Adakveo is claimed to be the first targeted sickle cell disease therapy for prevention of recurrent vaso-occlusive crises (VOCs) in Europe
Novartis manufacturing facility in Stein, Switzerland. (Credit: Novartis AG.)
Novartis has secured the European Commission (EC) has approval for Adakveo (crizanlizumab) to treat patients with a type of sickle cell disease.
The EC approval indicated the drug for recurrent vaso-occlusive crises (VOCs), or pain crises, in sickle cell disease patients, aged 16 years and older.
Also, the regulatory approval allows the use of Adakveo as an add-on therapy to hydroxyurea/hydroxycarbamide (HU/HC) or as monotherapy in patients who are not eligible for HU/HC.
Adakveo works by binding to P-selectin, a cell adhesion protein that plays important role in facilitating interactions between endothelial cells, platelets, red blood cells, and leukocytes, which may lead to vaso-occlusion.
The Swiss drugmaker claimed that its Adakveo is the first and only targeted biologic that binds to P-selectin, to prevent vaso-occlusion in sickle cell disease.
The drug is currently approved in 36 countries, including the US and EU member states.
Novartis Oncology Europe region head Kees Roks said: “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.
“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.”
EC approval for Adakveo is based positive results from SUSTAIN trial
The EC approval is based on a positive opinion by the Committee for Medicinal Products for Human Use (CHMP).
CHMP granted positive opinion in July this year, based on results from the 52-week, randomised, placebo-controlled SUSTAIN trial.
The clinical trial demonstrated that Adakveo significantly reduced the median annual rate of VOCs by nearly 45%, compared with placebo.
The proportion of patients who completed the study with no VOCs has increased by more than two times, compared to placebo.
Also, the reduction in the frequency of VOCs was observed among patients regardless of sickle cell disease genotype or use of hydroxyurea/hydroxycarbamide (HU/HC), said the company.
Furthermore, the study also showed that treatment using Adakveo reduced the median annual rate of hospitalised days by 42%.