- At 26 weeks, 54% of adults and 71% of
children treated with ULTOMIRIS demonstrated Complete Thrombotic
Microangiopathy (TMA) Response -
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
the U.S. Food and Drug Administration (FDA) approved
ULTOMIRIS® (ravulizumab-cwvz) for the treatment of atypical
hemolytic uremic syndrome (aHUS) to inhibit complement-mediated
thrombotic microangiopathy (TMA) for adult and pediatric (one month
of age and older) patients. This is the first pediatric approval
for ULTOMIRIS. 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. Atypical HUS
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.
“The primary approach to treatment is to prevent the body from
attacking itself, through the inhibition of uncontrolled complement
activation, referred to as C5 inhibition,” said Spero Cataland,
M.D., Professor of Clinical Internal Medicine, Wexner Medical
Center, The Ohio State University College of Medicine. “Clinical
study results showed adult and pediatric patients had complete C5
inhibition following the first dose of ULTOMIRIS. C5 inhibition was
sustained over time with only six or seven infusions a year in
adults—and that is important to consider for my patients.”
Atypical HUS 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.
“The consequences of uncontrolled complement activation, like
organ failure and potentially death, create significant challenges
and uncertainty for people and families facing aHUS,” said John
Orloff, M.D., Executive Vice President and Head of Research and
Development at Alexion. “Based on the Phase 3 data, which
demonstrated clinically meaningful benefits in people with aHUS, we
believe ULTOMIRIS has the potential to become the new standard of
care for this devastating disease.”
The FDA approval is based on data from two global, single-arm
open-label studies of ULTOMIRIS – one in adults and one in
children, referred to as pediatrics in the study – with aHUS. The
pediatric study is ongoing and a total of 14 out of 16 children
were enrolled and included in the interim analysis. Efficacy
evaluation of Complete TMA Response was defined by hematologic
normalization 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 71 percent (interim data) of
children demonstrated Complete TMA Response. Treatment with
ULTOMIRIS resulted in reduced thrombocytopenia (low blood platelet
count) in 84 percent of adults and 93 percent of children, reduced
hemolysis (the destruction of red blood cells) in 77 percent of
adults and 86 percent of children, and improved kidney function in
59 percent of adults and 79 percent (interim data) of children (for
patients on dialysis at enrollment, baseline was established after
they had come off dialysis).
The most frequently observed adverse reactions reported in these
studies were upper respiratory tract infection, diarrhea, nausea,
vomiting, headache, hypertension and pyrexia. Serious meningococcal
infections have occurred in patients treated with ULTOMIRIS. To
minimize the risk for patients, specific risk-mitigation plans,
including a REMS, have been established for ULTOMIRIS.
Regulatory filings for marketing authorizations of ULTOMIRIS for
the treatment of aHUS in the European Union (EU) and Japan are
under review with regulators.
About aHUS Atypical hemolytic uremic syndrome (aHUS) is
an ultra-rare disease that affects both children and adults and can
lead to potentially irreversible damage to kidneys and other vital
organs, sudden or progressive kidney failure (requiring dialysis or
transplant) and premature death. aHUS is characterized by
inflammation and the formation of blood clots in small blood
vessels throughout the body (thrombotic microangiopathy [TMA])
mediated by chronic, uncontrolled activation of the complement
system, which is part of the body’s immune system. TMA consists of
reduced platelet count (thrombocytopenia), hemolytic anemia (as a
result of hemolysis [destruction of red blood cells]) and acute
kidney injury (AKI). If left untreated, significant proportions of
adults (46 percent) and children (16 percent) can progress to
end-stage renal disease (ESRD) or die during first clinical
manifestations of aHUS despite supportive care, including plasma
exchange or plasma infusion (PE/PI). One year following clinical
manifestations, 56 percent of adults and 29 percent of children can
progress to ESRD or die, if left untreated. Early and careful
diagnosis of aHUS is critical, as many coexisting diseases and
events are known or suspected to activate the complement cascade,
and as patients may not necessarily present with the classic TMA
triad of thrombocytopenia, hemolytic anemia and renal impairment or
may have less severe renal involvement. Available tests can help
distinguish aHUS from other hemolytic diseases with similar
symptoms such as HUS caused by Shiga toxin-producing Escherichia
coli (STEC-HUS) and thrombotic thrombocytopenic purpura (TTP).
About ULTOMIRIS ULTOMIRIS (ravulizumab-cwvz) is the first
and only long-acting C5 complement inhibitor. It is administered
intravenously every eight weeks or every four weeks for pediatric
patients less than 20 kg, following a loading dose. ULTOMIRIS works
by inhibiting the C5 protein in the terminal complement cascade, a
part of the body’s immune system. The terminal complement cascade,
when activated in an uncontrolled manner, plays a role in severe
ultra-rare disorders. ULTOMIRIS is approved in the U.S., Japan, and
the EU as a treatment for adults with PNH and in the U.S. for aHUS
to inhibit complement-mediated thrombotic microangiopathy (TMA) in
adult and pediatric (one month of age and older) patients.
You can read more about the study results for this clinical
program on alexion.com.
INDICATIONS & IMPORTANT SAFETY INFORMATION FOR
ULTOMIRIS (ravulizumab-cwvz) 300 mg / 30 mL injection for
intravenous use
INDICATIONS ULTOMIRIS is a prescription medicine called a
monoclonal antibody. ULTOMIRIS is used to treat adults with a
disease called Paroxysmal Nocturnal Hemoglobinuria (PNH). ULTOMIRIS
is used to treat adults and children 1 month of age and older with
a disease called atypical Hemolytic Uremic Syndrome (aHUS).
ULTOMIRIS is not used in treating people with Shiga toxin E. coli
related hemolytic uremic syndrome (STEC-HUS). It is not known if
ULTOMIRIS is safe and effective in children with PNH. It is not
known if ULTOMIRIS is safe and effective in children younger than 1
month of age.
IMPORTANT SAFETY INFORMATION ULTOMIRIS is a medicine that
affects the immune system. ULTOMIRIS can lower the ability of the
immune system to fight infections. ULTOMIRIS increases the chance
of getting serious and life-threatening meningococcal infections.
Meningococcal infections may quickly become life-threatening and
cause death if not recognized and treated early.
Meningococcal vaccines must be received at least 2 weeks before
the first dose of ULTOMIRIS if one has not already had this
vaccine. If one’s doctor decided that urgent treatment with
ULTOMIRIS is needed, meningococcal vaccination should be
administered as soon as possible. If one has not been vaccinated
and ULTOMIRIS therapy must be initiated immediately, 2 weeks of
antibiotics should also be administered with the vaccinations. If
one had a meningococcal vaccine in the past, additional vaccination
might be needed before starting ULTOMIRIS. One’s doctor will decide
if additional meningococcal vaccination is needed. Meningococcal
vaccines reduce the risk of meningococcal infection but do not
prevent all meningococcal infections. Call one’s doctor or get
emergency medical care right away if any of these signs and
symptoms of a meningococcal infection occur: headache with nausea
or vomiting, headache and fever, headache with a stiff neck or
stiff back, fever, fever and a rash, confusion, muscle aches with
flu-like symptoms, and eyes sensitive to light. One’s doctor will
give a Patient Safety Card about the risk of meningococcal
infection. Carry the card at all times during treatment and for 8
months after your the ULTOMIRIS dose.
ULTOMIRIS is only available through a program called the
ULTOMIRIS REMS.
ULTOMIRIS may also increase the risk of other types of serious
infections. People who take ULTOMIRIS may have an increased risk of
getting infections caused by Streptococcus pneumoniae and
Haemophilus influenzae. Certain people may also have an increased
risk of gonorrhea infection. To find out if one is at risk for
gonorrhea infection, about gonorrhea prevention, and regular
testing, talk to the doctor. Call the doctor right away if one has
any new signs or symptoms of infection.
Do not receive ULTOMIRIS if one has a meningococcal infection,
or has not been vaccinated against meningococcal infection unless
the doctor decides that urgent treatment with ULTOMIRIS is
needed.
Before one receives ULTOMIRIS, tell the doctor about all of the
medical conditions, including if one: has an infection or fever,
are pregnant or plan to become pregnant, and are breastfeeding or
plan to breastfeed. It is not known if ULTOMIRIS will harm an
unborn baby. It is not known if ULTOMIRIS passes into the breast
milk. One should not breastfeed during treatment and for 8 months
after one’s final dose of ULTOMIRIS.
Tell the doctor about all the medicines one takes, including
prescription and over-the-counter medicines, vitamins, and herbal
supplements. ULTOMIRIS and other medicines can affect each other
causing side effects. Know the medicines one takes and the vaccines
one receives. Keep a list of them to show the doctor and pharmacist
when one gets a new medicine.
If one has PNH and stops receiving ULTOMIRIS, the doctor will
need to monitor closely for at least 16 weeks after one stops
ULTOMIRIS. Stopping ULTOMIRIS may cause breakdown of the red blood
cells due to PNH. Symptoms or problems that can happen due to red
blood cell breakdown include: drop in the red blood cell count,
tiredness, blood in the urine, stomach-area (abdomen) pain,
shortness of breath, blood clots, trouble swallowing, and erectile
dysfunction (ED) in males. If one has aHUS, the doctor will need to
monitor closely for at least 12 months after stopping treatment for
signs of worsening aHUS symptoms or problems related to a type of
abnormal clotting and breakdown of the red blood cells called
thrombotic microangiopathy (TMA). Symptoms or problems that can
happen with TMA may include: confusion or loss of consciousness,
seizures, chest pain (angina), difficulty breathing, and blood
clots or stroke. If one misses an ULTOMIRIS infusion, call the
doctor right away.
ULTOMIRIS can cause serious side effects including infusion
reactions. Infusion reactions may happen during one’s ULTOMIRIS
infusion. Symptoms of an infusion reaction with ULTOMIRIS may
include lower back pain, pain with the infusion, feeling faint or
discomfort in the arms or legs. Tell the doctor or nurse right away
if these symptoms develop, or any other symptoms during the
ULTOMIRIS infusion that may mean one is having a serious infusion
reaction, including: chest pain, trouble breathing or shortness of
breath, swelling of the face, tongue, or throat, and feel faint or
pass out. One’s doctor will treat the symptoms as needed.
The most common side effects of ULTOMIRIS in people treated for
PNH are upper respiratory infection and headache. The most common
side effects of ULTOMIRIS in people with aHUS are upper respiratory
infections, diarrhea, nausea, vomiting, headache, high blood
pressure, and fever.
Please see the accompanying full Prescribing Information and
Medication Guide for ULTOMIRIS, including Boxed WARNING regarding
serious and life-threatening meningococcal
infections/sepsis.
About Alexion Alexion is a global biopharmaceutical
company focused on serving patients and families affected by rare
diseases through the discovery, development and commercialization
of life-changing therapies. As the global leader in complement
biology and inhibition for more than 20 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). In addition, the company is developing several
mid-to-late-stage therapies, including a second complement
inhibitor, a copper-binding agent for Wilson disease and an
anti-neonatal Fc receptor (FcRn) antibody for rare Immunoglobulin G
(IgG)-mediated diseases as well as several early-stage therapies,
including one for light chain (AL) amyloidosis and a second
anti-FcRn therapy. 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, and metabolic disorders. Alexion has been named to the
Forbes’ list of the World’s Most Innovative Companies seven years
in a row and is headquartered in Boston, Massachusetts’ Innovation
District. The company also 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-G]
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 potential benefits of
ULTOMIRIS as a treatment for patients with aHUS, the impact of aHUS
on patients, the benefits of diagnosing aHUS in patients; the
timing for regulatory filings for marketing authorizations of
ULTOMIRIS for the treatment of aHUS in the EU and Japan; and that
ULTOMIRIS can provide benefits for patients with aHUS.
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; 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 TMA (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 (including intellectual property lawsuits
relating to ULTOMIRIS brought by third parties and inter partes
review petitions submitted by third parties); 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; uncertainties surrounding legal
proceedings, company investigations and government investigations,
including investigations of Alexion by the U.S. Securities and
Exchange Commission (SEC) and U.S. Department of Justice; 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 Syntimmune 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 quarter ended June 30, 2019 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/20191018005600/en/
Media Megan Goulart, 857-338-8634 Senior Director,
Corporate Communications Investors Susan Altschuller, Ph.D.,
857-338-8788 Vice President, Investor Relations
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