New Novartis medicine Adakveo® (crizanlizumab) approved by FDA to reduce frequency of pain crises in individuals living with sickle cell disease
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 ® (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
Adakveo ® (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
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/beta0-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.
Adakveo® (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.
This press release contains forward-looking statements within the 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,” “launch,” or similar terms, or by express or implied discussions regarding potential marketing approvals, new indications or labeling for the investigational or approved products described in this press release, or regarding potential future revenues from such products. You should not place undue reliance on these statements. Such forward-looking statements are based on our current beliefs and expectations regarding future events, and are subject to significant known and unknown risks and uncertainties. Should one or more of these risks or uncertainties materialize, or should underlying assumptions prove incorrect, actual results may vary materially from those set forth in the forward-looking statements. There can be no guarantee that the investigational or approved products described in this press release will be submitted or approved for sale or for any additional indications or labeling in any market, or at any particular time. Nor can there be any guarantee that such products will be commercially successful in the future. In particular, our expectations regarding such products could be affected by, among other things, the uncertainties inherent in research and development, including clinical trial results and additional analysis of existing clinical data; regulatory actions or delays or government regulation generally; global trends toward health care cost containment, including government, payor and general public pricing and reimbursement pressures and requirements for increased pricing transparency; our ability to obtain or maintain proprietary intellectual property protection; the particular prescribing preferences of physicians and patients; general political and economic conditions; safety, quality or manufacturing issues; potential or actual data security and data privacy breaches, or disruptions of our information technology systems, and other risks and factors referred to in Novartis AG’s current Form 20-F on file with the US Securities and Exchange Commission. Novartis is providing the information in this press release as of this date and does not undertake any obligation to update any forward-looking statements contained in this press release as a result of new information, future events or otherwise.
Novartis is reimagining medicine to improve and extend people’s lives. As a leading global medicines company, we use innovative science and digital technologies to create transformative treatments in areas of great medical need. In our quest to find new medicines, we consistently rank among the world’s top companies investing in research and development. Novartis products reach more than 750 million people globally and we are finding innovative ways to expand access to our latest treatments. About 109,000 people of more than 140 nationalities work at Novartis around the world. Find out more at
Novartis is on Twitter. Sign up to follow @Novartis at
and follow @NovartisCancer at
For Novartis multimedia content, please visit www.novartis.com/news/media-library
For questions about the site or required registration, please contact firstname.lastname@example.org
- Steinberg M. Management of sickle cell disease. N Engl J Med. 1999;340(13):1021-1030.
- 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.
- 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.
- Adakveo (crizanlizumab) prescribing information. East Hanover, New Jersey, USA. Novartis Pharmaceuticals Corporation; November 2019.
- Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010;376(9757):2018-2031.
- 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.
- 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.
- Sparkenbaugh E, Pawlinski R. Interplay between coagulation and vascular inflammation in sickle cell disease. Br J Haematol. 2013;162(1):1-22.
- Ballas SK, Gupta K, Adams-Graves P. Sickle cell pain: a critical reappraisal. Blood. 2012;120(18):3647-3656.
- 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.
# # #
Novartis Global External Communications
Novartis Global Media Relations
+41 61 324 1374 (direct)
+41 79 723 3681 (mobile)
Novartis US External Communications
+ 1 646 438 4335
Novartis Hematology Communications
+1 862 778 8656 (direct)
+1 201 400 1854 (mobile)
Novartis Investor Relations
Central investor relations line: +41 61 324 7944
|Samir Shah||+41 61 324 7944||Sloan Simpson||+1 862 778 5052|
|Pierre-Michel Bringer||+41 61 324 1065||Cory Twining||+1 862 778 3258|
|Thomas Hungerbuehler||+41 61 324 8425|
|Isabella Zinck||+41 61 324 7188|
# # #
One Liberty Plaza - 165 Broadway
NY 10006 New York
GlobeNewswire is one of the world's largest newswire distribution networks, specializing in the delivery of corporate press releases financial disclosures and multimedia content to the media, investment community, individual investors and the general public.
Subscribe to releases from GlobeNewswire
Subscribe to all the latest releases from GlobeNewswire by registering your e-mail address below. You can unsubscribe at any time.
Latest releases from GlobeNewswire
PCI Biotech: Update on the preclinical research collaboration agreement with AstraZeneca13.7.2020 19:31:34 CEST | Press release
Oslo (Norway), 13 July 2020 – PCI Biotech (OSE: PCIB), a cancer focused biopharmaceutical company, today announced a 6 month extension of the evaluation period under its preclinical research collaboration with AstraZeneca. The collaboration was initiated in September 2015 to determine whether PCI Biotech's fimaNAc technology has the potential to enhance the delivery of AstraZeneca's nucleic acid therapeutic compounds. The evaluation of the potential for a further collaboration now runs to the end of 2020. Contact information: Per Walday, CEO email@example.com Mobile: +47 917 93 429 About PCI Biotech PCI Biotech is a biopharmaceutical late stage clinical development company focusing on development and commercialisation of novel therapies for the treatment of cancer through its innovative photochemical internalisation (PCI) technology platform. PCI is applied to three distinct anticancer paradigms: fimaChem (enhancement of chemotherapeutics for localised treatment of cancer), fimaVacc (T-
EVS Broadcast Equipment reports update of share buyback program13.7.2020 17:45:00 CEST | Press release
Publication on July 13, 2020, after market closing Regulated information – reporting share buyback EVS Broadcast Equipment S.A.: Euronext Brussels (EVS.BR), Bloomberg (EVS BB), Reuters (EVSB.BR) EVS reports update of share buyback program EVS Broadcast Equipment reports that the following transactions, conducted within the framework of the share buyback program announced on May 6, 2020, took place between July 6, 2020 and July 10, 2020. DateNumber of shares acquiredAverage price (EUR)Highest price (EUR)Lowest price (EUR)Total (EUR)06/07/20202,11216.427616.5016.36 34,695 07/07/20202,10016.507816.6216.38 34,666 08/07/20202,50016.409116.4416.30 41,023 09/07/20201,89816.363216.4216.30 31,057 10/07/20202,20216.285916.3816.24 35,862 Total10,812 16.3987 16.62 16.24 177,303 As of July 10, 2020, and since the start of the buyback program, EVS has bought 95,521 shares at an average price of EUR 15,6592, representing in total EUR 1,495,785. After aforementioned transactions the total number of ow
XBT Provider AB (Publ) Publishes Half Year Interim Report 202013.7.2020 17:30:00 CEST | Press release
XBT Provider AB (Publ) Publishes Half Year Interim Report Stockholm, July 13, 2020 -- XBT Provider AB (Publ) ("XBT" or the "Company") is pleased to announce that the Interim Report for the period ended 30 June 2020 has been posted on the Company's website. The Guarantor, CoinShares (Jersey) Limited, has also provided an unaudited set, of their financial statements for the period ending on 30 June 2020, which are also posted on the Company's website. Interested persons may access an electronic copy of these documents at https://coinshares.com/etps/xbt-provider/investor-resources About XBT Provider XBT Provider AB (Publ) (XBT Provider), a CoinShares company, is the Swedish-domiciled issuer of the Bitcoin Tracker One (SE0007126024), Bitcoin Tracker Euro (SE0007525332), Ether Tracker One (SE0010296574) and Ether Tracker Euro (SE0010296582), Litecoin Tracker One (SE0011414465), Litecoin Tracker Euro (SE0011414457), XRP Tracker One (SE0011414481), XRP Tracker Euro (SE0011414473), series of c
Valmet’s comment on Alfa Laval’s tender offer for Neles’ shareholders13.7.2020 16:00:00 CEST | Press release
Valmet Oyj’s press release July 13, 2020 at 5:00 p.m. EET Valmet has today become aware of Alfa Laval’s announcement on public cash tender offer for all shares in Neles. Valmet completed 14.88 percent share acquisition of Neles’ shares from Solidium Oy on July 1, 2020. “Neles is a good quality company with a strong position in the pulp and paper industry and other process industries. Valmet and Neles have a common heritage, serve similar global industries and benefit from same global megatrends. Valmet sees a good amount of value in Neles and with the previously announced share acquisition our target is to participate in developing the company further. Therefore, as a Neles shareholder, Valmet does not consider Alfa Laval’s tender offer to be beneficial for Neles. Valmet will continue as an active shareholder of Neles,” says Pasi Laine, President and CEO of Valmet. Valmet acquires 14.9% ownership in the future Neles Valmet completes 14.9% ownership acquisition of Neles VALMET Corporate
Verisk to Announce Fiscal Second-Quarter 2020 Results on August 4, 202013.7.2020 15:29:14 CEST | Press release
Jersey City, NJ, July 13, 2020 (GLOBE NEWSWIRE) -- Verisk (Nasdaq:VRSK), a leading data analytics provider serving customers in insurance, energy and specialized markets, and financial services, will report its financial results for the fiscal second quarter ended June 30, 2020, on Tuesday, August 4, 2020, after the market close. The press release, with accompanying financial information, will be posted on the Verisk investor website at http://investor.verisk.com. Verisk’s management team will host a live audio webcast to discuss the financial results and business highlights on Wednesday, August 5, 2020, at 8:30 a.m. EDT (5:30 a.m. PDT, 1:30 p.m. BST). All interested parties are invited to listen to the live event via webcast on the Verisk investor website at http://investor.verisk.com. The discussion will also be available through dial-in number 1-877-755-3792 for U.S./Canada participants or 512-961-6560 for international participants. A replay of the webcast will be available for 30
CNA Transitions to AIR Worldwide as its Primary Catastrophe Risk Management Platform13.7.2020 13:02:10 CEST | Press release
Boston, July 13, 2020 (GLOBE NEWSWIRE) -- Catastrophe modeling firm AIR Worldwide announced that CNA has transitioned to AIR’s Touchstone solution as its primary catastrophe risk modeling platform. AIR Worldwide is a Verisk (Nasdaq:VRSK) business. “We are excited to leverage AIR’s powerful catastrophe modeling solution to support our multiple lines of business during the underwriting process,” said Tom Stone, vice president of aggregation and catastrophe management at CNA. “AIR has a track record of providing scientifically credible models and combined with Touchstone, this complete solution will provide us with more analytics to make risk management, pricing, and risk selection decisions.” As part of the agreement, CNA is accessing Touchstone in the AIR Cloud as a secure and cost-efficient alternative to on-premise deployment. CNA is also licensing AIR’s Web Services to connect to AIR’s cloud infrastructure and obtain hazard, and loss analysis output in seconds; and Touchstone APIs to