NHS England Commissions Soliris® for the Treatment of Atypical Haemolytic Uraemic Syndrome (aHUS)

  NHS England Commissions Soliris® for the Treatment of Atypical Haemolytic
                           Uraemic Syndrome (aHUS)

  PR Newswire

  WEYBRIDGE, England, September 12, 2013

WEYBRIDGE, England, September 12, 2013 /PRNewswire/ --

  Commissioning decision follows positive Clinical Priorities Advisory Group
(CPAG) recommendation and will enable initial national funding of Soliris for
                   children and adults with aHUS in England

  CPAG positive recommendation reflects 2 ^nd  independent body to issue a
 positive recommendation to fund Soliris for children and adults with aHUS in
                                   England

NHS England decision provides initial national patient a cc ess to Soliris as
 Ministers of Health have placed another hurdle to patient care with referral
    to the 3 ^rd  independent body asked to review the only approved aHUS
   treatment; the National Institute for Health and Care Excellence (NICE)
                         decision anticipated in 2014

Alexion Pharma UK, a subsidiary of Alexion Pharmaceuticals, Inc. (Nasdaq:
ALXN), today announced that Soliris ^® (eculizumab) is now commissioned by NHS
England for the treatment of children and adults with atypical haemolytic
uraemic syndrome (aHUS). The Department of Health has stated that this
policy, which follows a very similar and positive recommendation from both
the Advisory Group for National Specialized Services (AGNSS) and the Clinical
Priorities Advisory Group (CPAG) in 2013, will be an initial national policy
providing patient access to Soliris, pending evaluation and a final decision
by what would be the 3 ^rd independent body - the National Institute for
Health and Care Excellence (NICE) - which is currently anticipated in spring
2014.

Soliris was referred to NICE by Ministers of Health as the first topic for
evaluation in its Highly Specialised Technologies Programme. In the interim,
and according to NHS England , given the serious nature of the disease, NHS
England will fund eculizumab for the treatment of new patients with aHUS
(defined as those with a functioning kidney) and for existing patients who are
on dialysis and are suitable for a kidney transplant. A commissioning for
evaluation scheme will be developed for patients who are not suitable for
transplant. NHS England will not routinely commission eculizumab for patients
currently diagnosed with aHUS who have not received approval for eculizumab
from an existing commissioning body. ^[ ^1 ^]

Alexion looks forward to the completion and conclusion of the NICE evaluation,
which is expected in spring 2014, so that patients with aHUS in England may
continue to receive treatment.

aHUS is an ultra-rare, hereditary condition that is severe, life-threatening
and chronic, and progressively damages vital organs, often leading to sudden
and catastrophic damage to the kidney, brain, heart, and other vital organs,
and premature death. ^[ ^2 ^] ^, ^[ ^3 ^] ^, ^[ ^4 ^] It occurs when small
blood clots form in blood vessels throughout the body. ^[ ^2 ^] ^, ^[ ^3 ^]
aHUS affects both adults and children and often presents at a very young age.
^[ ^5 ^] Sixty-five percent of all patients with aHUS require kidney dialysis,
have permanent kidney damage or die within the first year after diagnosis. ^[
^6 ^] ^, ^[ ^7 ^]

"aHUS is a disease that is both ultra-rare and life-threatening. As such, aHUS
affects very few people but is extremely devastating for those patients and
their families. We are very pleased that NHS England has approved initial
national funding and patient access to the only approved treatment of aHUS in
England, and look forward to confirmation by NICE of a final policy," said
Mark Barrett, Country Manager, Alexion Pharma UK.

Soliris was approved in the European Union for the treatment of adult and
paediatric patients with aHUS in November 2011.

References:

1.NHS England (2013). Clinical commissioning policy statement: Eculizumab
    for atypical haemolytic uraemic syndrome. Available at
    http://bit.ly/17W9vTc . Last accessed 11 September 2013.
2.Noris M, Remuzzi G (2009). Atypical hemolytic-uremic syndrome. N Engl J
    Med 361:1676-87
3.Benz K, Amann K (2010). Thrombotic microangiopathy: new insights. Curr
    Opin Nephrol Hypertens ;19(3):242-7
4.Tsai HM (2006). The molecular biology of thrombotic microangiopathy.
    Kidney Int ;70(1):16-23
5.Bresin E, Daina E, Noris M, et al (2006) International Registry of
    Recurrent and Familial HUS/TTP. Outcome of renal transplantation in
    patients with non-Shiga toxin-associated hemolytic uremic syndrome:
    prognostic significance of genetic background. Clin J Am Soc Nephrol.
    1:88-99
6.Caprioli J, Noris M, Brioschi S, et al for the International Registry of
    Recurrent and Familial HUS/TTP (2006). Genetics of HUS: the impact of MCP,
    CFH, and IF mutations on clinical presentation, response to treatment, and
    outcome. Blood. 108:1267-1279
7.Loirat C, Garnier A, Sellier-Leclerc AL et el (2010). Plasmatherpay in
    atypical hemolytic uremic syndrome., Semin Thromb Hemost . 2010;36:673-681
8.Abstract 1587 entitled "A phase II study of eculizumab in patients with
    atypical hemolytic uremic syndrome receiving chronic plasma
    exchange/infusion," presented by Dr. Chantal Loirat at the 16th Congress
    of the European Hematology Association, Sunday, June 12, 2011.
9.Abstract 1588 entitled "Eculizumab efficacy and safety in patients with
    atypical hemolytic uremic syndrome resistant to plasma exchange/infusion,"
    presented by Dr. Chantal Loirat at the 16th Congress of the European
    Hematology Association, Sunday, June 12, 2011.
10.(13) Abstract 396 entitled "Eculizumab therapy for atypical hemolytic
    uremic syndrome in pediatric patients: Efficacy and safety outcomes from a
    retrospective study," presented by Dr. Giacomo Simonetti at the 16th
    Congress of the European Hematology Association, Friday, June 10, 2011.
11.Noris M, Caprioli J, Bresin E, et al (2010). Relative role of genetic
    complement abnormalities in sporadic and familial aHUS and their impact on
    clinical phenotype. Clin J Am Soc Nephrol. 5:1844-1859

Notes to Editors:

About aHUS

aHUS is a chronic, ultra-rare, and life-threatening disease in which a 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. ^[ ^3 ^] ^, ^[ ^4 ^] 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). ^[ ^6 ^] ^, ^[ ^7 ^] The majority of
patients with aHUS who receive a kidney transplant commonly experience
subsequent systemic TMA, resulting in a 90% transplant failure rate. ^[ ^5 ^]

aHUS affects both children and adults. In a large group of aHUS patients, 60%
were first diagnosed at younger than 18 years of age. ^[5] Complement-mediated
TMA also causes reduction in platelet count (thrombocytopenia) and red blood
cell destruction (haemolysis). While mutations have been identified in at
least ten different complement regulatory genes, mutations are not identified
in 30-50% of patients with a confirmed diagnosis of aHUS. ^[ ^11 ^]

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 US, European Union, Japan 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 also approved in the US, the European Union and Canada as the first
and only treatment for patients with atypical Hemolytic Uremic Syndrome (aHUS)
to inhibit complement-mediated thrombotic microangiopathy, a debilitating,
ultra-rare and life-threatening genetic disorder characterized by
complement-mediated thrombotic microangiopathy (blood clots in small vessels).
The effectiveness of Soliris in aHUS is based on the effects on thrombotic
microangiopathy (TMA) and renal function. Soliris is not indicated for the
treatment of patients with Shiga toxin E. coli related hemolytic uremic
syndrome (STEC-HUS).

More information including the full prescribing information on Soliris is
available at:

http://www.medicines.org.uk/emc/searchresults.aspx?term=Soliris&searchtype=QuickSearch

Important Safety Information

In Europe, the Summary of Product Characteristics (SmPC) for Soliris includes
a special warning and precaution for use: Due to its mechanism of action, the
use of Soliris increases the patient's susceptibility to meningococcal
infection (Neisseria meningitidis). These patients might be at risk of disease
by uncommon serogroups (particularly Y, W135 and X), although meningococcal
disease due to any serogroup may occur. To reduce the risk of infection, all
patients must be vaccinated at least 2 weeks prior to receiving Soliris. aHUS
patients who are treated with Soliris less than 2 weeks after receiving a
meningococcal vaccine must receive treatment with appropriate prophylactic
antibiotics until 2 weeks after vaccination. Patients must be re-vaccinated
according to current medical guidelines for vaccination use. Tetravalent
vaccines against serotypes A, C, Y and W135 are strongly recommended,
preferably conjugated ones. Vaccination may not be sufficient to prevent
meningococcal infection. Consideration should be given to official guidance on
the appropriate use of antibacterial agents.

The most common or serious adverse reactions are headache (occurred mostly in
the initial phase), leukopenia and meningococcal infection. The most common
(>10%) adverse reactions reported in paediatric aHUS patients were diarrhoea,
vomiting, pyrexia, upper respiratory tract infection and headache. Soliris
treatment should not alter anticoagulant management. Please see Summary of
Product Characteristics for full prescribing information for Soliris,
including all special warnings and precautions.

About Alexion

Alexion Pharmaceuticals, Inc. is a biopharmaceutical company focused on the
development of treatments for severe and ultra-rare disorders through the
innovation, development and commercialisation of innovative therapeutic
products. Alexion is the global leader in complement inhibition.

Contact: For further information please contact: Mitali Rajan, External
Communications Alexion Services Europe Tel: +32-(0)-476-941-840 E:
rajanm@alxn.com
 
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