– With this approval, ULTOMIRIS is the
first and only medicine approved in the U.S. to treat children and
adolescents with PNH –
– Approval based on interim results from Phase
3 study showing ULTOMIRIS demonstrated complete terminal complement
inhibition through 26 weeks –
Alexion Pharmaceuticals, Inc. (NASDAQ:ALXN) today announced the
U.S. Food and Drug Administration (FDA) has approved the expanded
use of ULTOMIRIS® (ravulizumab-cwvz) to include children
(one month of age and older) and adolescents with paroxysmal
nocturnal hemoglobinuria (PNH). ULTOMIRIS, a long-acting C5
inhibitor that offers immediate, complete and sustained complement
inhibition, is now the first and only FDA-approved medicine for
children and adolescents with PNH.
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Image of ULTOMIRIS® (ravulizumab-cwvz)
100 mg/mL vials (3 mL and 11 mL). (Photo: Business Wire)
“It can take months, and sometimes years, to receive a correct
diagnosis for PNH — a chronic, progressive and potentially
life-threatening rare disease – which can be an overwhelming
experience for children and their families,” said Satheesh Chonat,
M.D., principal investigator for the pediatric clinical trial and
pediatric hematologist and oncologist at the Aflac Cancer &
Blood Disorders Center at Children’s Healthcare of Atlanta, as well
as assistant professor of pediatrics at the Emory University School
of Medicine. “Managing the disease can be extremely burdensome for
these children and their families, who often miss school and work
for infusions, blood transfusions, and medical appointments. It’s
exciting to finally have an approved medicine for these patients
who are diagnosed as children.”
Since its initial approval in 2018, ULTOMIRIS has quickly become
the standard of care in the U.S. for the treatment of adults with
PNH. PNH is a complement-mediated disease, which means the symptoms
and complications are caused by a lack of regulation, or control,
of the complement system, an essential part of the immune system.
ULTOMIRIS is designed to target the part of the complement system
at the site of disease activity (terminal complement), while
preserving function of other parts of the immune system to be able
to fight common pathogens and infections. ULTOMIRIS reduces red
blood cell destruction in the blood vessels, also known as
intravascular hemolysis, and thrombosis (blood clot) risk by
providing immediate, complete, and sustained terminal complement
inhibition.
“This expanded approval is a significant step forward for the
PNH community as we work to elevate awareness of this rare disease
in children and adolescents and ensure patients, both pediatric and
adult, have meaningful treatment options available,” said Janice
Frey-Angel, Chief Executive Officer and Executive Director of the
Aplastic Anemia and Myelodysplastic Syndrome International
Foundation (AAMDSIF). “PNH can have significant physical, emotional
and/or psychological impacts on families, and we are pleased there
is now an approved medicine for the younger members of our
community and the families who care for them.”
This approval is based on interim Phase 3 study results, which
showed that ULTOMIRIS was effective in achieving complete C5
complement inhibition through 26 weeks in children and adolescents
up to 18 years of age. Additionally, ULTOMIRIS had no reported
treatment-related severe adverse events, and no patients
discontinued treatment during the primary evaluation period or
experienced breakthrough hemolysis, which can lead to disabling or
potentially fatal blood clots. The efficacy and safety of ULTOMIRIS
in children and adolescents is consistent with the established
profile of ULTOMIRIS in clinical studies involving adults with PNH
and is representative of the broad PNH patient population seen in
the real-world clinical setting. The results of the pediatric study
continue to demonstrate Alexion’s commitment to treating
complement-mediated diseases, which spans more than 17 clinical
trials – including the largest pediatric and adult trials in PNH
completed to date – and over 60,000 patient-years of trial and
real-world data, across more than 50 countries. Data from the
interim analysis will be presented as an e-poster during the
European Hematology Association 2021 Virtual Congress and will be
made available on the congress website on June 11, 2021 at 9:00
a.m. CEST (3:00 a.m. EDT).
“PNH can have a profound impact on a child's development and
quality of life. With its established safety and efficacy profile,
ULTOMIRIS has the potential to transform the lives of children and
adolescents suffering from this devastating rare disease,” said
John Orloff, M.D., Executive Vice President and Head of Research
and Development at Alexion. “We are inspired by the bravery and
resilience of the children and adolescents, as well as their
families, who participated in the study, and we are grateful for
their commitment — as well as that of the trial investigators — to
advancing the understanding of PNH and disease management in
younger people. We also appreciate the sense of urgency shown by
regulators in prioritizing reviewing and approving the first
treatment in the U.S. for children and adolescents with PNH.”
Alexion plans to make ULTOMIRIS available to pediatric patients
in the U.S. immediately. A regulatory filing for ULTOMIRIS in
pediatric patients with PNH is under review in the European Union
(EU).
About Paroxysmal Nocturnal Hemoglobinuria (PNH)
PNH is a serious ultra-rare blood disorder with 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 red blood 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. The
prognosis of PNH can be poor in many cases, so a timely and
accurate diagnosis — in addition to appropriate treatment — is
critical to improving patient outcomes.
About ULTOMIRIS®
ULTOMIRIS® (ravulizumab-cwvz) is the first and only long-acting
C5 complement inhibitor. The medication works by inhibiting the C5
protein in the terminal complement cascade, a part of the body’s
immune system. When activated in an uncontrolled manner, the
complement cascade over-responds, leading the body to attack its
own healthy cells. ULTOMIRIS is administered intravenously every
eight weeks or, for pediatric patients less than 20 kg, every four
weeks, following a loading dose. ULTOMIRIS is approved in the
United States (U.S.) for the treatment of adults and children (one
month of age and older) with paroxysmal nocturnal hemoglobinuria
(PNH), as well as in the European Union (EU) and Japan as a
treatment for adults with PNH. It is also approved in the U.S. and
Japan for atypical hemolytic uremic syndrome (aHUS) to inhibit
complement-mediated thrombotic microangiopathy (TMA) in adult and
pediatric (one month of age and older) patients, as well as in the
EU for the treatment of adults and children with a body weight of
at least 10 kg with aHUS. To learn more about the regulatory status
of ULTOMIRIS in the countries that we serve, please visit
www.alexion.com.
INDICATIONS & IMPORTANT SAFETY INFORMATION for ULTOMIRIS®
(ravulizumab-cwvz)
INDICATIONS
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).
It is not known if ULTOMIRIS is safe and effective in children
younger than 1 month of age.
IMPORTANT SAFETY INFORMATION
What is the most important information I should know about
ULTOMIRIS?
ULTOMIRIS is a medicine that affects your immune system and
can lower the ability of your immune system to fight
infections.
- ULTOMIRIS increases your chance of getting serious and
life-threatening meningococcal infections that may quickly become
life-threatening and cause death if not recognized and treated
early.
- You must receive meningococcal vaccines at least 2 weeks before
your first dose of ULTOMIRIS if you are not vaccinated.
- If your doctor decided that urgent treatment with ULTOMIRIS is
needed, you should receive meningococcal vaccination as soon as
possible.
- If you have not been vaccinated and ULTOMIRIS therapy must be
initiated immediately, you should also receive 2 weeks of
antibiotics with your vaccinations.
- If you had a meningococcal vaccine in the past, you might need
additional vaccination. Your doctor will decide if you need
additional vaccination.
- Meningococcal vaccines reduce but do not prevent all
meningococcal infections. Call your doctor or get emergency medical
care right away if you get any of these signs and symptoms of a
meningococcal infection: 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.
Your doctor will give you a Patient Safety Card about the
risk of meningococcal infection. Carry it with you at all times
during treatment and for 8 months after your last ULTOMIRIS dose.
It is important to show this card to any doctor or nurse to help
them diagnose and treat you quickly.
ULTOMIRIS is only available through a program called the
ULTOMIRIS REMS. Before you can receive ULTOMIRIS, your doctor
must: enroll in the ULTOMIRIS REMS program; counsel you about the
risk of meningococcal infection; give you information and a
Patient Safety Card about the symptoms and your risk of
meningococcal infection (as discussed above); and make sure that
you are vaccinated with a meningococcal vaccine, and if needed, get
revaccinated with the meningococcal vaccine. Ask your doctor if you
are not sure if you need to be revaccinated.
ULTOMIRIS may also increase the risk of other types of
serious infections. Make sure your child receives vaccinations
against Streptococcus pneumoniae and Haemophilis influenzae type b
(Hib) if treated with ULTOMIRIS. Call your doctor right away if you
have any new signs or symptoms of infection.
Who should not receive ULTOMIRIS?
Do not receive ULTOMIRIS if you have a meningococcal
infection or have not been vaccinated against meningococcal
infection unless your doctor decides that urgent treatment with
ULTOMIRIS is needed.
Before you receive ULTOMIRIS, tell your doctor 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 doctor 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 doctor
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 doctor 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, feeling faint
or discomfort in your arms or legs. Tell your doctor or nurse right
away if you develop these symptoms, or any other symptoms during
your ULTOMIRIS infusion that may mean you are having a serious
infusion 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 with aHUS
are upper respiratory tract infection, diarrhea, nausea, vomiting,
headache, high blood pressure and fever.
Tell your doctor 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 doctor or pharmacist.
Call your doctor 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 and life-threatening meningococcal
infections/sepsis.
About Alexion
Alexion is a global biopharmaceutical company focused on serving
patients and families affected by rare diseases and devastating
conditions through the discovery, development and commercialization
of life-changing medicines. As a leader in rare diseases for more
than 25 years, Alexion has developed and commercializes two
approved complement inhibitors to treat patients with paroxysmal
nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic
syndrome (aHUS), as well as the first and only approved complement
inhibitor to treat anti-acetylcholine receptor (AchR)
antibody-positive generalized myasthenia gravis (gMG) and
neuromyelitis optica spectrum disorder (NMOSD). Alexion also has
two highly innovative enzyme replacement therapies for patients
with life-threatening and ultra-rare metabolic disorders,
hypophosphatasia (HPP) and lysosomal acid lipase deficiency (LAL-D)
as well as the first and only approved Factor Xa inhibitor reversal
agent. In addition, the company is developing several
mid-to-late-stage therapies, including a copper-binding agent for
Wilson disease, an anti-neonatal Fc receptor (FcRn) antibody for
rare Immunoglobulin G (IgG)-mediated diseases and an oral Factor D
inhibitor as well as several early-stage therapies, including one
for light chain (AL) amyloidosis, a second oral Factor D inhibitor
and a third complement inhibitor. Alexion focuses its research
efforts on novel molecules and targets in the complement cascade
and its development efforts on hematology, nephrology, neurology,
metabolic disorders, cardiology, ophthalmology and acute care.
Headquartered in Boston, Massachusetts, Alexion has offices around
the globe and serves patients in more than 50 countries. This press
release and further information about Alexion can be found at:
www.alexion.com.
[ALXN-P]
For patient or advocacy inquiries please contact
patientadvocacy@alexion.com.
Forward-Looking Statements
This press release contains forward-looking statements that
involve risks and uncertainties relating to future events and the
future performance of Alexion and its products, including
statements related to: the anticipated benefits of ULTOMIRIS for
PNH patients (including children and adolescents); ULTOMIRIS’
continued safety and efficacy profile; anticipated timing to
receive a correct diagnosis for PNH; ULTOMIRIS’ expanded approval
is a significant step forward for the PNH community; PNH can have a
profound impact on a child's development and quality of life; with
its established safety and efficacy profile, ULTOMIRIS has the
potential to transform the lives of children and adolescents
suffering from this devastating rare disease; and Alexion plans to
make ULTOMIRIS available to pediatric patients in the U.S.
immediately. 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: ULTOMIRIS may not generate the expected benefits to
patients or the healthcare system that are anticipated; anticipated
regulatory approvals may be delayed or refused; the Company may
experience delays (or be prevented), due to manufacturing or other
reasons, from making available ULTOMIRIS to pediatric patients in
the U.S. immediately; results of clinical trials may not be
sufficient to satisfy regulatory authorities to approve ULTOMIRIS
as a treatment for PNH and/or aHUS or other indication (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
(including children and adolescents) 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 including ULTOMIRIS (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 trial 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 complement inhibitors; 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 the inability to launch product candidates due
to regulatory restrictions, anticipated expense, manufacturing
issues, 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;
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); the risk
that third party payors (including governmental agencies) will not
reimburse or continue to reimburse for the use of our products at
acceptable rates or at all; failure to realize the benefits and
potential of investments, collaborations, licenses and
acquisitions; the possibility that expected tax benefits will not
be realized; potential declines in sovereign credit ratings or
sovereign defaults in countries where we sell our products; delay
of collection or reduction in reimbursement due to adverse economic
conditions or changes in government and private insurer regulations
and approaches to reimbursement; adverse impacts on our supply
chain, clinical trials, manufacturing operations, financial
results, liquidity, hospitals, pharmacies and health care systems
from natural disasters and global pandemics, including the
coronavirus; uncertainties surrounding legal proceedings, company
investigations and government investigations; the risk that
estimates regarding the number of patients with PNH, aHUS, gMG,
NMOSD, HPP and LAL-D and other indications we are pursuing are
inaccurate; the impact of the proposed transaction between Alexion
and AstraZeneca plc; the risks of changing foreign exchange rates;
risks relating to the potential effects of the Company's
restructurings; 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 March 31, 2021 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.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20210607005638/en/
Media Lisa Taylor, 857-338-9025 Senior Director,
Corporate Communications
Investors Chris Stevo, 857-338-9309 Head of Investor
Relations
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