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NICE recommends Novartis’ Adakveo for sickle cell disease

The treatment for sickle cell disease is the first in more than 20 years


Eligible patients in England and Wales will soon have routine access to Novartis’ Adakveo (crizanlizumab) for sickle cell disease (SCD) through a Managed Access Agreement (MAA).

The National Institute for Health and Care Excellence (NICE) has published the Final Appraisal Determination (FAD) recommending Adakveo as an option for preventing recurrent sickle cell crises in people aged 16 or older with sickle cell disease.

Recurrent sickle cell crises are deemed to be two or more vaso-occlusive crises (VOCs) in a year, that managed at home or in hospital. VOCs can increase risk of organ damage, costly hospitalisations and early death.

“Sickle cell crises are much more than unpredictable, painful events: recurrence is a constant worry that pervades every aspect of daily life,” said John James OBE, Chief Executive, Sickle Cell Society.

“For the thousands of people living with this complex and life-long disease in this country, this decision offers hope of a life with less disruption and stress from their condition, as well as reducing the associated costs to the NHS,” he added.

“Vaso-occlusive crises are acute episodes of pain that often last for days, and have a substantial impact on the quality of life of people living with sickle cell disease. These episodes can also lead to organ damage and significant long-term complications,” said Baba Inusa, Chair of the National Haemoglobinopathy Panel, professor of paediatric haematology at King’s College London and lead consultant for paediatric haemoglobinopathies, Evelina London Children’s Hospital, Guy’s and St Thomas’ NHS Foundation Trust.

“Today’s decision is an important milestone for the medical community: for the first time in 20 years, there will now be a new treatment regime that we can offer this community, which has gone for so long without additional support in managing their condition,” he added.

Article by
Iona Everson

7th October 2021

From: Research, Regulatory



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