Approval of first-in-class, oral, Factor D
inhibitor based on results from pivotal ALPHA Phase III
trial
VOYDEYA™ (danicopan) has been approved in the US as add-on
therapy to ravulizumab or eculizumab for the treatment of
extravascular hemolysis (EVH) in adults with paroxysmal nocturnal
hemoglobinuria (PNH).1 VOYDEYA is a first-in-class, oral, Factor D
inhibitor developed as an add-on to standard-of-care
ULTOMIRIS® (ravulizumab-cwvz) or SOLIRIS®
(eculizumab) to address the needs of the approximately 10-20% of
patients with PNH who experience clinically significant EVH while
treated with a C5 inhibitor.2,3
The approval by the US Food and Drug Administration (FDA) was
based on positive results from the pivotal ALPHA Phase III trial.
Results from the 12-week primary evaluation period of the trial
were published in The Lancet Haematology.2
Bart Scott, MD, Professor, Division of Hematology and Oncology
at the University of Washington Medical Center, and Professor,
Clinical Research Division at Fred Hutchinson Cancer Center, said:
“The approval of VOYDEYA offers this small subset of PNH patients
an add-on therapy designed to address EVH, while maintaining
disease control with ULTOMIRIS or SOLIRIS. Terminal complement
inhibition with ULTOMIRIS can address the life-threatening
complications of PNH, building on the efficacy and safety of
SOLIRIS established over nearly 20 years.”
Marc Dunoyer, Chief Executive Officer, Alexion, said: “The
approval of first-in-class, Factor D inhibitor VOYDEYA marks an
important advancement in the treatment of PNH and builds on our
leadership and commitment to bring forward innovation in complement
science. As the ALPHA trial suggests, dual complement pathway
inhibition at Factor D and C5 may be an optimal treatment approach
for this subset of patients with EVH, enabling them to continue
with proven standard-of-care therapy.”
The ALPHA Phase III trial evaluated the efficacy and safety of
VOYDEYA as add-on to ULTOMIRIS or SOLIRIS in patients with PNH who
experienced clinically significant EVH. Results showed that VOYDEYA
met the primary endpoint of change in hemoglobin from baseline to
week 12 and all key secondary endpoints, including transfusion
avoidance and change in Functional Assessment of Chronic Illness
Therapy – Fatigue (FACIT-Fatigue) score.2
Results from the ALPHA Phase III trial showed VOYDEYA was
generally well tolerated, and no new safety concerns were
identified. In the trial, the most common treatment-emergent
adverse events were headache, nausea, arthralgia and diarrhea.2
VOYDEYA has been granted Breakthrough Therapy designation by the
US FDA and PRIority MEdicines (PRIME) status by the European
Medicines Agency. VOYDEYA has also been granted Orphan Drug
Designation in the US, European Union (EU) and Japan for the
treatment of PNH. VOYDEYA has been approved in Japan and
recommended for approval in the EU. Regulatory reviews are ongoing
in additional countries.
INDICATION & IMPORTANT SAFETY INFORMATION FOR VOYDEYA™
(danicopan)
INDICATION
What is VOYDEYA?
VOYDEYA is a prescription medicine used along with ravulizumab
or eculizumab to treat breakdown of red blood cells that takes
place outside of blood vessels (extravascular hemolysis), in adults
with paroxysmal nocturnal hemoglobinuria (PNH).
It is not known if VOYDEYA is safe and effective in
children.
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about
VOYDEYA?
VOYDEYA is a medicine that affects your immune system.
VOYDEYA may lower the ability of your immune system to fight
infections.
- VOYDEYA increases your chance of getting serious infections
caused by encapsulated bacteria. These serious infections may
quickly become life-threatening and cause death if not recognized
and treated early.
- You must complete or update meningococcal vaccine(s) and
streptococcus vaccine(s) at least 2 weeks before your first dose of
VOYDEYA.
- If you have not completed your vaccinations and VOYDEYA must be
started right away, you should receive the required vaccinations as
soon as possible.
- If you have not been vaccinated at least 2 weeks before your
first VOYDEYA dose and VOYDEYA must be started right away, you
should also receive antibiotics to take for as long as your
healthcare provider tells you.
- If you have been vaccinated against these bacteria in the past,
you might need additional vaccinations before starting VOYDEYA.
Your healthcare provider will decide if you need additional
vaccinations.
- Vaccines do not prevent all infections caused by encapsulated
bacteria. Call your healthcare provider or get emergency medical
care right away if you have any of these signs and symptoms of a
serious infection: fever with or without chills, fever and a
rash, fever with chest pain and cough, fever with
breathlessness/fast breathing, fever with high heart rate, headache
with nausea or vomiting, headache and a fever, headache with a
stiff neck or stiff back, confusion, body aches with flu-like
symptoms, clammy skin, eyes sensitive to light.
Your healthcare provider will give you a Patient Safety Card
about the risk of serious infections. Carry it with you at all
times during treatment and for 1 week after your last VOYDEYA dose.
Your risk of serious infections may continue for a few days after
your last dose of VOYDEYA. It is important to show this card to any
healthcare provider who treats you. This will help them diagnose
and treat you quickly.
VOYDEYA is only available through a program called the
VOYDEYA Risk Evaluation and Mitigation Strategy (REMS). Before
you can take VOYDEYA, your healthcare provider must: enroll in the
VOYDEYA REMS; counsel you about the risk of serious infections
caused by certain bacteria; give you information about the symptoms
of serious infections; make sure that you are vaccinated against
serious infections caused by encapsulated bacteria and that you
receive antibiotics if you need to start VOYDEYA right away and you
are not up to date on your vaccinations; give you a Patient
Safety Card about your risk of serious infections, as discussed
above.
Who should not receive VOYDEYA?
Do not take VOYDEYA if you have a serious infection
caused by encapsulated bacteria, including Neisseria meningitidis,
Streptococcus pneumoniae, or Haemophilus influenzae type B
infection.
Before taking VOYDEYA, tell your healthcare provider about
all of your medical conditions, including if you: have an
infection or fever, have liver problems, are pregnant or plan to
become pregnant or are breastfeeding. It is not known if VOYDEYA
will harm your unborn baby or if it passes into your breast milk.
Do not breastfeed during treatment with VOYDEYA and for 3 days
after the last dose.
Tell your healthcare provider about all the vaccines you
receive and medicines you take, including prescription and
over-the-counter medicines, vitamins, and herbal supplements which
could affect your treatment. VOYDEYA may affect the way other
medicines work.
If you stop taking VOYDEYA, your healthcare provider will
need to monitor you closely for at least 2 weeks after your last
dose. Stopping treatment with VOYDEYA may cause a breakdown of
red blood cells due to PNH. Symptoms or problems that can happen
due to breakdown of red blood cells include: decreased
hemoglobin level in your blood and tiredness.
What are the possible side effects of VOYDEYA?
VOYDEYA can cause serious side effects including increased
liver enzyme levels and increased cholesterol. Your healthcare
provider will do blood tests to check your liver enzyme levels and
cholesterol before and during treatment with VOYDEYA. Your
healthcare provider may temporarily or permanently stop treatment
with VOYDEYA if you develop increased liver enzyme levels.
The most common side effect of VOYDEYA is headache.
Tell your healthcare provider about any side effect that bothers
you or that does not go away. These are not all of the possible
side effects of VOYDEYA. For more information, ask your healthcare
provider or pharmacist. Call your healthcare provider for medical
advice about side effects. You may report side effects to FDA at
1-800-FDA-1088.
Please see the accompanying full Prescribing
Information and Medication Guide for VOYDEYA (danicopan),
including Boxed WARNING regarding serious infections caused by
encapsulated bacteria.
INDICATION(S) & IMPORTANT SAFETY INFORMATION FOR
ULTOMIRIS® (ravulizumab-cwvz)
INDICATION(S)
What is ULTOMIRIS?
ULTOMIRIS is a prescription medicine used to treat:
- adults and children 1 month of age and older with a disease
called Paroxysmal Nocturnal Hemoglobinuria (PNH).
- 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).
- adults with a disease called generalized Myasthenia Gravis
(gMG) who are anti-acetylcholine receptor (AChR) antibody
positive.
- adults with a disease called Neuromyelitis Optica Spectrum
Disorder (NMOSD) who are anti-aquaporin 4 (AQP4) antibody
positive.
It is not known if ULTOMIRIS is safe and effective in children
younger than 1 month of age.
It is not known if ULTOMIRIS is safe and effective for the
treatment of gMG or NMOSD in children.
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about
ULTOMIRIS?
ULTOMIRIS is a medicine that affects your immune system and
may lower the ability of your immune system to fight
infections.
- ULTOMIRIS increases your chance of getting serious
meningococcal infections that may quickly become life-threatening
or cause death if not recognized and treated early.
- You must complete or update meningococcal vaccine(s) at least 2
weeks before your first dose of ULTOMIRIS.
- If you have not completed your meningococcal vaccines and
ULTOMIRIS must be started right away, you should receive the
required vaccine(s) as soon as possible.
- If you have not been vaccinated and ULTOMIRIS must be started
right away, you should also receive antibiotics for as long as your
healthcare provider tells you.
- If you had a meningococcal vaccine in the past, you might need
additional vaccines before starting ULTOMIRIS. Your healthcare
provider will decide if you need additional meningococcal
vaccines.
- Meningococcal vaccines do not prevent all meningococcal
infections. Call your healthcare provider or get emergency
medical care right away if you get any of these signs and symptoms
of a meningococcal infection: fever, fever with high heart
rate, headache and fever, confusion, muscle aches with flu-like
symptoms, fever and a rash, headache with nausea or vomiting,
headache with a stiff neck or stiff back, or eyes sensitive to
light.
Your healthcare provider will give you a Patient Safety Card
about the risk of serious meningococcal infection. Carry it
with you at all times during treatment and for 8 months after your
last ULTOMIRIS dose. Your risk of meningococcal infection may
continue for several months after your last dose of ULTOMIRIS. It
is important to show this card to any healthcare provider who
treats you. This will help them diagnose and treat you quickly.
ULTOMIRIS is only available through a program called the
ULTOMIRIS and SOLIRIS Risk Evaluation and Mitigation Strategy
(REMS). Before you can receive ULTOMIRIS, your healthcare
provider must: enroll in the REMS program; counsel you about the
risk of serious meningococcal infections; give you information
about the signs and symptoms of serious meningococcal infection;
make sure that you are vaccinated against serious infections caused
by meningococcal bacteria, and that you receive antibiotics if you
need to start ULTOMIRIS right away and are not up to date on your
vaccines; give you a Patient Safety Card about your risk of
meningococcal infection.
ULTOMIRIS may also increase the risk of other types of
serious infections, including Streptococcus pneumoniae,
Haemophilus influenzae, and Neisseria gonorrhoeae. Your
child should receive vaccines against Streptococcus pneumoniae and
Haemophilus influenzae type b (Hib) if treated with ULTOMIRIS.
Certain people may be at risk of serious infections with
gonorrhea.
Who should not receive ULTOMIRIS?
Do not receive ULTOMIRIS if you have a serious meningococcal
infection when you are starting ULTOMIRIS.
Before you receive ULTOMIRIS, tell your healthcare provider
about all of your medical conditions, including if you: have 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 your unborn baby or if it passes into your
breast milk. You should not breastfeed during treatment and for 8
months after your final dose of ULTOMIRIS.
Tell your healthcare provider about all the vaccines you
receive and medicines you take, including prescription and
over-the-counter medicines, vitamins, and herbal supplements which
could affect your treatment.
If you have PNH and you stop receiving ULTOMIRIS, your
healthcare provider will need to monitor you closely for at least
16 weeks after you stop ULTOMIRIS. Stopping ULTOMIRIS may cause
breakdown of your red blood cells due to PNH. Symptoms or problems
that can happen due to red blood cell breakdown include: drop
in your red blood cell count, tiredness, blood in
your urine, stomach-area (abdomen) pain, shortness of
breath, blood clots, trouble swallowing, and erectile
dysfunction (ED) in males.
If you have aHUS, your healthcare provider will need to
monitor you closely for at least 12 months after stopping treatment
for signs of worsening aHUS or problems related to a type of
abnormal clotting and breakdown of your 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.
What are the possible side effects of ULTOMIRIS?
ULTOMIRIS can cause serious side effects including
infusion-related reactions. Symptoms of an infusion-related
reaction with ULTOMIRIS may include lower back pain, abdominal
pain, muscle spasms, changes in blood pressure, tiredness, feeling
faint, shaking chills (rigors), discomfort in your arms or legs,
bad taste, or drowsiness. Stop treatment of ULTOMIRIS and tell your
healthcare provider right away if you develop these symptoms, or
any other symptoms during your ULTOMIRIS infusion that may mean you
are having a serious infusion-related reaction, including: chest
pain, trouble breathing or shortness of breath, swelling of your
face, tongue, or throat, and feel faint or pass out.
The most common side effects of ULTOMIRIS in people treated
for PNH are upper respiratory tract infection and headache.
The most common side effects of ULTOMIRIS in people treated
for aHUS are upper respiratory tract infection, diarrhea, nausea,
vomiting, headache, high blood pressure and fever.
The most common side effects of ULTOMIRIS in people with gMG
are diarrhea and upper respiratory tract infections.
The most common side effects of ULTOMIRIS in people with
NMOSD are COVID-19 infection, headache, back pain, urinary tract
infection, and joint pain (arthralgia).
Tell your healthcare provider about any side effect that bothers
you or that does not go away. These are not all the possible side
effects of ULTOMIRIS. For more information, ask your healthcare
provider or pharmacist. Call your healthcare provider right away if
you miss an ULTOMIRIS infusion or for medical advice about side
effects. You may report side effects to FDA at 1-800-FDA-1088.
Please see the accompanying full Prescribing
Information and Medication Guide for ULTOMIRIS, including
Boxed WARNING regarding serious meningococcal infections.
INDICATION(S) & IMPORTANT SAFETY INFORMATION FOR SOLIRIS®
(eculizumab)
INDICATION(S)
What is SOLIRIS?
SOLIRIS is a prescription medicine used to treat:
- people with paroxysmal nocturnal hemoglobinuria (PNH).
- people with atypical hemolytic uremic syndrome (aHUS). SOLIRIS
is not for use in treating people with Shiga toxin E. coli related
hemolytic uremic syndrome (STEC-HUS).
- adults with generalized myasthenia gravis (gMG) who are
anti-acetylcholine receptor (AChR) antibody positive.
- 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
What is the most important information I should know about
SOLIRIS?
SOLIRIS is a medicine that affects your immune system and may
lower the ability of your immune system to fight
infections.
- SOLIRIS increases your chance of getting serious
meningococcal infections that may quickly become life-threatening
or cause death if not recognized and treated early.
- You must complete or update your meningococcal vaccine(s)at
least 2 weeks before your first dose of SOLIRIS.
- If you have not been vaccinated and SOLIRIS must be started
right away, you should receive the required vaccine(s) as soon as
possible.
- If you have not been vaccinated and SOLIRIS must be started
right away, you should also receive antibiotics for as long as your
healthcare provider tells you.
- If you had a meningococcal vaccine in the past, you might need
additional vaccines before starting SOLIRIS. Your healthcare
provider will decide if you need additional meningococcal
vaccines.
- Meningococcal vaccines do not prevent all meningococcal
infections. Call your healthcare provider or get emergency
medical care right away if you get any of these signs and symptoms
of a serious meningococcal infection: fever, fever with high
heart rate, headache and fever, confusion, muscle aches with
flu-like symptoms, fever and rash, headache with nausea or
vomiting, headache with a stiff neck or stiff back, or eyes
sensitive to light.
Your healthcare provider will give you a Patient Safety Card
about the risk of serious meningococcal infection. Carry it
with you at all times during treatment and for 3 months after your
last dose of SOLIRIS. Your risk of meningococcal infection may
continue for several weeks after your last dose of SOLIRIS. It is
important to show this card to any healthcare provider who treats
you. This will help them diagnose and treat you quickly.
SOLIRIS is only available through a program called the
ULTOMIRIS and SOLIRIS Risk Evaluation and Mitigation Strategy
(REMS). Before you can receive SOLIRIS, your healthcare
provider must: enroll in the REMS program; counsel you about the
risk of serious meningococcal infections; give you information
about the signs and symptoms of serious meningococcal infection;
make sure that you are vaccinated against serious infections caused
by meningococcal bacteria, and that you receive antibiotics if you
need to start SOLIRIS right away and you are not up to date on your
vaccines; give you a Patient Safety Card about your risk of
meningococcal infection.
SOLIRIS may also increase the risk of other types of serious
infections, including Streptococcus pneumoniae, Haemophilus
influenzae, and Neisseria gonorrhoeae. Your child should receive
vaccines against Streptococcus pneumoniae and Haemophilus
influenzae type b (Hib) if treated with SOLIRIS. Certain people may
be at risk of serious infections with gonorrhea. Certain fungal
infections (Aspergillus) may occur if you take SOLIRIS and have a
weak immune system or a low white blood cell count.
Who should not receive SOLIRIS?
Do not receive SOLIRIS if you have a serious meningococcal
infection when you are starting SOLIRIS.
Before you receive SOLIRIS, tell your healthcare provider
about all of your medical conditions, including if you: have an
infection or fever, are pregnant or plan to become pregnant, and
are breastfeeding or plan to breastfeed. It is not known if SOLIRIS
will harm your unborn baby or if it passes into your breast
milk.
Tell your healthcare provider about all the vaccines you
receive and medicines you take, including prescription and
over-the-counter medicines, vitamins, and herbal supplements which
could affect your treatment.
If you have PNH, your healthcare provider will need to
monitor you closely for at least 8 weeks after stopping SOLIRIS.
Stopping treatment with SOLIRIS may cause breakdown of your red
blood cells due to PNH. Symptoms or problems that can happen
due to red blood cell breakdown include: drop in the number of your
red blood cell count, drop in your platelet count, confusion,
kidney problems, blood clots, difficulty breathing, and chest
pain.
If you have aHUS, your healthcare provider will need to
monitor you closely during and for at least 12 weeks after stopping
SOLIRIS 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, swelling in arms or legs, and a drop in
your platelet count.
What are the possible side effects of SOLIRIS?
SOLIRIS can cause serious side effects including serious
infusion-related reactions. Tell your healthcare provider or
nurse right away if you get any of these symptoms during your
SOLIRIS infusion: chest pain, trouble breathing or shortness of
breath, swelling of your face, tongue, or throat, and feel faint or
pass out. If you have an infusion-related reaction to SOLIRIS, your
healthcare provider may need to infuse SOLIRIS more slowly, or stop
SOLIRIS.
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 your
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 your 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).
Tell your healthcare provider about any side effect that bothers
you or that does not go away. These are not all the possible side
effects of SOLIRIS. For more information, ask your healthcare
provider or pharmacist. Call your healthcare provider for medical
advice about side effects. You may report side effects to FDA at
1-800-FDA-1088.
Please see the accompanying full Prescribing
Information and Medication Guide for SOLIRIS,
including Boxed WARNING regarding serious meningococcal
infections.
Notes
PNH
PNH is a rare, chronic, progressive and potentially
life-threatening blood disorder. It is characterized by red blood
cell destruction within blood vessels (also known as intravascular
hemolysis) and white blood cell and platelet activation, which can
result in thrombosis (blood clots).4-6
PNH is caused by an acquired genetic mutation that may happen
any time after birth and results in the production of abnormal
blood cells that are missing important protective blood cell
surface proteins. These missing proteins enable the complement
system, which is part of the immune system and is essential to the
body’s defense against infection, to ‘attack’ and destroy or
activate these abnormal blood cells.4 Living with PNH can be
debilitating, and signs and symptoms may include blood clots,
abdominal pain, difficulty swallowing, erectile dysfunction,
shortness of breath, excessive fatigue, anemia and dark-colored
urine.4,7,8
Clinically Significant EVH
EVH, the removal of red blood cells outside of the blood
vessels, can sometimes occur in PNH patients who are treated with
C5 inhibitors.9,10 Since C5 inhibition enables PNH red blood cells
to survive and circulate, EVH may occur when these now surviving
PNH red blood cells are marked by proteins in the complement system
for removal by the spleen and liver.4,6,11 PNH patients with EVH
may continue to experience anemia, which can have various causes,
and may require blood transfusions.9,10,12,13 A small subset of
people living with PNH who are treated with a C5 inhibitor
experience clinically significant EVH, which results in continued
symptoms of anemia and may require blood transfusions.4,7,14,15
ALPHA
ALPHA is a pivotal, global Phase III trial designed as a
superiority study to evaluate the efficacy and safety of VOYDEYA as
an add-on to C5 inhibitor therapy SOLIRIS or ULTOMIRIS in patients
with PNH who experience clinically significant EVH. In the
double-blind, placebo-controlled, multiple-dose trial, patients
were enrolled and randomized to receive VOYDEYA or placebo (2:1) in
addition to their ongoing SOLIRIS or ULTOMIRIS therapy for 12
weeks. A prespecified interim analysis was performed once 63
randomized patients had completed 12 weeks of the primary
evaluation period or discontinued treatment as of June 28, 2022. At
12 weeks, patients on placebo plus SOLIRIS or ULTOMIRIS were
switched to VOYDEYA plus SOLIRIS or ULTOMIRIS, and patients on
VOYDEYA plus SOLIRIS or ULTOMIRIS remained on this treatment for an
additional 12 weeks. Patients who completed both treatment periods
(24 weeks) had the option to participate in a two-year long-term
extension period and continue to receive VOYDEYA in addition to
SOLIRIS or ULTOMIRIS. The open-label period of the study is
ongoing.2,16
VOYDEYA™ (danicopan)
VOYDEYA™ (danicopan) is a first-in-class oral Factor D
inhibitor. The medication works by selectively inhibiting Factor D,
a complement system protein that plays a key role in the
amplification of the complement system response. When activated in
an uncontrolled manner, the complement cascade over-responds,
leading the body to attack its own healthy cells. VOYDEYA has been
granted Breakthrough Therapy designation by the US Food and Drug
Administration and PRIority MEdicines (PRIME) status by the
European Medicines Agency. VOYDEYA has also been granted Orphan
Drug Designation in the US, EU and Japan for the treatment of
PNH.
VOYDEYA is approved in the US as add-on therapy to ravulizumab
or eculizumab for the treatment of EVH in adults with PNH.
VOYDEYA is also approved in Japan for certain adults with PNH in
combination with C5 inhibitor therapy.
Alexion is also evaluating VOYDEYA as a potential monotherapy
for geographic atrophy in a Phase II clinical trial.
ULTOMIRIS® (ravulizumab-cwvz)
ULTOMIRIS® (ravulizumab-cwvz), the first and only long-acting C5
complement inhibitor, provides immediate, complete and sustained
complement inhibition. 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 in adult patients, following a loading dose.
ULTOMIRIS is approved in the US, EU, Japan and other countries
for the treatment of certain adults with generalized myasthenia
gravis (gMG).
ULTOMIRIS is also approved in the US, EU, Japan and other
countries for the treatment of certain adults with PNH and for
certain children with PNH in the US and EU.
Additionally, ULTOMIRIS is approved in the US, EU, Japan and
other countries for certain adults and children with atypical
hemolytic uremic syndrome to inhibit complement-mediated thrombotic
microangiopathy (aHUS).
Further, ULTOMIRIS is approved in the US, EU and Japan for the
treatment of certain adults with neuromyelitis optica spectrum
disorder (NMOSD).
As part of a broad development program, ULTOMIRIS is being
assessed for the treatment of additional hematology and neurology
indications.
SOLIRIS® (eculizumab)
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.
SOLIRIS is approved in the US, EU, Japan, China and other
countries for the treatment of patients with PNH and aHUS.
Additionally, SOLIRIS is approved in Japan and the EU for the
treatment of certain adult and pediatric patients with gMG, and in
the US, China and other countries for certain adults with gMG.
Further, SOLIRIS is approved in the US, EU, Japan, China and
other countries for the treatment of certain adults with NMOSD.
SOLIRIS is not indicated for the treatment of patients with
Shiga-toxin E. coli-related hemolytic uremic syndrome.
Alexion
Alexion, AstraZeneca Rare Disease, is the group within
AstraZeneca focused on rare diseases, created following the 2021
acquisition of Alexion Pharmaceuticals, Inc. As a leader in rare
diseases for more than 30 years, Alexion is focused on serving
patients and families affected by rare diseases and devastating
conditions through the discovery, development and commercialization
of life-changing medicines. Alexion focuses its research efforts on
novel molecules and targets in the complement cascade and its
development efforts on hematology, nephrology, neurology, metabolic
disorders, cardiology and ophthalmology. Headquartered in Boston,
Massachusetts, Alexion has offices around the globe and serves
patients in 70 countries. For more information, please visit
www.alexion.com.
AstraZeneca
AstraZeneca (LSE/STO/Nasdaq: AZN) is a global, science-led
biopharmaceutical company that focuses on the discovery,
development, and commercialization of prescription medicines in
Oncology, Rare Diseases, and BioPharmaceuticals, including
Cardiovascular, Renal & Metabolism, and Respiratory &
Immunology. Based in Cambridge, UK, AstraZeneca operates in over
100 countries and its innovative medicines are used by millions of
patients worldwide. Please visit astrazeneca-us.com and follow the
Company on social media @AstraZeneca.
References
- VOYDEYA (danicopan) US prescribing information; March
2024.
- Lee JW, et al. Addition of danicopan to ravulizumab or
eculizumab in patients with paroxysmal nocturnal haemoglobinuria
and clinically significant extravascular haemolysis (ALPHA): a
double-blind, randomised, phase 3 trial. The Lancet Haematology.
2023;10(12):E955-E965.
- Kulasekararaj AG, et al. Prevalence of clinically significant
extravascular hemolysis in stable C5 inhibitor-treated patients
with PNH and its association with disease control, quality of life
and treatment satisfaction. Presented at: European Hematology
Association (EHA) Hybrid Congress. 8-11 June 2023; Frankfurt,
Germany. Abs PB2056.
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