MHRA withdraws marketing authorisation for Crizanlizumab in a setback for the Sickle Cell community

In disappointing news for the Sickle Cell Community, the Medicines and Healthcare products Regulatory Agency (MHRA) last week announced that it was withdrawing the marketing authorisation for Crizanlizumab (Adakveo), the first new drug in over 20 years to be introduced for people living with Sickle Cell Disease (SCD). Sanius Health’s own groundbreaking research into quality of life (QoL) for SCD patients being treated with Crizanlizumab revealed significant improvements in QoL scores following treatment with the drug.

The communications released by the drug’s manufacturer Novartis on January 10th indicated that the Phase III study of Crizanlizumab had not provided evidence of the drug’s clinical benefit for SCD patients who experience vaso-occlusive pain crises (VOCs). Following a benefit-risk analysis performed by the MHRA, authorisation for the drug has been revoked, although there are no questions surrounding its safety.

The news represents a significant setback for the SCD community. While SCD is the UK’s fastest growing genetic condition, with almost 300 babies born with the condition each year, there remains only one treatment licensed by the NHS. As a condition that predominantly affects those with African or Caribbean heritage, SCD patients already face numerous obstacles in the way of proper access to care, including systemic racism and a lack of education amongst clinicians. Crizanlizumab was approved for use in 2020, and many considered it to be the dawn of a new era for the SCD community, marking significantly improved access to treatments. Crizanlizumab works by binding to a protein in the blood cells to prevent the restriction of blood and oxygen supply that leads to a VOC.

At Sanius, we have already harnessed our cutting-edge AI and patient ecosystem to examine how Crizanlizumab impacts SCD patients’ QoL. A majority of the patients who participated in the study reported an improvement in the severity of their SCD symptoms, while 60% of participants reported better QoL scores. The MHRA’s verdict demonstrates how there is a critical need for real-world data to provide deeper and more comprehensive data on the impacts of treatments on patients’ lives.

?As the graph above demonstrates, the gathering of real-world data indicates that although Crizanlizumab might not prevent the onset of a VOC, participants reported that the treatment contributed towards lower frequency, lower severity and shorter in length VOCs.

While last week’s news was disappointing, there remains hope that Casgevy and Voxelotor, two other approved therapies, will receive final approval for use in the NHS.

To learn more about the work that Sanius Health does, visit www.saniushealth.com