– ULTOMIRIS is the first and only long-acting
C5 inhibitor for aHUS, reducing the treatment burden for adults and
children with administration every other month –
– ULTOMIRIS has the potential to become the new
standard of care in Japan for the treatment of aHUS, an ultra-rare
disease which may progressively damage the kidney and other organs
–
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
Japan’s Ministry of Health, Labour and Welfare (MHLW) approved
ULTOMIRIS® (ravulizumab) for adults and children living with
atypical hemolytic uremic syndrome (aHUS). ULTOMIRIS is the first
and only long-acting C5 inhibitor for aHUS and is administered
every other month for adults and children (20 kg or more) and
monthly for children (<20 kg). Atypical HUS is an ultra-rare
disease that can cause progressive injury to vital organs,
primarily the kidneys, via damage to the walls of blood vessels and
blood clots.
“The goal of aHUS treatment is to prevent the body from
attacking its own immune system through the inhibition of
uncontrolled C5 complement activation,” said Prof. Shoichi
Maruyama, Director, Department of Nephrology, Nagoya University
Hospital. “Importantly, ULTOMIRIS demonstrated good control, while
also offering more time between infusions, which provides a
relevant difference to patients and providers.”
Atypical HUS affects both adults and children and many patients
present in critical condition in the hospital setting, often
requiring supportive care, including dialysis, in an intensive care
unit. The prognosis of aHUS can be poor in many cases, with 56
percent of adults and 29 percent of children developing end-stage
renal disease or dying within a year of diagnosis with supportive
care alone, so a timely and accurate diagnosis in addition to
treatment, is critical to improving patient outcomes.
“Today’s approval marks another important step in our efforts to
continue innovating for patients and improving their treatment
experience,” said John Orloff, M.D., Executive Vice President and
Head of Research & Development at Alexion. “ULTOMIRIS’ extended
dosing interval offers patients more flexibility and time to focus
on living their lives beyond their disease while also reducing the
burden on healthcare systems, hospitals and providers, which are
all under a tremendous amount of stress in the current
environment.”
The approval is based on data from two ongoing, global,
single-arm open-label studies of ULTOMIRIS – one in adults and one
in children, referred to as pediatrics in the study. A total of 18
out of 21 complement inhibitor treatment-naïve children and 56 out
of 58 complement inhibitor treatment-naïve adults were enrolled and
included in the interim analysis. Efficacy evaluation of Complete
TMA Response was defined by normalization of hematologic parameters
(platelet count and LDH) and improved kidney function (as measured
by ≥25 percent improvement in serum creatinine from baseline). In
the initial 26-week treatment periods, 54 percent of adults and
77.8 percent (interim data) of children demonstrated Complete TMA
Response. Treatment with ULTOMIRIS resulted in normalization of
platelet count in 84 percent of adults and 94 percent of children,
normalization of LDH (marker of hemolysis) in 77 percent of adults
and 90 percent of children, and improved kidney function in 59
percent of adults and 83 percent (interim data) of children (for
patients on dialysis at enrollment, baseline was established after
they had come off dialysis). In the 52-week follow-up period, 4
additional adult patients and 3 pediatric patients had a Complete
TMA Response that was confirmed after the 26-week Initial
Evaluation Period resulting in an overall Complete TMA Response of
61 percent in adults and 94 percent in children (interim data). A
second cohort of 10 pediatric patients who were SOLIRIS-experienced
were included in the pediatric study, demonstrating that switching
to ULTOMIRIS maintained disease control as evidenced by stable
hematologic and renal parameters, with no apparent impact on
safety.
The most frequently observed adverse reactions reported in these
studies were upper respiratory tract infection, diarrhea, nausea,
fatigue, headache, nasopharyngitis, and pyrexia. Serious
meningococcal infections have occurred in patients treated with
ULTOMIRIS, however in aHUS studies, no meningococcal infections
occurred in the 89 patients receiving treatment with ULTOMIRIS. To
minimize the risk for patients, specific risk-mitigation plans,
including a Risk Management Plan, have been established for
ULTOMIRIS.
ULTOMIRIS will be available at approval for the treatment of
aHUS.
About Atypical Hemolytic Uremic Syndrome (aHUS)
aHUS is an ultra-rare disease that can cause progressive injury
to vital organs, primarily the kidneys, via damage to the walls of
blood vessels and blood clots. aHUS occurs when the complement
system—a part of the body’s immune system—over-responds, leading
the body to attack its own healthy cells. aHUS can cause sudden
organ failure or a slow loss of function over time—potentially
resulting in the need for a transplant, and in some cases, death.
aHUS affects both adults and children, and many patients present in
critical condition, often requiring supportive care, including
dialysis, in an intensive care unit. The prognosis of aHUS can be
poor in many cases, so a timely and accurate diagnosis—in addition
to treatment—is critical to improving patient outcomes. Available
tests can help distinguish aHUS from other hemolytic diseases with
similar symptoms.
About ULTOMIRIS®
ULTOMIRIS® (ravulizumab-cwvz) is the first and only long-acting
C5 complement inhibitor. The medication works by inhibiting the C5
protein in the terminal complement cascade, a part of the body’s
immune system. When activated in an uncontrolled manner, the
complement cascade over-responds, leading the body to attack its
own healthy cells. ULTOMIRIS is administered intravenously every
eight weeks or every four weeks for pediatric patients less than 20
kg, following a loading dose. ULTOMIRIS is approved in Japan as a
treatment for adults with paroxysmal nocturnal hemoglobinuria
(PNH). To learn more about the regulatory status of ULTOMIRIS in
the countries that we serve, please visit www.alexion.com.
About Alexion
Alexion is a global biopharmaceutical company focused on serving
patients and families affected by rare diseases and devastating
conditions through the discovery, development and commercialization
of life-changing medicines. As a leader in rare diseases for more
than 25 years, Alexion has developed and commercializes two
approved complement inhibitors to treat patients with paroxysmal
nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic
syndrome (aHUS), as well as the first and only approved complement
inhibitor to treat anti-acetylcholine receptor (AchR)
antibody-positive generalized myasthenia gravis (gMG) and
neuromyelitis optica spectrum disorder (NMOSD). Alexion also has
two highly innovative enzyme replacement therapies for patients
with life-threatening and ultra-rare metabolic disorders,
hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D)
as well as the first and only approved Factor Xa inhibitor reversal
agent. In addition, the company is developing several
mid-to-late-stage therapies, including a copper-binding agent for
Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for
rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D
inhibitor as well as several early-stage therapies, including one
for light chain (AL) amyloidosis, a second oral Factor D inhibitor
and a third complement inhibitor. Alexion focuses its research
efforts on novel molecules and targets in the complement cascade
and its development efforts on the core therapeutic areas of
hematology, nephrology, neurology, metabolic disorders and
cardiology. Headquartered in Boston, Massachusetts, Alexion has
offices around the globe and serves patients in more than 50
countries. This press release and further information about Alexion
can be found at: www.alexion.com.
[ALXN-P]
For patient or advocacy inquiries please contact
patientadvocacy@alexion.com.
Forward-Looking Statement
This press release contains forward-looking statements that
involve risks and uncertainties relating to future events and the
future performance of Alexion, including statements related to: the
impact to Alexion of ULTOMIRIS approval in Japan for adults and
children living with atypical hemolytic uremic syndrome (aHUS);
that ULTOMIRIS provides effective disease control and together with
its dosing, makes a difference to patients; that ULTOMIRIS has the
potential to become the new standard of care in Japan for the
treatment of aHUS; timely and accurate diagnosis of aHUS patients–
in addition to treatment – is critical to improving patient
outcomes; the approval of ULTOMIRIS in Japan for aHUS marks another
important step in our efforts to continue innovating for patients
and improving their treatment experience; and ULTOMIRIS’ extended
eight-week dosing interval offers patients more flexibility and
time to focus on living their lives beyond their disease while also
reducing the burden on healthcare systems, hospitals and providers.
Forward-looking statements are subject to factors that may cause
Alexion's results and plans to differ materially from those
expected by these forward looking statements, including for
example: the anticipated benefits of ULTOMIRIS for aHUS patients
may not be realized (and the results of the clinical trials may not
be indicative of the results in Japan); results of clinical trials
may not be sufficient to satisfy any other regulatory authority in
order to approve ULTOMIRIS as a treatment for aHUS (or they may
request additional trials or additional information); results in
clinical trials may not be indicative of results from later stage
or larger clinical trials (or in broader patient populations once
the product is approved for use by regulatory agencies); the
possibility that results of clinical trials are not predictive of
safety and efficacy and potency of our products (or we fail to
adequately operate or manage our clinical trials) which could cause
us to discontinue sales of the product (or halt trials, delay or
prevent us from making regulatory approval filings or result in
denial of approval of our product candidates); unexpected delays in
clinical trials; unexpected concerns regarding products and product
candidates that may arise from additional data or analysis obtained
during clinical trials or obtained once used by patients following
product approval; future product improvements may not be realized
due to expense or feasibility or other factors; delays (expected or
unexpected) in the time it takes regulatory agencies to review and
make determinations on applications for the marketing approval of
our products; inability to timely submit (or failure to submit)
future applications for regulatory approval for our products and
product candidates; inability to timely initiate (or failure to
initiate) and complete future clinical trials due to safety issues,
IRB decisions, CMC-related issues, expense or unfavorable results
from earlier trials (among other reasons); our dependence on sales
from our principal product (Soliris); future competition from
biosimilars and novel products; decisions of regulatory authorities
regarding the adequacy of our research, marketing approval or
material limitations on the marketing of our products; delays or
failure of product candidates to obtain regulatory approval; delays
or the inability to launch product candidates due to regulatory
restrictions, anticipated expense or other matters; interruptions
or failures in the manufacture and supply of our products and our
product candidates; failure to satisfactorily address matters
raised by regulatory agencies regarding products and product
candidates; uncertainty of long-term success in developing,
licensing or acquiring other product candidates or additional
indications for existing products; inability to complete
acquisitions or grow the product pipeline through acquisitions
(including due to failure to obtain antitrust approvals); the
possibility that current rates of adoption of our products are not
sustained; the adequacy of our pharmacovigilance and drug safety
reporting processes; failure to protect and enforce our data,
intellectual property and proprietary rights and the risks and
uncertainties relating to intellectual property claims, lawsuits
and challenges against us; 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; failure to realize the benefits and potential of investments,
collaborations, licenses and acquisitions; the possibility that
expected tax benefits will not be realized; potential declines in
sovereign credit ratings or sovereign defaults in countries where
we sell our products; delay of collection or reduction in
reimbursement due to adverse economic conditions or changes in
government and private insurer regulations and approaches to
reimbursement; adverse impacts on our supply chain, clinical
trials, manufacturing operations, financial results, liquidity,
hospitals, pharmacies and health care systems from natural
disasters and global pandemics, including the coronavirus;
uncertainties surrounding legal proceedings, company investigations
and government investigations; the risk that estimates regarding
the number of patients with PNH, aHUS, gMG, NMOSD, HPP and LAL-D
and other indications we are pursuing are inaccurate; the risks of
changing foreign exchange rates; risks relating to the potential
effects of the Company's restructuring; risks related to the
acquisition of Achillion, Portola and other companies and
co-development efforts; and a variety of other risks set forth from
time to time in Alexion's filings with the SEC, including but not
limited to the risks discussed in Alexion's Quarterly Report on
Form 10-Q for the period ended June 30, 2020 and in our other
filings with the SEC. Alexion disclaims any obligation to update
any of these forward-looking statements to reflect events or
circumstances after the date hereof, except when a duty arises
under law.
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version on businesswire.com: https://www.businesswire.com/news/home/20200925005244/en/
Alexion: Media Megan Goulart, 857-338-8634 Executive
Director, Corporate Communications
Investors Chris Stevo, 857-338-9309 Head of Investor
Relations
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