MHRA withdraws approval of Adakveo for sickle cell disease

UK— The Medicines and Healthcare Products Regulatory Agency (MHRA) in the United Kingdom has revoked Novartis’ conditional marketing authorization for Adakveo, a sickle cell disease medication.

Adakveo gained conditional clearance in October 2020 to prevent recurrent vasoocclusive crises (VOCs) in sickle cell disease (SCD) patients.

Sickle cell disease is a hereditary ailment in which abnormal blood cells cause strokes, organ damage, extreme pain, and premature death.

Adakveo was developed as an add-on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients aged 16 years and older for whom HU/HC is unsuitable or insufficient.

Data supporting Adakveo’s effects were not considered thorough at the time of approval due to ambiguity regarding the extent of Adakveo’s effect.

The drug was therefore approved for sale on the condition that the manufacturer produce results from the STAND (CSEG101A2301), Phase III study1 to validate the treatment’s effectiveness and safety.

However, the Phase III research (STAND) of Adakveo in sickle cell disease patients with vaso-occlusive crises did not confirm its therapeutic advantage, and the decision by the Swiss manufacturer to recall a batch of the medicine has led to the withdrawal of the conditional marketing authorization in the UK.

Following this revocation, MRSA has advised health practitioners to discontinue the provision of the medicine batch, quarantine any remaining stock, and await contact from the Novartis Pharmaceuticals UK Limited customer service team, who will contact healthcare professionals directly to arrange a return.

Furthermore, clinicians and prescribers who have begun patients on Adakveo have been instructed to inform patients of the license revocation and explore other treatment options with them, and no new patients should be started on Adakveo in the UK.

The MHRA has recommended healthcare practitioners who receive inquiries from patients regarding the recall letter to refer them to their prescribing healthcare professional.

Patients who are currently on Adakveo, on the other hand, MRHA noted, will be contacted by their prescriber to explore alternate treatment choices.

Additionally, patients who have adverse reactions or have any queries about the medicine have been asked to seek medical assistance and report any suspected adverse reactions.

Earlier in May 2023, the European Medicines Agency (EMA) revoked Adakveo’s marketing authorization following a review by the EMA’s human medicines committee (CHMP) that decided the drug’s benefits did not exceed its dangers.

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