Switzerland-based Novartis International has received European Union (EU) approval for its Signifor (pasireotide) drug, used as a medication to treat adult patients with Cushing's disease for whom surgery is not an option or for whom surgery has failed.
Signifor is a multireceptor targeting somatostatin analog (SSA) that binds with high affinity to four of the five somatostatin receptor subtypes, and is now being evaluated as a long-acting release (LAR), once-monthly intramuscular injection as part of a global Phase III programme.
The approval is backed by a prospective randomised double-blind Phase III Pasireotide clinical trial Portfolio-Cushing's disease (PASPORT-CUSHINGS) study that assessed the efficacy and safety of Signifor in 162 adult patients with urinary-free cortisol (UFC) levels greater than 1.5 times the upper limit of normal (ULN).
Of the 162 patients treated with Signifor 900ug and 600ug subcutaneous injection twice daily, respectively, at month six, the mean UFC levels were normalised in 26.3% and 14.6%.
The study's primary endpoint, the proportion of patients who achieved normalisation of UFC after six months without dose up-titration relative to randomised dose, was met in patients treated with 900ug twice daily.
At six and 12 months, patients with both full and partial mean UFC control showed overall reductions in the clinical manifestations of Cushing's disease, including blood pressure, total cholesterol, weight and body mass index.
Novartis Oncology president Herve Hoppenot said that as the first therapeutic option to specifically target Cushing's disease, Signifor has the potential to redefine treatment of the debilitating disease.
"By focusing research efforts on our understanding of this rare disease where there is significant unmet need, we have been able to successfully bring a novel treatment option to patients in the European Union," Hoppenot added.
The decision follows the positive opinion from the Committee for Medicinal Products for Human Use (CHMP) and applies to all 27 EU member states, including Iceland and Norway.
Image: Novartis headquarters in Basel, Switzerland. Photo: Andrew.