Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) announced today that the
National Institute for Health and Clinical Excellence (NICE) Highly
Specialised Technologies Evaluation Committee (EC) has reaffirmed the
very significant clinical value of eculizumab (Soliris®) for
the treatment of atypical hemolytic uremic syndrome (aHUS) and the lack
of other effective therapies, and has issued a recommendation that it be
nationally commissioned for all patients suffering from this severe and
life-threatening genetic disorder.
In the second Evaluation Consultation Document (ECD) released today, the
NICE EC again confirmed the 2013 Advisory Group for National Specialised
Services (AGNSS) positive assessment as well as the NICE assessment from
earlier this year that eculizumab is a very effective treatment for aHUS
patients and produces substantial quality-adjusted life year gains of a
magnitude rarely seen for any new drug treatment. The Committee
recommended the commissioning of eculizumab for aHUS patients subject to
the following conditions:
-
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.
“We are pleased that NICE has once again confirmed that patients with
aHUS are at constant risk of sudden, progressive and life-threatening
damage to vital organs including the kidney and other organs, and that
eculizumab is a significant breakthrough for patients with this
devastating disorder,” said Leonard Bell, M.D., Chief Executive Officer
of Alexion. “Alexion supports the use of eculizumab consistent with the
EMA-approved label, which specifically directs that treatment is
recommended to continue for the patient’s lifetime, unless the
discontinuation of eculizumab is clinically indicated. We believe that
it is important that NICE work within its remit and that decisions
regarding continuation of eculizumab should be made by the treating
physician based on best clinical judgment. We will provide specific
comments to NICE to address its conditions and look forward to working
to ensure equity such that patients with aHUS in England, like patients
with PNH in England, have equal and sustained access to this
life-transforming therapy.”
Currently, new and existing patients with aHUS in England are able to
receive eculizumab through an interim policy commissioned by NHS England
last year, and NICE confirmed today that this interim policy will remain
in place pending the final outcome of NICE’s appraisal. Alexion looks
forward to confirmation by NICE of a final policy, which is expected to
follow a public EC meeting scheduled on October 9, 2014.
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.1,2
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.1,3 Sixty-five percent of all patients with
aHUS die, require kidney dialysis or have permanent kidney damage within
the first year after diagnosis despite plasma exchange or plasma
infusion (PE/PI).4,5 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.6
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.7
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), a debilitating,
ultra-rare and life-threatening blood disorder, characterized by
complement-mediated hemolysis (destruction of red blood cells). Soliris
is indicated to reduce hemolysis. 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), a debilitating, ultra-rare and life-threatening genetic
disorder characterized by complement-mediated thrombotic
microangiopathy, or TMA (blood clots in small vessels). Soliris is
indicated to inhibit 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 innovation in
complement inhibition, Alexion and Soliris have received the
pharmaceutical industry's highest honors: the 2008 Prix Galien USA Award
for Best Biotechnology Product with broad implications for future
biomedical research and the 2009 Prix Galien France Award in the
category of Drugs for Rare Diseases.
More information including the full U.S. prescribing information on
Soliris is available at: http://soliris.net/sites/default/files/assets/soliris_pi.pdf.
The full prescribing information on Soliris in Europe is available at: http://www.ema.europa.eu/docs/en_GB/document_library/EPAR_Product_Information/human/000791/WC500054208.pdf.
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, 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, whether eculizumab will be
nationally commissioned for aHUS in England and the timing of such
commissioning, 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 June 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) Benz K, Amann K. Thrombotic microangiopathy: new insights. Curr Opin
Nephrol Hypertens. 2010;19(3):242-7.
(2) 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.
(3) Tsai HM. The molecular biology of thrombotic microangiopathy. Kidney
Int. 2006;70(1):16-23.
(4) Caprioli J, Noris M, Brioschi S, et al. The impact of MCP, CFH, and
IF mutations on clinical presentation, response to treatment, and
outcome. Blood. 2006;108:1267-9.
(5) Loirat C, Garnier A, Sellier-Leclerc AL, Kwon T. Plasmatherapy in
atypical hemolytic uremic syndrome. Semin Thromb Hemost. 2010;36:673-81.
(6) 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.
(7) 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.
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