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Novartis sickle cell drug Adakveo put on path to EU approval

Following the adoption of a positive opinion by the EMA's Committee for Medicinal Products for Human Use

Novartis announced last Friday that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) adopted a positive opinion recommending conditional marketing authorization of Adakveo® (crizanlizumab) for the prevention of recurrent vaso-occlusive crises (VOCs), or pain crises, in patients with sickle cell disease (SCD) aged 16 years and older.

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.

If approved by the European Commission, Adakveo will be the first targeted medicine available in Europe for the prevention of VOCs in patients with sickle cell disease. Though considered a rare condition, tens of thousands of people in Europe have sickle cell disease.

“The positive CHMP opinion for Adakveo underscores the potential of this new medicine to prevent recurrent sickle cell pain crises, which can affect all aspects of patients’ lives,” said Susanne Schaffert, PhD, President, Novartis Oncology. “Novartis is dedicated to innovation where there is significant unmet need, and we are grateful for the support we have received from the community of sickle cell patients, advocates and medical experts in Europe and around the world who continue to help us reimagine medicine for this devastating disease.”

The European Commission reviews the CHMP recommendation and usually delivers its final decision in approximately two months. In addition, the Committee for Orphan Medicinal Products is currently reviewing the maintenance of the orphan designation of Adakveo. Adakveo is currently approved in the United States and seven other countries for reducing the frequency of vaso-occlusive crises in patients with SCD aged 16 years and older.

Efficacy Proven in Clinical Studies

The CHMP opinion was 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), 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.6

Additional results from the SUSTAIN study include:

  • A decrease in the median annual rate of days hospitalized to 4 vs 6.87 days when compared with placebo (a 42% reduction)
  • Median time to first VOC, 4.1 months for Adakveo vs 1.4 months for placebo

Read the Full CHMP Summary of Positive Opinion for Adakveo HERE

Read More Information about Adakveo HERE

 

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