
Safe Harbor Statement
This news release contains forward-looking statements, including
statements related to potential health and medical benefits of Soliris®
(eculizumab) for the treatment of patients with aHUS and PNH, pricing
for Soliris in England,and the continuation of existing programs
in England that provide access to Soliris. 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 reimbursement of Soliris, 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 Annual Report on Form 10-Q for the period
ended September 30, 2014. 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 Soliris eMPC. Available at http://www.medicines.org.uk/emc/medicine/19966.
Last downloaded on 20 Oct 2014.
2 Benz K, Amann K. Thrombotic microangiopathy: new insights. Curr
Opin Nephrol Hypertens. 2010;19(3):242-7
3 Ariceta G, Besbas N, Johnson S, et al. Guideline for the
investigation and initial therapy of diarrhea-negative hemolytic uremic
syndrome. Pediatr Nephrol. 2009;24:687-96.
4 Tsai HM. The molecular biology of thrombotic
microangiopathy. Kidney Int. 2006;70(1):16-23
5 Noris M, Remuzzi G. Atypical hemolytic-uremic syndrome. N
Engl J Med. 2009;361:1676-87.
6 Noris M, Caprioli J, Bresin E, et al. Relative role of
genetic complement abnormalities in sporadic and familial aHUS and their
impact on clinical phenotype. Clin J Am Soc Nephrol.
2010;5:1844-59
7 Bresin E, Daina E, Noris M, et al. Outcome of renal
transplantation in patients with non-Shiga toxin-associated hemolytic
uremic syndrome: prognostic significance of genetic background. Clin
J Am Soc Nephrol. 2006;1:88-99.
Contacts:
Media:
Irving Adler, 203-271-8210
Executive
Director, Corporate Communications
or
Kim Diamond, 203-439-9600
Senior
Director, Corporate Communications
or
Investors:
Elena
Ridloff, CFA, 203-699-7722
Executive Director, Investor Relations
Source: Alexion Pharmaceuticals, Inc.
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