– European Commission decision anticipated in
June 2020 –
– If approved, ULTOMIRIS has the potential to
become the new standard of care in Europe for the treatment of
atypical hemolytic uremic syndrome (aHUS) –
– aHUS is an ultra-rare disease which may
progressively damage the kidney and other organs1,2 –
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
the Committee for Medicinal Products for Human Use (CHMP) has
adopted a positive opinion, recommending marketing authorization in
the European Union for ULTOMIRIS® (ravulizumab) for the treatment
of patients with a body weight of 10 kg or above with atypical
hemolytic uremic syndrome (aHUS) who are complement inhibitor
treatment-naïve or have received SOLIRIS® (eculizumab) for at least
3 months and have evidence of response to eculizumab.
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 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.
“If approved in Europe, ULTOMIRIS will be the first and only
long-acting C5 inhibitor for the treatment of people with aHUS,”
said John Orloff, M.D., Executive Vice President and Head of
Research & Development at Alexion. “The consequences of aHUS
are severe and potentially devastating, including living with the
ongoing risk of life-threatening symptoms and complications. For
people with aHUS and their families, this creates significant
uncertainty and challenges. Today’s positive opinion marks an
important step in our efforts to bring ULTOMIRIS to the aHUS
patient community, where we believe it has the potential to become
the new standard of care for this devastating disease.”
The CHMP positive opinion 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.
Both studies are ongoing. 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).
Treatment with ULTOMIRIS resulted in normalization of platelet
count in 86 percent of adults and 94 percent of children,
normalization of LDH (marker of hemolysis) in 84 percent of adults
and 94 percent of children, and improved kidney function in 63
percent of adults and 94 percent (interim data) of children (for
patients on dialysis at enrollment, baseline was established after
they had come off dialysis). 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,
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 Risk Management Plan, have been established for
ULTOMIRIS.
The European Commission will review the CHMP recommendation and
typically delivers its final decision in approximately two months.
The U.S. Food and Drug Administration (FDA) approved ULTOMIRIS
(ravulizumab-cwvz) for the treatment of aHUS to inhibit TMA for
adult and pediatric (one month of age and older) patients in
October 2019. A regulatory filing for marketing authorization of
ULTOMIRIS for the treatment of aHUS in Japan is currently under
review. ULTOMIRIS is also approved for the treatment of adult
patients with paroxysmal nocturnal hemoglobinuria (PNH) in the U.S
and Japan, and in the EU as a treatment for adult patients with PNH
with hemolysis with clinical symptoms indicative of high disease
activity and for adult patients who are clinically stable after
having been treated with SOLIRIS® (eculizumab) for at least the
past six months.
About Atypical Hemolytic Uremic Syndrome (aHUS)
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 occurs when
the complement system—a part of the body’s immune
system—over-responds, leading the body to attack its own healthy
cells. 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. 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. Available
tests can help distinguish aHUS from other hemolytic diseases with
similar symptoms.
About ULTOMIRIS®
ULTOMIRIS® (ravulizumab) 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 the United
States (U.S.), European Union (EU) and Japan as a treatment for
adults with paroxysmal nocturnal hemoglobinuria (PNH) and in the
U.S. for atypical hemolytic uremic syndrome (aHUS) to inhibit
complement-mediated thrombotic microangiopathy (TMA) in adult and
pediatric (one month of age and older) patients. To learn more
about the regulatory status of ULTOMIRIS in the countries that we
serve, please visit www.alexion.com.
U.S. INDICATIONS & IMPORTANT SAFETY INFORMATION FOR
ULTOMIRIS (ravulizumab-cwvz) 300 mg / 30 mL injection for
intravenous use
U.S. 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.
U.S. 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 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 SOLIRIS®
SOLIRIS® (eculizumab) is a first-in-class 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 terminal complement
cascade over-responds, leading the body to attack its own healthy
cells. SOLIRIS is administered intravenously every two weeks,
following an introductory dosing period. In many countries around
the world, SOLIRIS is approved to treat paroxysmal nocturnal
hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS),
adults with generalized myasthenia gravis (gMG) who are
acetylcholine receptor (AchR) antibody positive and/or adults with
neuromyelitis optica spectrum disorder (NMOSD) who are
anti-aquaporin-4 (AQP4) antibody positive. SOLIRIS is not indicated
for the treatment of patients with Shiga-toxin E. coli-related
hemolytic uremic syndrome (STEC-HUS). To learn more about the
regulatory status of SOLIRIS in the countries that we serve, please
visit www.alexion.com.
U.S. Important Safety Information for SOLIRIS®
(eculizumab)
SOLIRIS is a medicine that affects the immune system. SOLIRIS
can lower the ability of the immune system to fight infections.
SOLIRIS 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 SOLIRIS if one has not already had this vaccine.
If one’s doctor decided that urgent treatment with SOLIRIS is
needed, meningococcal vaccination should be administered as soon as
possible. If one has not been vaccinated and SOLIRIS 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 SOLIRIS. 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.
SOLIRIS may also increase the risk of other types of serious
infections. If one’s child is treated with SOLIRIS, make sure that
the child receives vaccinations against Streptococcus pneumoniae
and Haemophilus influenzae type b (Hib). Certain people may be at
risk of serious infections with gonorrhea. Talk to the doctor about
whether one is at risk for gonorrhea infection, about gonorrhea
prevention, and regular testing. Certain fungal infections
(Aspergillus) may also happen if one takes SOLIRIS and has a weak
immune system or a low white blood cell count.
Before one receives SOLIRIS, tell the doctor about all of the
medical conditions, including if one: has an infection or fever, is
pregnant or plans to become pregnant, and is breastfeeding or plans
to breastfeed. It is not known if SOLIRIS will harm an unborn baby.
It is not known if SOLIRIS passes into the breast milk.
Tell the doctor about all the medicines one takes, including
prescription and over-the-counter medicines, vitamins, and herbal
supplements. SOLIRIS and other medicines can affect each other
causing side effects.
It is important that one: has all recommended vaccinations
before starting SOLIRIS, receives 2 weeks of antibiotics if one
immediately starts SOLIRIS, and stays up-to-date with all
recommended vaccinations during treatment with SOLIRIS. Know the
medications 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, the doctor will need to monitor closely for at
least 8 weeks after stopping SOLIRIS. Stopping treatment with
SOLIRIS 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 number of the red blood cell count,
drop in the platelet counts, confusion, kidney problems, blood
clots, difficulty breathing, and chest pain.
SOLIRIS can cause serious side effects including serious
allergic reactions. Serious allergic reactions can happen during
one’s SOLIRIS infusion. Tell the doctor or nurse right away if one
gets any of these symptoms during the SOLIRIS infusion: chest pain,
trouble breathing or shortness of breath, swelling of the face,
tongue, or throat, and feeling faint or pass out. If one has an
allergic reaction to SOLIRIS, the doctor may need to infuse SOLIRIS
more slowly, or stop SOLIRIS. The most common side effects in
people with PNH treated with SOLIRIS include: headache, pain or
swelling of the nose or throat (nasopharyngitis), back pain, and
nausea.
For more information, please see the accompanying full U.S.
Prescribing Information and Medication Guide for SOLIRIS, including
Boxed WARNING regarding serious and life-threatening meningococcal
infections, also available at: www.soliris.net.
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
medicines. 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 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-G]
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
timing for and final decision of the European Commission decision
on the potential approval of ULTOMIRIS as a treatment for aHUS to
inhibit complement-mediated TMA in adult and pediatric patients
(one month of age and older); ULTOMIRIS has the potential to become
the new standard of care in Europe for the treatment of aHUS;
timely and accurate diagnosis of aHUS – in addition to treatment –
is critical to improving patient outcomes; the CHMP opinion marks
an important step in our efforts to bring ULTOMIRIS to the aHUS
patient community, where we believe it has the potential to become
the new standard of care for this devastating disease; and the
anticipated timing of the review and decision of regulatory
agencies with respect to the potential approval of ULTOMIRIS as a
treatment for 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 European Commission may not approve
ULTOMIRIS as a treatment for aHUS to inhibit complement-mediated
TMA (regardless of the opinion of the CHMP) or may be delayed in
providing its approval beyond the anticipated approval date (and
such delay may be significant); 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 once
approved for use in the European Union); results of clinical trials
may not be sufficient to satisfy the European Commission or 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); the severity of the impact of the COVID-19
pandemic on Alexion’s business, including on commercial and
clinical development programs; 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 the European Commission and other 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; 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, 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 Achillion 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 Annual Report on
Form 10-K for the year ended December 31, 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.
References
1 Laurence J. Atypical hemolytic uremic syndrome (aHUS): making
the diagnosis. Clin Adv Hematol Oncol. 2012;10(suppl 17):1-12. 2
Campistol JM, Arias M, Ariceta G, et al. An update for atypical
haemolytic uraemic syndrome: diagnosis and treatment. Nefrologia.
2013 Jan 18;33(1): 27-45
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Media Megan Goulart, 857-338-8634 Senior Director,
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