Novartis sickle cell medicine Adakveo® approved in Europe to prevent recurrent vaso-occlusive crises

• Adakveo is the first targeted sickle cell disease therapy for prevention of recurrent vaso-occlusive crises (VOCs) available for use in Europe
  
  
• Clinical data showed that use of Adakveo led to a significant reduction in the rate of VOCs and to fewer days spent in hospital
  
  
• VOCs disrupt patients’ lives physically, socially, and emotionally – and can increase risk of organ damage and early death
  
  

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.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 inappropriate or inadequate.

Adakveo binds to P-selectin, a cell adhesion protein that plays a central role in the multicellular interactions that can lead to vaso-occlusion.^2,3 Sickle cell disease is considered a rare blood condition, affecting about 50,000 people in Europe.^4,5

“Data shows that nine out of ten people living with sickle cell disease experience one or more VOCs in a year, with a third of those crises leading to hospitalization, underscoring the significant unmet need among a vulnerable group of patients,” said Kees Roks, Head Region Europe, Novartis Oncology. “Just one VOC could be catastrophic for the patient, so preventing these sudden, unpredictable and life-threatening events is hugely important. Today’s decision gives people living with sickle cell disease a chance to achieve that goal.”

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. There was also a greater than two-fold increase in the proportion of patients with no VOCs who completed the study, compared to placebo. Reductions in the frequency of VOCs were observed among patients regardless of sickle cell disease genotype and/or hydroxyurea/hydroxycarbamide (HU/HC) use. In the same study, Adakveo was shown to reduce the median annual rate of days hospitalized by 42% (4.0 days for Adakveo vs. 6.87 days for placebo).⁶ Read more about the positive CHMP opinion and the SUSTAIN clinical trial results here.

About Adakveo

Adakveo (crizanlizumab) – previously known as SEG101 – is indicated for the prevention of recurrent VOCs in patients with sickle cell aged 16 years and older. It can be given as an add-on therapy to HU/HC or as monotherapy in patients for whom HU/HC is inappropriate or inadequate. 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.

Adakveo is now approved in 36 countries around the world including the United States and European Union member states.

Important Safety Information

Adakveo may cause serious side effects, including infusion-related reactions. Infusion-related reactions may happen within 24 hours of receiving an infusion of Adakveo. Patients should tell their doctor or nurse immediately if they experience any of the following, which may be signs and symptoms of an infusion-related reaction, such as fever, chills or shivering, nausea, vomiting, tiredness, dizziness, sweating, hives, itching, shortness of breath or wheezing, or pain. In the event of a severe reaction, crizanlizumab should be discontinued and appropriate therapy should be instituted.

The most common side effects (incidence > 10%) were arthralgia, nausea, back pain, pyrexia, and abdominal pain. Other side effects which may affect up to 1 in every 10 people are diarrhea, itching (including vulvovaginal itching), vomiting, muscle pain (myalgia), pain in the muscles or bones of the chest (musculoskeletal chest pain), sore throat (oropharyngeal pain), and redness or swelling and pain at the site of the infusion.

Adakveo may interfere with a laboratory test used to measure the number of platelets in the blood. Patients should tell their doctor or nurse that they are on treatment with Adakveo. It is recommended to run the tests as soon as possible (within 4 hours of blood collection) or use tubes containing citrate.

It is preferable to avoid the use of Adakveo during pregnancy and in women of childbearing potential not using contraception.

Disclaimer
This media update 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,” “may,” “could,” “would,” “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 media update, 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 media update 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, economic and business conditions, including the effects of and efforts to mitigate pandemic diseases such as COVID-19; safety, quality, data integrity 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 media update as of this date and does not undertake any obligation to update any forward-looking statements contained in this media update as a result of new information, future events or otherwise.

About Novartis
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 nearly 800 million people globally and we are finding innovative ways to expand access to our latest treatments. About 110,000 people of more than 140 nationalities work at Novartis around the world. Find out more at
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References

1. Adakveo (crizanlizumab) prescribing information. East Hanover, New Jersey, USA. Novartis Pharmaceuticals Corporation; November 2019.
2. Rees DC, Williams TN, Gladwin MT. Sickle-cell disease. Lancet. 2010;376(9757):2018-2031.
3. 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.
4. European Medicines Agency. Crizanlizumab Orphan Designation. August 2012. Available at: https://www.ema.europa.eu/en/medicines/human/orphan-designations/eu3121034.
5. Roberts I, de Montalambert M. Sickle cell disease as a paradigm of immigration haematology: new challenges for hematologists in Europe. Haematologica. 2007;92(7):865-871.
6. 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.

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