FDA Approves Pasireotide for Treating Acromegaly

Miriam E Tucker


December 17, 2014

The US Food and Drug Administration has approved the injectable long-acting somatostatin analog pasireotide (Signifor Long-Acting Release [LAR], Novartis) for the treatment of acromegaly in patients who have had an inadequate response to surgery or for those in whom surgery is not an option.

Pasireotide is already FDA-approved to treat Cushing's disease.

Acromegaly is a rare endocrine disorder characterized by excess skeletal growth, soft-tissue enlargement, and disfigurement resulting from excess production of growth hormone (GH) and insulinlike growth factor-1 (IGF-1), usually from a pituitary tumor.

Affected individuals undergo extreme physical changes, including the enlargement of hands, feet, and facial features, and acromegaly is also associated with two- to threefold increased mortality rates and serious health complications. These include heart disease — which is responsible for approximately 60% of deaths among people with acromegaly — hypertension, diabetes, arthritis, and colon cancer.

Surgery is first-line treatment but often is unsuccessful because of the late stage at which the disease is typically diagnosed.

The new approval was based on two multicenter phase 3 studies, one in medically naive acromegaly patients who had had prior surgery or in whom surgery was not an option, and the other in patients inadequately controlled on first-generation somatostatin analogs (octreotide [Sandostatin, Novartis Pharmaceuticals] and lanreotide [Somatuline, Ipsen Pharma]).

In both studies, higher rates of full biochemical control (defined as a mean GH of less than 2.5 μg/L and normal IGF-1 levels) were achieved with pasireotide LAR compared with a first-generation somatostatin analog.

Pasireotide is available as an injectable suspension in 20-mg, 40-mg, and 60-mg powder-filled vials, to be reconstituted with the provided 2-mL diluent. The initial dose is 40 mg by intramuscular injection once every 4 weeks.

The FDA label includes a warning about the risk of developing hyperglycemia and diabetes with pasireotide and advises that glucose levels be monitored periodically during therapy and glucose-lowering therapy be initiated if necessary.

Results from one of the phase 3 pasireotide LAR studies were published online September 24 in Lancet Diabetes & Endocrinology.

In an editorial accompanying that paper, Dr Nicholas A Tritos (Massachusetts General Hospital and Harvard Medical School, Boston) said that while pasireotide seems to be an exciting advance for the management of patients with acromegaly, improved efficacy with this agent will need to be balanced against the increased risk for hyperglycemia.

A better understanding of the risk factors associated with the development of hyperglycemia in response to this therapy should be a "goal of future studies," he said.

In November, the European Medicines Agency approved pasireotide LAR for the treatment of adult patients with acromegaly for whom surgery is not an option or has not been curative and for those who are inadequately controlled on treatment with a first-generation somatostatin analog.


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