- The U.S. Food and Drug Administration approved the use of
IMBRUVICA for the treatment of pediatric patients one year and
older with cGVHD after failure of one or more lines of systemic
therapy
- This is the first approved treatment option for children
with cGVHD under 12 years of age and the only Bruton's tyrosine
kinase inhibitor (BTKi) treatment for a pediatric patient
population
- cGVHD is a rare condition that can occur in patients after
receiving peripheral blood or bone marrow stem cell transplantation
often related to blood cancer treatment
- IMBRUVICA was the first treatment approved in the U.S. in
2017 for adult patients with cGVHD after failure of one or more
lines of systemic therapy
NORTH
CHICAGO, Ill., Aug. 24,
2022 /PRNewswire/ -- AbbVie (NYSE: ABBV) today
announced that the U.S. Food and Drug Administration (FDA) approved
the use of IMBRUVICA® (ibrutinib) for the treatment of
pediatric patients one year and older with chronic graft versus
host disease (cGVHD) after failure of one or more lines of systemic
therapy.
The approval is AbbVie's first pediatric indication for
IMBRUVICA, marking the 12th FDA approval for IMBRUVICA
and the first Bruton's tyrosine kinase inhibitor (BTKi) treatment
approved for a pediatric patient population. This approval also
marks the first approved treatment option for children under 12
years of age suffering from cGVHD. The approval is primarily based
on positive results from the iMAGINE Phase 1/2 clinical trial.
cGVHD occurs when donated peripheral blood or bone marrow stem
cells view the recipient's body as foreign and the donated cells
launch an immune attack on the body.1 cGVHD impacts
major organs, including the skin, eyes, mouth and liver as the most
commonly affected.2 About 35% of the estimated
8,000 patients who undergo life-saving allogeneic hematopoietic
stem cell transplant (HSCT) per year develop cGVHD that requires
systemic treatment.1 Additionally, cGVHD is the
most common cause of morbidity after an allogeneic
transplant.3 Steroids are the current standard
treatment for pediatric cGVHD.4
"For a substantial fraction of children who develop moderate or
severe chronic graft versus host disease after blood or marrow
transplantation, treatment options supported by methodical, formal
study in children are limited," said Dr. Paul A. Carpenter, attending physician at
Seattle Children's Hospital and a study principal investigator.
"Now, healthcare professionals have another effective treatment
option for both pediatric and adult patients living with this
disease after failure of one or more lines of systemic therapy.
Additionally, having an oral suspension formulation designed for
children is a helpful alternative."
"This approval is incredibly meaningful as AbbVie's first
pediatric approval for IMBRUVICA as well as within our company's
oncology portfolio. As a Pediatric Oncologist, when my patients
describe the physical pain they experience from simply hugging
their parents due to their cGVHD, the importance of researching
alternative treatment options in this patient population is further
validated," said Gauri Sunkersett, D.O., associate medical director
at AbbVie. "At AbbVie, we are committed to driving oncology
research and developing therapies that have the potential to help
patients of all ages, including underserved populations like
children diagnosed with cGVHD."
The iMAGINE study demonstrated an Overall Response Rate (ORR)
through week 25 of 60% (Confidence Interval [CI] 95%; 44-74) in
patients median age 13 years (range, 1-19 years) (n=47) with
relapsed/refractory (R/R) moderate to severe cGVHD.5 The
median duration of response was 5.3 months (95% CI: 2.8,
8.8).5 Safety was consistent with the established
profile for IMBRUVICA, with observed adverse reactions (ARs)
consistent with those observed in adult patients with moderate to
severe cGVHD.5 The most common ARs (occurring in 20% or
more of patients), including laboratory abnormalities, were anemia,
musculoskeletal pain, pyrexia, diarrhea, pneumonia, abdominal pain,
stomatitis, thrombocytopenia, and headache.5
Since 2017, IMBRUVICA has been approved as a single-agent
therapy for adult patients with cGVHD who have experienced failure
of prior systemic therapy, becoming the first FDA-approved
treatment for cGVHD.
"In some ways, children struggling with cGVHD have been an
'orphan population' in relation to having treatment options
available. Although there has been progress in approved treatment
options for adults with cGVHD, the safety and efficacy of these
have not been well studied in children," said Susan Stewart, executive director of BMT
InfoNet, a non-profit advocacy organization for bone marrow, stem
cell and cord blood transplant patients. "This FDA approval of
IMBRUVICA gives children and their families struggling with this
very difficult disease newfound hope."
The recommended dose of IMBRUVICA for cGVHD patients 12 years
and older is 420 mg taken orally once daily until disease
progression or unacceptable toxicity.6 In cGVHD patients
one year old to less than 12 years of age, the recommended dose of
IMBRUVICA is 240 mg/m2 taken orally once daily (up to a
dose of 420 mg) until disease progression or unacceptable
toxicity.6 This FDA approval also includes an oral
suspension formulation of IMBRUVICA to provide an alternative and
child-friendly administration option.
About the iMAGINE Study5
iMAGINE
(PCYC-1146-IM) is an open-label, multi-center, single-arm trial of
IMBRUVICA for the treatment of pediatric and young adult patients
aged one year to less than 22 years with moderate or severe cGVHD
as defined by NIH Consensus Criteria. The study included 47
patients who required additional therapy after failure of one or
more prior lines of systemic therapy. Patients aged 12 years and
older were treated with IMBRUVICA 420 mg orally once daily, and
patients aged one year to less than 12 years were treated with
IMBRUVICA 240 mg/m2 orally once daily. Primary endpoints
included pharmacokinetics (PK) and safety, and secondary endpoints
included overall response rate (ORR; CR/PR) per 2014 NIH
criteria, overall survival, and duration of response (DOR).
About IMBRUVICA®
IMBRUVICA®
(ibrutinib) is a once-daily oral medication that is jointly
developed and commercialized by Janssen Biotech, Inc. and
Pharmacyclics LLC, an AbbVie company. IMBRUVICA® blocks
the Bruton's tyrosine kinase (BTK) protein, which is needed by
normal and abnormal B cells, including specific cancer cells, to
multiply and spread. By blocking BTK, IMBRUVICA® may
help move abnormal B cells out of their nourishing environments and
inhibits their proliferation.7,8,9
IMBRUVICA® is approved in more than 100 countries and
has been used to treat more than 250,000 patients worldwide. There
are more than 50 company-sponsored clinical trials, including 18
Phase 3 studies, over 11 years evaluating the efficacy and safety
of IMBRUVICA®.
IMBRUVICA® was first approved by the U.S. Food and
Drug Administration (FDA) in November
2013, and today is indicated for adult patients in six
disease areas, including five hematologic cancers. These include
indications to treat adults with CLL/SLL with or without 17p
deletion (del17p), adults with Waldenström's macroglobulinemia
(WM), adults with previously treated mantle cell lymphoma (MCL)*,
adult patients with previously treated marginal zone lymphoma (MZL)
who require systemic therapy and have received at least one prior
anti-CD20-based therapy*, as well as adult and pediatric patients
with previously treated chronic graft versus host disease (cGVHD)
after failure of one or more lines of systemic
therapy.6
*Accelerated approval was granted for MCL and MZL based on
overall response rate. Continued approval for MCL and MZL may be
contingent upon verification and description of clinical benefit in
confirmatory trials.
Since 2019, the National Comprehensive Cancer
Network® (NCCN®), recommends ibrutinib
(IMBRUVICA®) as a preferred regimen for the initial
treatment of CLL/SLL and has Category 1 treatment status for
treatment-naïve patients without deletion 17p/TP53 mutation
and as a preferred treatment for treatment-naïve patients with
deletion 17p/TP53 mutation. The NCCN Guidelines®
also recommend IMBRUVICA®, with or without rituximab, as
a preferred regimen for the treatment of relapsed/refractory MCL,
as a Category 1 preferred regimen for both untreated and previously
treated WM patients, and as a preferred regimen for
relapsed/refractory MZL.10
For more information, visit www.IMBRUVICA.com.
IMPORTANT SAFETY INFORMATION
Before taking IMBRUVICA®, tell your
healthcare provider about all of your medical conditions, including
if you:
- have had recent surgery or plan to have surgery. Your
healthcare provider may stop IMBRUVICA® for any planned
medical, surgical, or dental procedure.
- have bleeding problems
- have or had heart rhythm problems, smoke, or have a medical
condition that increases your risk of heart disease, such as high
blood pressure, high cholesterol, or diabetes
- have an infection
- have liver problems
- are pregnant or plan to become pregnant. IMBRUVICA®
can harm your unborn baby. If you are able to become pregnant, your
healthcare provider will do a pregnancy test before starting
treatment with IMBRUVICA®. Tell your healthcare provider
if you are pregnant or think you may be pregnant during treatment
with IMBRUVICA®.
-
- Females who are able to become pregnant should use
effective birth control (contraception) during treatment with
IMBRUVICA® and for 1 month after the last dose.
- Males with female partners who are able to become
pregnant should use effective birth control, such as condoms,
during treatment with IMBRUVICA® and for 1 month after
the last dose.
- are breastfeeding or plan to breastfeed. Do not
breastfeed during treatment with IMBRUVICA® and for 1
week after the last dose.
Tell your healthcare provider about all the medicines you
take, including prescription and over-the-counter medicines,
vitamins, and herbal supplements. Taking IMBRUVICA® with
certain other medicines may affect how IMBRUVICA® works
and can cause side effects.
How should I take IMBRUVICA®?
- Take IMBRUVICA® exactly as your healthcare provider
tells you to take it.
- Take IMBRUVICA® 1 time a day at about the same time
each day.
IMBRUVICA® comes as capsules, tablets, and oral
suspension.
- If your healthcare provider prescribes IMBRUVICA®
capsules or tablets:
-
- Swallow IMBRUVICA® capsules or tablets whole with a
glass of water.
- Do not open, break, or chew IMBRUVICA®
capsules.
- Do not cut, crush, or chew IMBRUVICA® tablets.
- If your healthcare provider prescribes IMBRUVICA®
oral suspension:
-
- See the detailed Instructions for Use that comes with
IMBRUVICA® oral suspension for information about the
correct way to give a dose to your child. If you have questions
about how to give IMBRUVICA® oral suspension, talk to
your healthcare provider.
- Do not use if the carton seal is broken or missing.
- If you miss a dose of IMBRUVICA® take it as soon as
you remember on the same day. Take your next dose of
IMBRUVICA® at your regular time on the next day. Do not
take extra doses of IMBRUVICA® to make up for a missed
dose.
- If you take too much IMBRUVICA® call your healthcare
provider or go to the nearest hospital emergency room right
away.
What should I avoid while taking
IMBRUVICA®?
- You should not drink grapefruit juice, eat grapefruit, or eat
Seville oranges (often used in
marmalades) during treatment with IMBRUVICA®. These
products may increase the amount of IMBRUVICA® in your
blood.
What are the possible side effects of
IMBRUVICA®?
IMBRUVICA®
may cause serious side effects, including:
- Bleeding problems (hemorrhage) are common during
treatment with IMBRUVICA®, and can also be serious and
may lead to death. Your risk of bleeding may increase if you are
also taking a blood thinner medicine. Tell your healthcare provider
if you have any signs of bleeding, including: blood in your stools
or black stools (looks like tar), pink or brown urine, unexpected
bleeding, or bleeding that is severe or that you cannot control,
vomit blood or vomit looks like coffee grounds, cough up blood or
blood clots, increased bruising, dizziness, weakness, confusion,
change in your speech, or a headache that lasts a long time or
severe headache.
- Infections can happen during treatment with
IMBRUVICA®. These infections can be serious and may lead
to death. Tell your healthcare provider right away if you have
fever, chills, weakness, confusion, or other signs or symptoms of
an infection during treatment with IMBRUVICA®.
- Heart problems. Serious heart rhythm problems
(ventricular arrhythmias, atrial fibrillation and atrial flutter),
heart failure and death have happened in people treated with
IMBRUVICA®, especially in people who have an infection,
an increased risk for heart disease, or have had heart rhythm
problems in the past. Your heart function will be checked before
and during treatment with IMBRUVICA®. Tell your
healthcare provider if you get any symptoms of heart problems, such
as feeling as if your heart is beating fast and irregular,
lightheadedness, dizziness, shortness of breath, swelling of the
feet, ankles or legs, chest discomfort, or you faint. If you
develop any of these symptoms, your healthcare provider may do
tests to check your heart and may change your IMBRUVICA®
dose.
- High blood pressure (hypertension). New or worsening
high blood pressure has happened in people treated with
IMBRUVICA®. Your healthcare provider may start you on
blood pressure medicine or change current medicines to treat your
blood pressure.
- Decrease in blood cell counts. Decreased blood counts
(white blood cells, platelets, and red blood cells) are common with
IMBRUVICA®, but can also be severe. Your healthcare
provider should do monthly blood tests to check your blood
counts.
- Second primary cancers. New cancers have happened during
treatment with IMBRUVICA®, including cancers of the skin
or other organs.
- Tumor lysis syndrome (TLS). TLS is caused by the fast
breakdown of cancer cells. TLS can cause kidney failure and the
need for dialysis treatment, abnormal heart rhythm, seizure, and
sometimes death. Your healthcare provider may do blood tests to
check you for TLS.
The most common side effects of IMBRUVICA®
in adults with B-cell malignancies (MCL, CLL/SLL, WM and MZL)
include:
- diarrhea
- tiredness
- muscle and bone pain
- rash
- bruising
The most common side effects of IMBRUVICA®
in adults or children 1 year of age and older with cGVHD
include:
- tiredness
- low red blood cell count (anemia)
- bruising
- diarrhea
- low platelet count
- muscle and joint pain
- fever
- muscle spasms
- mouth sores (stomatitis)
- bleeding
- nausea
- stomach pain
- pneumonia
- headache
Diarrhea is a common side effect in people who take
IMBRUVICA®. Drink plenty of fluids during
treatment with IMBRUVICA® to help reduce your
risk of losing too much fluid (dehydration) due to diarrhea. Tell
your healthcare provider if you have diarrhea that does not go
away.
These are not all the possible side effects of
IMBRUVICA®. Call your doctor for medical advice about
side effects. You may report side effects to FDA at
1-800-FDA-1088.
General information about the safe and effective use of
IMBRUVICA®
Medicines are sometimes prescribed for
purposes other than those listed in a Patient Information leaflet.
Do not use IMBRUVICA® for a condition for which it was
not prescribed. Do not give IMBRUVICA® to other people,
even if they have the same symptoms that you have. It may harm
them. You can ask your pharmacist or healthcare provider for
information about IMBRUVICA® that is written for health
professionals.
Please see the full Important Product
Information.
Uses
What is IMBRUVICA®
(ibrutinib)?
IMBRUVICA® (ibrutinib) is a
prescription medicine used to treat:
- Adults with mantle cell lymphoma (MCL) who have received at
least one prior treatment.
- Adults with chronic lymphocytic leukemia (CLL)/Small
lymphocytic lymphoma (SLL).
- Adults with chronic lymphocytic leukemia (CLL)/Small
lymphocytic lymphoma (SLL) with 17p deletion.
- Adults with Waldenström's macroglobulinemia (WM).
- Adults with marginal zone lymphoma (MZL) who require a medicine
by mouth or injection (systemic therapy) and have received a
certain type of prior treatment.
- Adults and children 1 year of age and older with chronic graft
versus host disease (cGVHD) after failure of 1 or more lines of
systemic therapy.
It is not known if IMBRUVICA® is safe and effective
in children under 1 year of age.
About AbbVie in Oncology
At AbbVie, we are committed
to transforming standards of care for multiple blood cancers while
advancing a dynamic pipeline of investigational therapies across a
range of cancer types. Our dedicated and experienced team joins
forces with innovative partners to accelerate the delivery of
potentially breakthrough medicines. We are evaluating more than 20
investigational medicines in over 300 clinical trials across some
of the world's most widespread and debilitating cancers. As we work
to have a remarkable impact on people's lives, we are committed to
exploring solutions to help patients obtain access to our cancer
medicines. For more information, please visit
http://www.abbvie.com/oncology.
About AbbVie
AbbVie's mission is to discover and
deliver innovative medicines that solve serious health issues today
and address the medical challenges of tomorrow. We strive to have a
remarkable impact on people's lives across several key therapeutic
areas: immunology, oncology, neuroscience, eye care, virology,
women's health and gastroenterology, in addition to products and
services across its Allergan Aesthetics portfolio. For more
information about AbbVie, please visit us at www.abbvie.com. Follow
@abbvie on Twitter, Facebook, Instagram, YouTube and LinkedIn.
Forward-Looking Statements
Some statements in this
news release are, or may be considered, forward-looking statements
for purposes of the Private Securities Litigation Reform Act of
1995. The words "believe," "expect," "anticipate," "project" and
similar expressions, among others, generally identify
forward-looking statements. AbbVie cautions that these
forward-looking statements are subject to risks and uncertainties
that may cause actual results to differ materially from those
indicated in the forward-looking statements. Such risks and
uncertainties include, but are not limited to, failure to realize
the expected benefits from AbbVie's acquisition of Allergan plc
("Allergan"), failure to promptly and effectively integrate
Allergan's businesses, competition from other products, challenges
to intellectual property, difficulties inherent in the research and
development process, adverse litigation or government action,
changes to laws and regulations applicable to our industry and the
impact of public health outbreaks, epidemics or pandemics, such as
COVID-19. Additional information about the economic, competitive,
governmental, technological and other factors that may affect
AbbVie's operations is set forth in Item 1A, "Risk Factors," of
AbbVie's 2021 Annual Report on Form 10-K, which has been filed with
the Securities and Exchange Commission, as updated by its
subsequent Quarterly Reports on Form 10-Q. AbbVie undertakes no
obligation to release publicly any revisions to forward-looking
statements as a result of subsequent events or developments, except
as required by law.
IMBRUVICA is a registered trademark of Pharmacyclics LLC.
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Lee SJ, Przepiorka D, et al. National Institutes of Health
Consensus Development Project on Criteria for Clinical Trials in
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http://ghr.nlm.nih.gov/condition/isolated-growth-hormone-deficiency.
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