TIDMNOVN
-- Sickle cell pain crises are unpredictable, severe events associated with
life-threatening complications1
-- Adakveo reduced the annual rate of sickle cell pain crises by 45%
compared to placebo (1.63 vs 2.98) and the annual rate of days
hospitalized (4 vs 6.87) in a 52-week study2
-- Approximately 100,000 people in the United States, most of whom are of
African descent, have sickle cell disease3
-- Approval comes approximately two months ahead of FDA's priority review
action date, allowing Adakveo to be available to patients more quickly
Basel, November 15, 2019 -- Novartis announced today that the US Food
and Drug Administration (FDA) approved Adakveo(R) (crizanlizumab),
previously known as SEG101, to reduce the frequency of vaso-occlusive
crises (VOCs), or pain crises, in adult and pediatric patients aged 16
years and older with sickle cell disease.(4) Adakveo represents the
first FDA-approved medicine in sickle cell disease that binds to
P-selectin --a cell adhesion protein that plays a central role in the
multicellular interactions that can lead to vaso-occlusion.(5,6) The
medicine is expected to be available to patients in the coming weeks.
The FDA's decision to approve Adakveo 5 mg/kg is based on results of the
52-week, randomized, placebo-controlled SUSTAIN trial, which showed that
Adakveo significantly lowered the median annual rate of VOCs to 1.63 vs
2.98 compared to placebo (P=.010), which is equivalent to a 45%
reduction. Reductions in the frequency of VOCs were observed among
patients regardless of sickle cell disease genotype and/or hydroxyurea
use.(2,4)
"We know this drug can decrease the frequency of sickle cell pain crises
in a significant and clinically meaningful way," said Kenneth Ataga, MD,
Director, Center for Sickle Cell Disease, University of Tennessee Health
Science Center at Memphis, and Principal Investigator of the SUSTAIN
trial. "The approval of crizanlizumab is an important advancement for
people living with this very difficult condition."
Additional results from the SUSTAIN study include:(4)
-- A decrease in the median annual rate of days hospitalized to 4 vs 6.87
days when compared with placebo (a 42% reduction)
-- Thirty-six percent of patients treated with Adakveo did not experience a
VOC, compared to 17% of placebo-treated patients
-- The median time to first VOC was 4.1 for Adakveo vs 1.4 months for
placebo
The most common adverse reactions (incidence > 10%) were nausea (18%),
arthralgia (18%), back pain (15%) and pyrexia (11%).(4)
"The approval of Adakveo marks a new era in the treatment of sickle cell
disease, a genetic condition that places an extraordinary burden of
unpredictable pain crises on patients and their families," said Susanne
Schaffert, PhD, President, Novartis Oncology. "The stories we have heard
from patients about their sickle cell pain crises are devastating. We
are pleased to help reimagine medicine together with the sickle cell
community and offer new hope for fewer VOCs."
Considered the clinical hallmark of the disease, sickle cell pain crises
are triggered, in part, by multicellular interactions that form clusters
of cells, which can block or reduce the blood flow to organs.(1,7)
Sickle cell pain crises can be frequent and sudden, and are associated
with an increased risk of life-threatening complications.(1) They also
are the main reason why individuals living with sickle cell disease go
to the emergency room and are admitted to the hospital.(7)
"Patients with sickle cell disease often face unique challenges, and
have long suffered silently through unimaginable pain crises," said
Beverley Francis-Gibson, President and CEO of the Sickle Cell Disease
Association of America. "We are excited to have a new medicine that may
help many of the thousands of people living with sickle cell disease by
reducing the frequency of these potentially dangerous and painful
episodes."
About Sickle Cell Disease
Sickle cell disease is a complex and debilitating, genetic blood
disorder that goes beyond sickle-shaped red blood cells. The disease is
associated with chronic inflammation, causing higher levels of cell
adhesion proteins, including P-selectin, which make both the blood
vessels and certain blood cells stickier and prone to multicellular
interactions, or clusters, in the bloodstream. This environment can lead
to the acute episodes of pain known as sickle cell pain crises, or VOCs,
as well as life-threatening complications.(1,7,8) VOCs are the main
reason why individuals living with sickle cell disease seek medical care
in hospitals,(7) leading to approximately 200,000 ER visits in the US
every year.(9,10)
Approximately 100,000 people in the US have sickle cell disease.(3)
People of African ancestry make up 90% of the population with sickle
cell disease in the US. However, sickle cell disease is also prevalent
among people of Hispanic, South Asian, Southern European, and Middle
Eastern ancestry. Sickle cell disease occurs in about 1 in 365 and 1 in
16,300 African-American and Hispanic-American births, respectively.(3)
About Adakveo
Adakveo(R) (crizanlizumab) -- previously known as SEG101 -- is indicated
to reduce the frequency of VOCs, or pain crises, in adults and pediatric
patients aged 16 years and older with sickle cell disease. It is the
first and only targeted biologic that works by binding to P-selectin, a
cell adhesion protein that plays a central role in the multicellular
interactions that can lead to vaso-occlusion in sickle cell disease.
By binding to P-selectin on the surface of the activated endothelium and
platelets, Adakveo blocks interactions between endothelial cells,
platelets, red blood cells, and leukocytes.(4)
About SUSTAIN
SUSTAIN is a randomized, multicenter, placebo-controlled, double-blind
study. A total of 198 patients with any genotype of sickle cell disease
(HbSS, HbSC, HbS/beta(0) -thalassemia, HbS/beta(+) -thalassemia, and
others) and a history of 2-10 VOCs in the previous 12 months were
eligible for inclusion. Patients were randomized 1:1:1 to Adakveo 5
mg/kg (N = 67), Adakveo 2.5 mg/kg (N = 66), or placebo (N = 65)
administered over a period of 30 minutes by intravenous infusion on Week
0, Week 2, and every 4 weeks thereafter, for a treatment duration of 52
weeks.
The primary efficacy outcome was the annual rate of VOCs leading to a
healthcare visit. A VOC leading to a healthcare visit was defined as an
acute episode of pain with no cause other than a vaso-occlusive event
that required a medical facility visit and treatment with oral or
parenteral opioids, or parenteral NSAIDs. Acute chest syndrome, hepatic
sequestration, splenic sequestration, and priapism (requiring a visit to
a medical facility) were also considered VOCs. Key secondary and other
efficacy endpoints include annual rate of days hospitalized, time to
first VOC leading to healthcare visit, and number of patients that did
not experience a VOC.
Patient Access and Support
Novartis is committed to helping ensure that our medicines are
accessible to as many patients as possible. With the approval of Adakveo
in the United States, we now offer resources and support to address a
range of needs. Adakveo Support at PANO (Patient Assistance Now
Oncology) is a support center staffed by insurance specialists and case
managers who can help eligible patients start and stay on treatment.
Dedicated support specialists are available to help direct callers to
services that best fit their needs. Patients or providers can call
800-282-7630 or visit Patients.NovartisOncology.com or
HCP.Novartis.com/Access to learn more about eligibility and to enroll.
Novartis Commitment to Sickle Cell Disease in Africa
Sickle cell is a global disease and is most widespread in sub-Saharan
Africa. Unfortunately, we can see a clear disparity when comparing
Africa with other parts of the world, where sickle cell is often managed
as a chronic disease. Building on years of engagement in Africa, working
to reduce the impact of malaria and other conditions, Novartis is taking
steps to help address the needs of sickle cell patients as well,
beginning in Ghana. Our partnership with the Ghana Ministry of Health,
the Ghana Health Service, and the Sickle Cell Foundation of Ghana aims
to improve the diagnosis and treatment of people with sickle cell
disease through a comprehensive approach to screening and diagnosis,
treatment and disease management, training and education, and elevating
basic and clinical research and scientific capabilities. These
activities include facilitating access to high-quality hydroxyurea and
other basic medicines to enhance the standard of care.
To date, Novartis has delivered more than 20,000 hydroxyurea treatments
to Ghana, with plans to deliver a total of 60,000 treatments by the end
of the year. In addition, Novartis is developing a child-friendly
formulation of hydroxyurea and is committed to implementing two clinical
trials with crizanlizumab in Ghana and Kenya -- an important step to
bringing this innovative medicine to patients. Crizanlizumab trials in
Africa are expected to start in 2020.
Indication
Adakveo(R) (crizanlizumab-tmca) is used in people 16 years of age and
older, who have sickle cell disease, to help reduce how often certain
episodes of pain (crises) happen. It is not known if Adakveo is safe and
effective in children under 16 years of age.
Important Safety Information
Adakveo may cause serious side effects, including infusion reactions.
Infusion reactions may happen within 24 hours of receiving an infusion
of Adakveo. Patients should tell their health care provider right away
if they get any of the following signs and symptoms of an infusion
reaction such as fever, chills or shivering, nausea, vomiting, tiredness,
dizziness, sweating, hives, itching, or shortness of breath or wheezing.
Health care providers may monitor their patients for signs and symptoms
of infusion reactions.
Adakveo may interfere with automated platelet counts (platelet
clumping). Patients should tell their health care provider that they are
receiving Adakveo before having any blood tests. Health care providers
should run blood samples as soon as possible or use tubes containing
citrate.
Before receiving Adakveo, patients should tell their health care
provider if they are pregnant or plan to become pregnant. It is not
known if Adakveo may harm an unborn baby.
The most common side effects (incidence >=10%) include nausea, back pain,
joint pain, and fever.
Please see full Prescribing Information for Adakveo at
https://www.pharma.us.novartis.com/sites/www.pharma.us.novartis.com/files/adakveo.pdf.
Disclaimer
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meaning of the United States Private Securities Litigation Reform Act of
1995. Forward-looking statements can generally be identified by words
such as "potential," "can," "will," "plan," "expect," "anticipate,"
"look forward," "believe," "committed," "investigational," "pipeline,"
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or regarding potential future revenues from such products. You should
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About Novartis
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References
1. Steinberg M. Management of sickle cell disease. N Engl J Med.
1999;340(13):1021-1030.
2. Ataga KI, Kutlar A, Kanter J et al. Crizanlizumab for the prevention of
pain crises in sickle cell disease. N Engl J Med. 2017;376(5):429-439.
3. American Society of Hematology. State of sickle cell disease 2016 report.
http://www.scdcoalition.org/pdfs/ASH%20State%20of%20Sickle%20Cell%20Disease%202016%20Report.pdf.
Accessed October 24, 2019.
4. Adakveo (crizanlizumab) prescribing information. East Hanover, New Jersey,
USA. Novartis Pharmaceuticals Corporation; November 2019.
5. Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet.
2010;376(9757):2018-2031.
6. Lawrence MB, Springer TA. Leukocytes roll on a selectin at physiologic
flow rates: distinction from and prerequisite for adhesion through
integrins. Cell. 1991;65(5):859-873.
7. Gutsaeva D, Parkerson J, Yerigenahally S, et al. Inhibition of cell
adhesion by anti--P-selectin aptamer: a new potential therapeutic agent
for sickle cell disease. Blood. 2011;117(2):727-735.
8. Sparkenbaugh E, Pawlinski R. Interplay between coagulation and vascular
inflammation in sickle cell disease. Br J Haematol. 2013;162(1):1-22.
9. Ballas SK, Gupta K, Adams-Graves P. Sickle cell pain: a critical
reappraisal. Blood. 2012;120(18):3647-3656.
10. Yusuf HR, Atrash HK, Grosse SD, Parker CS, Grant AM. Emergency department
visits made by patients with sickle cell disease: a descriptive study,
1999-2007.Am J Prev Med. 2010;38(Suppl):S536-S541.
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E-mail: media.relations@novartis.com
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(END) Dow Jones Newswires
November 15, 2019 15:06 ET (20:06 GMT)
Copyright (c) 2019 Dow Jones & Company, Inc.
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