Novartis' acromegaly therapy cleared by FDA

A long-acting version of Novartis' Signifor has been approved by the FDA to treat the growth disorder acromegaly, just weeks after it was given a green light in Europe.

The US regulator has cleared Signifor LAR (pasireotide) for the treatment of patients with acromegaly who have had an inadequate response to surgery or for whom surgical intervention is not an option.

Acromegaly is caused by a non-cancerous tumour in the pituitary gland that causes excessive secretion of growth hormone (GH), which in turn leads to elevated levels of insulin-like growth factor-1 (IGF-1). It leads to an increase in bone size, particularly those in the hands, feet and face, alongside other complications such as diabetes, arthritis, kidney disease and cancer.

Recent estimates put the prevalence of acromegaly at between 115 and 295 cases per million, around double earlier estimates because of a higher-than-thought prevalence of pituitary adenomas.

For years, acromegaly has been managed using long-acting formulations of somatostatin analogues such as Novartis' own Sandostatin LAR (octreotide) and Ipsen's Somatuline Autogel (lanreotide), which are used to reduce levels of GH and IGF-1.

In trials, Signifor LAR showed superior efficacy to older somatostatin analogues, as measured by higher rates of biochemical control of GH and IGF-1 levels.

"Treating acromegaly can be extremely challenging and the consequences of inadequate normalisation of hormone levels can be serious for patients," said Monica Gadelha of the Federal University of Rio de Janeiro in Brazil, one of the lead clinical investigators in the pivotal trials.

"Physicians now have a new acromegaly therapy that provides an enhanced mechanism to address elevated hormone levels," she added.

A short-acting formulation of Signifor that requires twice-daily dosing is already approved to treat Cushing's disease, but was not considered commercially viable to treat acromegaly given the availability of rival depot formulations. Signifor LAR needs to be given just once a month, allowing it to compete head-to-head with its rivals in this setting.

Novartis' Sandostatin franchise has long been a money-spinner for the pharma company, bringing in $1.24bn in the first three months of 2014, a 5% increase on the year-earlier period despite losing patent protection in many world markets. Novartis has defended the franchise with the roll out of a new, patient-friendly delivery device.

In January, the Italian authorities opened an investigation to assess whether Novartis and Italfarmaco had colluded on the supply of long-acting octreotide to prevent competition in tenders.