An early separation of the time to first VOC was observed at Week 2 with ADAKVEO. Full prescribing information for Adakveo, including recommended dosing schedules and safety warnings, can be found here. We are sorry that this post was not useful for you! 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. In the SUSTAIN clinical trial, infusion-related reactions (defined as occurring within 24 hours of infusion) were observed in 2 (3%) patients treated with ADAKVEO 5 mg/kg. 1 Adakveo can be given as an add-on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom … Ataga KI, Kutlar A, Kanter J, et al. In the 52-week SUSTAIN study, ADAKVEO was proven to reduce the frequency of VOCs 1 Significant 45% (1.63 vs 2.98) reduction in the median annual rate of VOCs vs placebo 1,2 * Reduction in the frequency of VOC s was observed regardless of VOC baseline … On 15 November 2019, the US Food and Drug Administration (FDA) approved crizanlizumab-tmca (ADAKVEO, Novartis) to reduce the frequency of vaso-occlusive crises (VOCs) in adults and … } font-size: 21px !important; Interference with automated platelet counts (platelet clumping) has been observed following administration of ADAKVEO, in particular, when blood samples were collected in tubes containing EDTA. The agency announced its decision two months ahead of its priority review action date, allowing SCD patients in the U.S. to have access to the medication sooner. East Hanover, NJ: Novartis Pharmaceuticals Corp; 2019. Discontinue ADAKVEO infusion for severe reactions and institute appropriate medical care. The EC approval is based on a positive opinion by the Committee for Medicinal Products for Human Use (CHMP). Findings from SUSTAIN demonstrated that Adakveo significantly reduced the frequency of annual VOCs by 45% compared to placebo (1.63 versus 2.98) at the 5 mg/kg dose. Adakveo is a monoclonal antibody that binds to P-selectin on the surface of the activated endothelium—the inside walls of blood vessels—and platelets. The SUSTAIN trial enrolled one pediatric patient treated with ADAKVEO 5 mg/kg aged 16 years old [see Adverse Reactions (6.1), Clinical Pharmacology (12.3), Clinical Studies (14)].The safety and efficacy of ADAKVEO in pediatric patients below the age of 16 years have not been established. That dose was shown to lower the frequency of annual VOCs in SCD patients in the company’s Phase 2 SUSTAIN (NCT01895361) trial. Copyright © 2013-2021 All rights reserved. That puts the therapy’s list price, which patients typically do not pay, at $7,071 to $9,428 per month or between $84,852 and $113,135 each year. Joana holds a BSc in Biology, a MSc in Evolutionary and Developmental Biology and a PhD in Biomedical Sciences from Universidade de Lisboa, Portugal. FDA approval of Adakveo was based on the results of the SUSTAIN trial, a 52-week, randomized, placebo-controlled, double-blind study of 198 patients with sickle cell disease and a history of VOCs. An icon used to represent a menu that can be toggled by interacting with this icon. Crizanlizumab, sold under the brand name Adakveo, is a monoclonal antibody medication developed by Novartis targeted towards P-selectin.It was announced by the company as an effective drug to prevent vaso-occlusive crisis in patients with sickle cell anemia.The result of the Phase II SUSTAIN clinical trial was published in December 2016. In SCD, P-selectin promotes the sticking of sickle-shaped red blood cells to each other and to the walls of blood vessels, obstructing the vessels and increasing the risk of VOCs. Clinically relevant adverse reactions (all grades) that were reported in <10% of patients treated with ADAKVEO included: oropharyngeal pain, abdominal pain (abdominal pain, upper abdominal pain, lower abdominal pain, abdominal discomfort, and abdominal tenderness), diarrhea, vomiting, pruritus (pruritus and vulvovaginal pruritus), musculoskeletal chest pain, myalgia, infusion-site reaction (infusion-site extravasation, infusion-site pain, and infusion-site swelling), and infusion-related reaction. Adakveo’s recommended dosing is 5 mg/kg by intravenous infusion over a period of 30 minutes for a first infusion and again two weeks later, then once every four weeks thereafter. VOCs, vaso-occlusive crises; ITT, intent to treat; HL, Hodges-Lehmann; CI, confidence interval; ER, emergency room. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Novartis Pharmaceuticals Corp; July 2013. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. color: #d57800 !important; “We are pleased to help reimagine medicine together with the sickle cell community and offer new hope for fewer VOCs.”. The FDA previously granted Adakveo orphan drug and breakthrough therapy status, and its approval review was given priority. Other Clinically Important Adverse Reactions. Sickle Cell Disease Association of America, nonsteroidal anti-inflammatory drugs (NSAIDs), University of Tennessee Health Science Center, It’s Difficult to Manage Sickle Cell Pain at Home During a Pandemic, VEST Lowers Duration of Painful SCD Crises, Why It’s Important for Everyone to Prioritize Their Health, Heat Waves Can Exacerbate My Sickle Cell Symptoms, Finding My ‘New Normal’ as Lockdown Restrictions Ease, Here Are Ways to Help Friends with Sickle Cell Disease. All rights reserved. Adakveo’s recommended dosing is 5 mg/kg by intravenous infusion over a period of 30 minutes for a first infusion and again two weeks later, then once every four weeks thereafter. “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 of Novartis Oncology. margin-left: 0px !important; Results are based on 2 post hoc analyses of the key secondary end point and are observational in nature; as such, they were not powered to show statistical significance. Note: REQUIRES PRECERTIFICATION Precertification of crizanlizumab (Adakveo) is required of all Aetna participating providers and members in applicable plan designs. color: #333 !important; Tagged Adakveo, crizanlizumab, FDA approval, Novartis, SUSTAIN study, vaso-occlusive crises, VOCs. The trial showed that Adakveo lowered the median annual rate of VOCs to 1.63 compared to 2.98 for the placebo group, a reduction of 45%. font-size: 11px !important; Data on file. Enrolled patients, ages 16 and older, were randomly assigned either to an intravenous infusion of Adakveo at one of two doses (2.5 mg/kg or 5 mg/kg) or to a placebo every other week for the first weeks, and then every four weeks over about one year of treatment. Transcranial Doppler Ultrasound Screening, Pain Management Guidelines for Sickle Cell Disease. When needed, estimate platelet count via peripheral blood smear. The study’s main goal was to evaluate the impact of treatment on the frequency of annual VOCs requiring a visit to a medical facility and treatment with either opioids or nonsteroidal anti-inflammatory drugs (NSAIDs) to control pain. This site is for US health care professionals only. This trial’s purpose is to find an appropriate dose for pediatric patients; study site and contact information can be found here. Reductions in annual VOCs were observed in all treated patients, regardless of the type of mutations they carried or prior treatment with hydroxyurea. line-height: 1.2; Full prescribing information for Adakveo, including recommended dosing schedules and safety warnings, can be found here. .uso-brand-details h3.brandColor { Sickle cellrelated pain crises are the primary cause of health care encounters in patients with sickle cell disease.1 These crises result in a decrease in quality of life2 and an increase in the risk of death.3 Crises are th… For precertification of crizanlizumab (Adakveo), call (866) 752-7021, or fax (866) 267-3277. “The stories we have heard from patients about their sickle cell pain crises are devastating,” Schaffert added. sup { ADAKVEO should only be used during pregnancy if the expected benefit to the patient justifies the potential risk to the fetus. Initial Therapy • Patient is 16 years of age or older; and Effect of crizanlizumab on pain crises in subgroups of patients with sickle cell disease: a SUSTAIN study analysis. The most common adverse reactions (≥10%) were nausea,arthralgia, back pain,andpyrexia. Key secondary goals included the number of days patients remained hospitalized due to VOCs within a year, and the time until participants experienced a first VOC as well as the percentage without any VOCs throughout the study. Table 1:Adverse Reactions(≥10%)in PatientsReceiving ADAKVEO With a Difference Between Arms of >3% Compared to Placebo in SUSTAIN Adverse Reactions ADAKVEO 5 mg/kg N = 66 n (%) Placebo N= 62 n (%) IMPORTANT SAFETY INFORMATION AND INDICATION. In the SUSTAIN clinical trial, infusion-related reactions (defined as occurring within 24 hours of infusion) were observed in 2 (3%) patients treated with ADAKVEO 5 mg/kg. Member is not concurrently enrolled in a clinical trial to receive an experimental therapy for SCD e. Dose does not exceed the following: i. These analyses should be interpreted with caution as the data are from a noncomparative post hoc subgroup analysis and not powered to detect statistical significance or superiority of ADAKVEO over placebo. “Adakveo is the first targeted therapy approved for sickle cell disease, specifically inhibiting selectin, a substance that contributes to cells sticking together and leads to vaso-occlusive crisis,” Richard Pazdur, MD, director of the Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the Center for Drug Evaluation and Research, said in an agency press release. Or click OK to continue. “We are excited to have a new medicine that may help many of the thousands of people.”. 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. Data on file. By inhibiting the activity of P-selectin, Adakveo helps to stop red blood cells from adhering to blood vessel walls, allowing for better blood flow and working to prevent inflammation and pain. Date: 05 Dec 2019. You are now leaving the Novartis site and moving to an external website independently operated and not managed by Novartis Pharmaceuticals Corporation. The per-protocol population was analyzed according to the randomized treatment arm and not powered to show statistical significance. References: 1. It is not known if Adakveo is safe and effective in children under 16 years of age. ADAKVEO (Crizanlizumab-tmca) 12 Version date: April 2, 2018 1 Executive Summary Product Introduction Crizanlizumab-tmca (ADAKVEO®, SEG101) is a humanized IgG2 kappa monoclonal antibody that binds to P-selectin and blocks interactions with its ligands including P-selectin glycoprotein ligand 1. .uso-brand-details h4 { ADAKVEO® is indicated to reduce the frequency of vasoocclusive crises (VOCs) in adults and pediatric patients aged 16 years and older with sickle cell disease. If ADAKVEO is administered within 2 weeks after the missed dose, continue dosing according to the patient's original schedule. to ADAKVEO. Aetna considers crizanlizumab-tmca (… Results are based on a noncomparative post hoc analysis and are observational in nature; as such, they are not powered to show statistical significance. Adakveo [prescribing information]. In the SUSTAIN clinical trial, infusion-related reactions (defined as occurring within 24 hours of infusion) were observed in 2 (3%) patients treated. For information on site of service for crizanlizumab (Adakveo), see Utilization Management Policy on Site of Care for Specialty Drug Infusions. font-weight: normal; Kutlar A, Kanter J, Liles DK, et al. ADAKVEO® (crizanlizumab-tmca) is indicated to reduce the frequency of vaso-occlusive crises (VOCs) in adults and pediatric patients, aged 16 years and older, with sickle cell disease. -->, Reduction in the frequency of VOCs was observed regardless of VOC baseline frequency, genotype, OR hydroxyurea use1,2, Reduction in THE FREQUENCY OF VOCs was observed regardless of VOC baseline frequency, genotype, OR hydroxyurea use1,2, The subgroup analyses should be interpreted with caution because the data are from a prespecified, noncomparative subgroup analysis of the primary end point and are not powered to detect statistical significance or superiority of ADAKVEO over placebo.3, VOCs, vaso-occlusive crises; ITT, intent to treat; HL, Hodges-Lehmann; CI, confidence interval; ER, emergency room. Novartis Pharmaceuticals Corp; February 2020. Novartis Pharmaceuticals Corp; 2016. The most common side effects of treatment included joint pain (18%), nausea (18%), back pain (15%), and fever (11%). 2. Join us at www.hcp.novartis.com—our new location for health care professionals to find product, access, and medical information. Adakveo’s use also reduced the number of days patients remained hospitalized due to VOCs, by 42% compared to the placebo; a median of 4.0 days that year for the 67 patients treated with Adakveo at 5 mg/kg, and 6.87 days/year for those given a placebo. 2 DOSAGE AND ADMINISTRATION 2.1 Recommended Dosage - Administer ADAKVEO 5 mg/kg by intravenous infusion over a period of 30 minutes at Week 0, Week 2, and every 4 weeks thereafter. Data on file. “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. line-height: 1.2; The CHMP opinion was 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), equivalent to a 45% reduction. Crizanlizumab for the prevention of pain crises in sickle cell disease. Laboratory Test Interference: Platelet Counts. An open-label Phase 2 trial (NCT03474965) of crizanlizumab in up to 100 children with sickle cell disease who have at least one VOC in the past year is now recruiting infants and children, starting at age 6 months, at sites in the U.S., Canada, Europe, and elsewhere. 6. 46% of patients treated with ADAKVEO were not hospitalized during the trial vs 35% with placebo (31 of 67 patients vs 23 of 65 patients), Median time to first hospitalization of 6.3 months with ADAKVEO vs 3.2 months with placebo, 22% of patients who completed the 12-month treatment period did not experience a VOC with ADAKVEO vs 8% with placebo (15 of 67 patients vs 5 of 65 patients; OR=3.57; 95% CI: 1.20, 10.63). Initial: 5 mg/kg at week 0 and week 2 ii. font-family: arial, sans-serif !important; Now you can find the tools you need to help manage your patients, all in one place! Copyright © 2020 Novartis Pharmaceuticals Corporation. According to the manufacturer’s safety information for Adakveo, the most common adverse reactions (>10% incidence) include nausea, joint pain, back pain, and fever. font-weight: bold; Run blood samples within 4 hours of blood collection or collect blood samples in tubes containing citrate. font-size: 16px !important; * The study included 198 patients evaluated at 60 study centers, the majority of which were located in the United States (51 centers with 151 study patients)1-3†, Prespecified, noncomparative subgroup analysis evaluated the annual rate of VOCs with or without hydroxyurea. The range of days hospitalized for the ADAKVEO and placebo population was 0 to 131 days and 0 to 307 days, respectively.3, ADAKVEO achieved a clinically meaningful reduction in the median annual rate of days hospitalized1,2. A first targeted treatment for sickle cell disease (SCD) — Adakveo (crizanlizumab), by Novartis, that works to lower the frequency of the painful vaso-occlusive crises (VOCs) common in this disease — has been approved by the U.S. Food and Drug Administration (FDA). Sickle Cell Disease News is strictly a news and information website about the disease. Eligible patients were diagnosed with sickle cell disease (any genotype including HbSS, HbSC, HbS beta 0-thalassemia, HbSbeta+thalassemia, and others). Topics: Haematologic malignancies . } The approval was based on the results of the 52-week SUSTAIN trial. Sickle cell disease is characterized by the presence of sickle hemoglobin (HbS), chronic hemolysis, recurrent pain episodes (called sickle cellrelated pain crises or vaso-occlusive crises), multiorgan dysfunction, and early death. Am J Hematol. The CHMP opinion was based on results of the 52-week, randomized, placebo-controlled SUSTAIN trial, which showed that Adakveo significantly lowered the median annual rate of … If a dose is missed, administer ADAKVEO as soon as possible. Novatis put Adakveo’s wholesale list price at$ $2,357 a vial, and estimates are that most patients will need three or four vials for each month’s treatment. The SUSTAIN study, conducted primarily in the United States, evaluated clinically meaningful end points across a diverse patient population aged 16 years and over1,2. Basel, October 30, 2020 — Novartis today announced that the European Commission (EC) has approved Adakveo (crizanlizumab) for the prevention of recurrent vaso-occlusive crises (VOCs), or pain crises, in patients with sickle cell disease aged 16 years and older. ADAKVEO® (crizanlizumab-tmca) is indicated to reduce the frequency of vaso-occlusive crises (VOCs) in adults and pediatric patients, aged 16 years and older, with sickle cell disease. Be the first to rate this post. A greater expanse of time until a first pain crisis was also seen in treated patients against placebo, 4.1 months vs. 1.4 months, and 36% of those given Adakveo had no VOC during the study compared to 17% of those on placebo, again at the 5 mg/kg dose, which is now the recommended dose. EC approval for Adakveo is based positive results from SUSTAIN trial. 4. Adakveo should be administered by a healthcare professional and may be given with or without hydroxyurea. You’ll have access to the site in a moment, or click continue to close this message and advance directly to the site. 2017;376(5):429-439. Table 1summarizes the adverse reactionsin the SUSTAIN trial. } font-family: arial !important; margin-top: 20px !important; 3. Note: Site of Care Utilization Management Policy applies. This banner text can have markup.. web; books; video; audio; software; images; Toggle navigation Novartis assumes no responsibility for the site. The efficacy of ADAKVEO® (crizanlizumab-tmca) was evaluated based on the annual rate of VOCs in patients (16 to 63 years of age) with SCD in a pivotal, phase 2, 52-week, randomized, multicenter, placebo-controlled, double-blind study. 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. .uso-brand-details .footnotes { 7. Novartis Pharmaceuticals Corp; November 2019. } Adakveo, a man-made antibody, works to block the activity of P-selectin, an adhesion protein that makes sickled blood cells more sticky and more likely to clog blood vessels, causing inflammation and pain crises. The CHMP opinion was based on results of the 52-week, randomized, placebo-controlled SUSTAIN trial, which showed that Adakveo significantly lowered the median annual rate of … N Engl J Med. In the SUSTAIN clinical trial, infusion-related reactions (defined as occurring within 24 hours of infusion) were observed in 2 (3%) patients treated with ADAKVEO 5 mg/kg. Medical Information  |  Contact Us  |  Non-US Residents  |  Interest-Based Ads. 2019;94(1):55-61. The SUSTAIN Study Video Summary of Original Article Feb 02, 2017 Crizanlizumab for the Prevention of Pain Crises in Sickle Cell Disease K. I. Ataga and Others In addition, interference with Study NCT01895361. $7,071 to $9,428 per month or between $84,852 and $113,135 each year. The most frequently reported adverse reactions (≥10%) in patients treated with ADAKVEO were nausea (18%), arthralgia (18%), back pain (15%), and pyrexia (11%). Never disregard professional medical advice or delay in seeking it because of something you have read on this website. Her work has been focused on the impact of non-canonical Wnt signaling in the collective behavior of endothelial cells — cells that made up the lining of blood vessels — found in the umbilical cord of newborns. Monitor patients for signs and symptoms of infusion-related reactions, which may include fever, chills, nausea, vomiting, fatigue, dizziness, pruritus, urticaria, sweating, shortness of breath or wheezing. “The approval of crizanlizumab is an important advancement for people living with this very difficult condition.”. Click here to subscribe to the Sickle Cell Anemia News newsletter! Adakveo is indicated to treat adults and adolescents, starting at age 16, and should soon be available to patients in the U.S., Novartis said in its press release. If you do not wish to leave this site, click Cancel. Basel, October 30, 2020 — Novartis today announced that the European Commission (EC) has approved Adakveo (crizanlizumab) for the prevention of recurrent vaso-occlusive crises (VOCs), or pain crises, in patients with sickle cell disease aged 16 years and older. 5. SCD, sickle cell disease; NSAIDs, non-steroidal anti-inflammatory drugs. Watch Nirmish R. Shah, MD, describe the clinical data for ADAKVEO, and a hypothetical patient case study. Watch Ifeyinwa (Ify) Osunkwo, MD, MPH, discuss key points of the pivotal trial design to help you determine the clinical profile of ADAKVEO. Advise pregnant women of the potential risk to a fetus. Margarida graduated with a BS in Health Sciences from the University of Lisbon and a MSc in Biotechnology from Instituto Superior Técnico (IST-UL). CHMP granted positive opinion in July this year, based on results from the 52-week, randomised, placebo-controlled SUSTAIN trial. The CHMP opinion was 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), equivalent to a 45% reduction. The approval follows a positive opinion by the Committee for Medicinal Products for Human Use (CHMP) in July based on results of the SUSTAIN trial, which showed that Adakveo significantly lowered the median annual rate of VOCs to 1.63 vs 2.98 compared with placebo (P=.010), equivalent to a 45% reduction. We’re moving on! 1 Adakveo can be given as an add-on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients for whom HU/HC is …