- Accepted abstracts include presentations of
ULTOMIRIS® (ravulizumab-cwvz) Phase 3 PNH data through 52 weeks
-
- ULTOMIRIS is the first and only long-acting
C5 complement inhibitor, now available in the U.S. for the
treatment of PNH and aHUS -
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced that
five scientific data presentations from the company’s complement
program will be showcased during the annual meeting of the American
Society of Hematology (ASH) in Orlando, Fla., from Dec. 7 to 10,
2019. During the Congress, researchers will present new, long-term
safety and efficacy findings through 52 weeks of treatment from the
Phase 3 trial of ULTOMIRIS® (ravulizumab-cwvz) in adult patients
with paroxysmal nocturnal hemoglobinuria (PNH) who had previously
received eculizumab treatment, as well as data on breakthrough
hemolysis in patients treated with ULTOMIRIS during the extension
phase (weeks 27-52) of the two Phase 3 studies involving adult
patients with PNH, which together comprise the largest PNH clinical
program ever conducted. Collectively, the breadth of the data to be
presented at ASH demonstrates the company’s continued commitment to
improving the care for people living with rare, complement-mediated
diseases.
ULTOMIRIS is approved in the United States (U.S.), Canada,
European Union (EU) and Japan for the treatment of adult patients
with PNH. ULTOMIRIS was also recently approved by the U.S. Food and
Drug Administration for the treatment of atypical hemolytic uremic
syndrome (aHUS) to inhibit complement-mediated thrombotic
microangiopathy (TMA) in adult and pediatric (one month of age and
older) patients. ULTOMIRIS is the first and only long-acting
complement inhibitor that provides immediate and complete C5
inhibition sustained with every eight-week dosing in adult
patients.
The accepted abstracts are listed below and are now available on
the ASH website.
Paroxysmal Nocturnal Hemoglobinuria (PNH) Abstracts
Breakthrough Hemolysis in Adult Patients with Paroxysmal Nocturnal
Hemoglobinuria Treated with Ravulizumab: Results of a 52-Week
Extension from Two Phase 3 Studies. Abstract ID#: 952 – Poster
Presentation, Dec. 7, 2019, 5:30 – 7:30 p.m. EST, Hall B.
One-Year Efficacy and Safety from A Phase 3 Trial of Ravulizumab
in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria
Receiving Prior Eculizumab Treatment. Abstract ID#: 2231 – Poster
Presentation, Dec. 8, 2019, 6:00 – 8:00 p.m. EST, Hall B.
Comparison of Lost Productivity Due to Eculizumab and
Ravulizumab Treatments for Paroxysmal Nocturnal Hemoglobinuria in
France, Germany, Italy, Russia, Spain, the United Kingdom, and the
United States. Available online.
Prophylactic Antibiotic Use and Risk of Meningococcal Infections
in Patients with Paroxysmal Nocturnal Hemoglobinuria (PNH) Treated
with Eculizumab Who Received Meningococcal Vaccination: Results
from the International PNH Registry. Available online.
Atypical Hemolytic Uremic Syndrome (aHUS) Abstracts
Discordance between Free C5 and CH50 Complement Assays in Measuring
Complement C5 Inhibition in Patients with aHUS Treated with
Ravulizumab. Abstract ID#: 1099 – Poster Presentation, Dec. 7,
2019, 5:30 – 7:30 p.m. EST, Hall B.
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
Paroxysmal nocturnal hemoglobinuria (PNH) is a serious ultra-rare
blood disorder with potentially devastating consequences. It is
characterized by the destruction of red blood cells, which is also
referred to as hemolysis. PNH occurs when the complement system, a
part of the body’s immune system, over-responds, leading the body
to attack its own healthy cells. PNH often goes unrecognized, with
delays in diagnosis from one to more than five years. Patients with
PNH may experience a range of symptoms, such as fatigue, difficulty
swallowing, shortness of breath, abdominal pain, erectile
dysfunction, dark-colored urine and anemia. The most devastating
consequence of chronic hemolysis is the formation of blood clots,
which can occur in blood vessels throughout the body, damage vital
organs, and potentially lead to premature death. PNH can strike men
and women of all races, backgrounds and ages without warning, with
an average age of onset in the early 30s. Patients with certain
types of hemolytic anemia, bone marrow disorders and unexplained
venous or arterial thrombosis are at increased risk of PNH.
About Atypical Hemolytic Uremic Syndrome (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® (ravulizumab-cwvz) 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 www.alexion.com.
About SOLIRIS® (eculizumab) SOLIRIS® (eculizumab) is a
first-in-class complement inhibitor that works by inhibiting the C5
protein in the terminal part of the complement cascade, a part of
the immune system. The terminal complement cascade, when activated
in an uncontrolled manner, plays a role in severe rare and
ultra-rare disorders. SOLIRIS, an intravenously administered
therapy, is approved in the U.S., EU, Japan and other countries as
a treatment for adult patients with PNH and for adults and children
with aHUS. SOLIRIS is not indicated for the treatment of patients
with Shiga-toxin E. coli-related hemolytic uremic syndrome
(STEC-HUS). In the U.S., SOLIRIS is also approved for the treatment
of generalized MG (gMG) in adult patients who are anti-AchR
antibody-positive and for the treatment of neuromyelitis optica
spectrum disorder (NMOSD) in adult patients who are anti-AQP4
antibody-positive, in the EU as the first and only treatment for
refractory gMG in adults who are anti-AchR antibody-positive and
for the treatment of NMOSD in adult patients who are anti-AQP4
antibody-positive with a relapsing course of the disease, and in
Japan for the treatment of patients with gMG who are anti-AChR
antibody-positive and whose symptoms are difficult to control with
high-dose intravenous immunoglobulin (IVIG) therapy or
plasmapheresis (PLEX).
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 the last 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.
INDICATIONS & IMPORTANT SAFETY INFORMATION FOR SOLIRIS®
(eculizumab) 300 mg / 30 mL injection for intravenous use
INDICATIONS What is SOLIRIS? SOLIRIS is a prescription
medicine called a monoclonal antibody. SOLIRIS is used to treat
patients with a disease called Paroxysmal Nocturnal Hemoglobinuria
(PNH). SOLIRIS is used to treat adults and children with a disease
called atypical Hemolytic Uremic Syndrome (aHUS). SOLIRIS is not
for use in treating people with Shiga toxin E. coli related
hemolytic uremic syndrome (STEC-HUS). SOLIRIS is used to treat
adults with a disease called generalized myasthenia gravis (gMG)
who are anti-acetylcholine receptor (AchR) antibody positive.
SOLIRIS is used to treat adults with a disease called neuromyelitis
optica spectrum disorder (NMOSD) who are anti-aquaporin-4 (AQP4)
antibody positive. It is not known if SOLIRIS is safe and effective
in children with PNH, gMG, or NMOSD.
IMPORTANT SAFETY INFORMATION 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 two 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, two 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. Patients should ask their doctor if an 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 provide a
Patient Safety Card about the risk of meningococcal infection.
Carry the card at all times during treatment and for 3 months after
the last SOLIRIS dose.
SOLIRIS is only available through a program called the SOLIRIS
REMS.
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.
Do not receive SOLIRIS if one has a meningococcal infection, or
has not been vaccinated against meningitis infection unless one’s
doctor decides that urgent treatment with SOLIRIS is needed.
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
or 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. If one has aHUS, the
doctor will need to monitor closely during and for at least 12
weeks after stopping treatment for signs of worsening aHUS symptoms
or problems related to abnormal clotting (thrombotic
microangiopathy). Symptoms or problems that can happen with
abnormal clotting may include: stroke, confusion, seizure, chest
pain (angina), difficulty breathing, kidney problems, swellings in
arms or legs, and a drop in the platelet count.
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. The most common side
effects in people with aHUS treated with SOLIRIS include: headache,
diarrhea, high blood pressure (hypertension), common cold (upper
respiratory infection), stomach-area (abdominal) pain, vomiting,
pain or swelling of the nose or throat (nasopharyngitis), low red
blood cell count (anemia), cough, swelling of legs or feet
(peripheral edema), nausea, urinary tract infections, and fever.
The most common side effects in people with gMG treated with
SOLIRIS include: muscle and joint (musculoskeletal) pain. The most
common side effects in people with NMOSD treated with SOLIRIS
include: common cold (upper respiratory infection); pain or
swelling of the nose or throat (nasopharyngitis); diarrhea; back
pain; dizziness; flu like symptoms (influenza) including fever,
headache, tiredness, cough, sore throat, and body aches; joint pain
(arthralgia); throat irritation (pharyngitis), and bruising
(contusion).
Please see the accompanying full Prescribing Information and
Medication Guide for SOLIRIS, including Boxed WARNING regarding
serious and life-threatening meningococcal infections.
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.
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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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