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EMA recommends approval for Novartis Cushing’s disease drug Signifor

Puts it inline to become the first approved drug for the rare, life-threatening illness

Novartis' Cushing's disease drug Signifor (pasireotide) has been recommended for approval by European regulatory advisors.

The positive opinion from the Committee for Medicinal Products for Human Use (CHMP) puts it in line to be the first approved drug for the rare, life-threatening illness in Europe, which involves a small tumour of the pituitary gland making too much of the hormone adrenocorticotrophin.

If approved Signifor, a somatostatin analogue, would also provide a treatment option for Cushing's patients who cannot have surgery or for whom surgery has not been successful.

CHMP recommendations are usually translated into full European approval within three months.

"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."

The condition causes an individual's adrenal glands to both grow and release excessive amounts of cortisol in the blood, leading to a series of symptoms, including weight gain, bruising, weakening of the muscles and bones and high blood pressure.

Due to its rarity in Europe – where it affects 0.4 in 10,000 people - Signifor has orphan drug status, giving Novartis incentives to development the treatment, such as fee reductions.

Signifor works by attaching itself to somatostatin receptors in the affected pituitary gland, blocking the release of adrenocorticotrophin. This aims to lower levels of cortisol to a safer concentration.

The drug's efficacy was demonstrated in Novartis' phase III PASPORT-CUSHINGS trial, with a 900μg dose halving levels of cortisol in the urine of 41 per cent of patients.

A similar reduction was recorded in 34 per cent of patients taking a 600μg dose.

Safety of the drug was reported as being similar to other approved somatostatin analogues, although adverse events such as diarrhoea, nausea, hyperglycaemia and diabetes mellitus were all reported.

As such, a patient's blood sugar levels need to be assessed before treatment with Signifor and patients taking the drug need to be monitored for hyperglycaemia.

Meanwhile, another treatment for Cushing's disease, Laboratoire HRA Pharma's ketoconazole, was given orphan drug designation in the January 2012 list of orphan drug recommendations from the EMA.

23rd January 2012

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