The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has adopted a positive opinion for Signifor® (SOM230, pasireotide) for the treatment of Cushing’s disease.
“We are pleased with the decision by the CHMP in support of pasireotide in the European Union,” said Hervé Hoppenot, President, Novartis Oncology.
“We are now one step closer to being able to offer patients in Europe the first approved medical treatment for Cushing’s disease.”
In the EU, the European Commission generally follows the recommendations of the CHMP and delivers its final decision within three months of the CHMP recommendation.
The decision will be applicable to all 27 EU member states plus Iceland and Norway.
Pasireotide has orphan drug designation for Cushing’s disease, a condition which affects no more than five in 10,000 people in the EU, the threshold for orphan designation.
The CHMP positive opinion is based on data from the Phase III PASPORT-CUSHINGS (PASireotide clinical trial PORTfolio – CUSHING’S disease) trial, the largest randomised study to evaluate a medical therapy in patients with Cushing’s disease.
In the study, patients were randomised to receive pasireotide subcutaneous (sc) injection in doses of 900µg and 600µg twice daily.
For the 900µg group, the study met the primary endpoint of normalising urinary-free cortisol (UFC) levels, the key measure of biochemical control of the disease.
Urinary-free cortisol levels were normalised in 26.3% and 14.6% of patients randomised to receive pasireotide 900µg and 600µg twice daily, respectively, at six months of treatment.
After 12 months of treatment, results confirmed the durability of the effect.
On average, as UFC levels were reduced, clinical manifestations of Cushing’s disease improved including reduction of blood pressure, total cholesterol, weight and body mass index.
The most frequently reported adverse events (AE) (>10%) by investigators for pasireotide were diarrhoea, nausea, hyperglycaemia, cholelithiasis, abdominal pain, diabetes mellitus, injection site reactions, fatigue and increased glycosylated haemoglobin (HbA1c), with most events being Grade 1-2.
The tolerability profile of pasireotide was similar to that of other somatostatin analogues with the exception of the greater degree of hyperglycaemia.