US FDA Ushers In New Drug To Tame SCD

US FDA Ushers In New Drug To Tame SCD

***Sickle cell’s best known drug, Hydroxyurea, can have serious side effects and requires weekly blood tests. It does not work for everyone with sickle cell anaemia …

***Crizanlizumab may be prescribed alone or in combination with other approved SCD medications …

***Safety and Effectiveness not evaluated in children below 16 years

***Costs $85000 to $113000 per annum

***Not known if it could harm an unborn baby

New Novartis medicine Adakveo® (crizanlizumab-tmca) 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

East Hanover, NJ, November 15, 2019 – Novartis announced today that the US Food and Drug Administration (FDA) approved Adakveo® (crizanlizumab-tmca), 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 Adakveo

Adakveo® (crizanlizumab-tmca) – 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

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 Patient.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® (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.