FDA Grants Orphan Drug Designation to Soliris® (eculizumab) for Prevention of Delayed Graft Function (DGF) in Renal Transplant

  FDA Grants Orphan Drug Designation to Soliris® (eculizumab) for Prevention
  of Delayed Graft Function (DGF) in Renal Transplant Patients

Business Wire

CHESHIRE, Conn. -- January 21, 2014

Alexion Pharmaceuticals (Nasdaq:ALXN) today announced that the U.S. Food and
Drug Administration (FDA) has granted an orphan drug designation (ODD) to
Soliris^® (eculizumab), a first-in-class terminal complement inhibitor, for
the prevention of delayed graft function (DGF) in renal transplant patients.
DGF is an early and serious complication of organ transplantation that is
characterized by the failure of a transplanted organ to function normally
immediately following transplantation. In patients undergoing a kidney
transplant, DGF leads to the patient requiring dialysis in order to survive. ^
1-3

Soliris is currently approved for the treatment of patients with paroxysmal
nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS),
two debilitating, ultra-rare and life-threatening disorders caused by chronic
uncontrolled complement activation. Soliris is not approved in any country to
prevent or treat DGF following kidney or other solid organ transplantation.

“For kidney transplant patients with increased risk, there is a serious unmet
medical need for a treatment to prevent delayed graft function and its harmful
consequences,” said Martin Mackay, Ph.D., Executive Vice President, Global
Head of R&D at Alexion. “By specifically inhibiting the terminal complement
pathway, which is believed to play a critical role in the development of DGF,
Soliris has the potential to lower the risk of DGF, a benefit that may have
positive implications for longer-term kidney function and clinical outcomes
for patients. In addition, a significant number of donor kidneys are
reportedly never used and thus discarded each year due to the risk of poor
outcomes associated with DGF, therefore reducing the risk of DGF may enable
more patients to receive a kidney transplant.”

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 U.S. Orphan drug
designation 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.

Alexion plans to initiate a single multinational DGF registration study with
Soliris later this year. Alexion looks forward to working closely with the FDA
to gather the clinical evidence needed to support approval for this
indication.

About Delayed Graft Function (DGF)

DGF is an early and serious complication of organ transplantation that is
characterized by the failure of a transplanted organ to function normally
immediately following transplantation. In the case of DGF in the setting of
kidney transplantation, the patient requires dialysis after the
transplantation procedure.^1-3 Most often, DGF results from organ injury
caused by reduction and/or restoration of blood flow, and the associated
inflammation, including complement activation.^1-4 DGF has a substantial
negative impact on graft function both in the short and long term, which can
result in premature graft loss, prolonged hospitalization or patient
death.^5,6 In addition, as kidney donors are in short supply, reducing the
risk of DGF may allow more donor kidneys to be transplanted. At present, 15-20
percent of donor kidneys are reportedly never used and thus discarded each
year in the U.S. and Europe due to the risk of poor outcomes associated with
DGF,^7,8 denying many patients the benefit of solid organ transplantation.

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), 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. The
effectiveness of Soliris in aHUS is based on its effects on TMA and renal
function. Prospective clinical trials in additional patients, the preliminary
results of which were reported at international nephrology and hematology
conferences in 2013, are ongoing to confirm the benefit of Soliris in patients
with aHUS. 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 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. 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 Serious Meningococcal Infections
(5.1) for additional guidance on the management 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. 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 hypertension, upper respiratory
tract infection, diarrhea, headache, anemia, vomiting, nausea, urinary tract
infection, and leukopenia. 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 the United States, European Union, Japan and other
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 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 medical benefits of Soliris^® (eculizumab) for the
prevention of delayed graft function (DGF) in renal transplant patients.
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 Soliris for DGF, delays in arranging
satisfactory manufacturing capabilities and establishing commercial
infrastructure for Soliris for DGF, the possibility that results of clinical
trials are not predictive of safety and efficacy results of Soliris for DGF in
broader or different patient populations, the risk that third party payors
(including governmental agencies) will not reimburse for the use of Soliris
for DGF (if approved) at acceptable rates or at all, the risk that estimates
regarding the number of patients with Soliris for DGF and observations
regarding the natural history of patients with Soliris for DGF are inaccurate,
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 Quarterly Report on Form 10-Q for the period
ended Sept. 30, 2013. 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

     Jayaram D, Kommareddi M, Sung RS, Luan FL. Delayed graft function
1.  requiring more than one-time dialysis treatment is associated with
     inferior clinical outcomes. Clin Transplant. 2012.;26:E536-43.
     
2.   Siedlecki, A, Irish, W, and Brennan, DC (2011). Delayed graft function in
     the kidney transplant. Am. J. Transplant. 11, 2279-2296.
     
3.   Perico N, Cattaneo D, Sayegh MH, Remuzzi G. Delayed graft function in
     kidney transplantation. Lancet. 2004;364:1814-27.
     
4.   Yarlagadda,SG, Klein,CL, and Jani,A (2008). Long-term renal outcomes
     after delayed graft function. Adv. Chronic. Kidney Dis. 15, 248-256.
     
     Butala NM, Reese PP, Doshi MD, Parikh CR. Is delayed graft function
5.   causally associated with long-term outcomes after kidney transplantation?
     Instrumental variable analysis. Transplantation. 2013;95:1008-14.
     
     Yarlagadda SG, Coca SG, Formica RN Jr, Poggio ED, Parikh CR. Association
6.   between delayed graft function and allograft and patient survival: a
     systematic review and meta-analysis. Nephrol Dial Transplant.
     2009;24:1039-47.
     
     US Organ Procurement and Transplantation Network/Scientific Registry of
7.   Transplant Recipients. OPTN/SRTR Annual Report, 2009. Chapter II: Organ
     donation and utilization in the United States, 1999-2008.
     http://www.ustransplant.org/annual_reports/current/.
     
8.   Eurotransplant. Statistics Report Library.
     http://statistics.eurotransplant.org/. 2013.
     

Contact:

Alexion Pharmaceuticals, Inc.
Irving Adler, 203-271-8210
Exec. Director, Corporate Communications
or
Kim Diamond, 203-439-9600
Senior Director, Corporate Communications
or
Investors:
Rx Communications
Rhonda Chiger, 917-322-2569