Swiss pharmaceutical giant Novartis AG (NVS) Tuesday said the US Food and Drug Administration or FDA has approved Signifor long-acting release or LAR to treat patients with acromegaly, a rare and life-threatening hormonal disorder, who have had an inadequate response to surgery and/or for whom surgery is not an option.
Acromegaly is a rare endocrine disorder caused by elevated growth hormone and insulin-like growth factor-1 levels. The company noted that Signifor LAR, a next-generation somatostatin analog or SSA, provides a new option for patients with acromegaly with inadequately controlled disease.
Signifor LAR (pasireotide) for injectable suspension, for intramuscular use, is an SSA administered intramuscularly once-monthly that exerts its pharmacological activity via binding to somatostatin receptors.
This FDA approval was based on two multicenter Phase III studies, C2305 and C2402. These studies respectively examined medically naïve patients who have had prior surgery or for whom surgery was not an option and patients with acromegaly inadequately controlled on first generation SSAs.
Signifor LAR has been found effective in both medically naïve patients with acromegaly who have had prior surgery or for whom surgery was not an option, and patients whose disease is not fully controlled on first generation SSAs.
In both studies, higher rates of full biochemical control were achieved with Signifor LAR compared to a first generation SSA.
According to the firm, the approval of Signifor LAR helps address a critical unmet need among the acromegaly patient population.
Monica Gadelha, Professor, Federal University of Rio de Janeiro and pivotal trial study author, said, "Treating acromegaly can be extremely challenging and the consequences of inadequate normalization of hormone levels can be serious for patients. With the approval of Signifor LAR, physicians now have a new acromegaly therapy that provides an enhanced mechanism to address elevated hormone levels."
In the US, Signifor LAR has orphan drug designation for acromegaly. Orphan drug designation is granted for products that treat a condition that affects fewer than 200,000 people in the US. In November 2014, the European Medicines Agency or EMA approved Signifor to treat adult patients with acromegaly for whom surgery is not an option or has not been curative and who are inadequately controlled on treatment with a first-generation SSA.
Novartis has also submitted additional regulatory applications for Signifor LAR worldwide.
The safety and efficacy profile of Signifor LAR has not yet been established in countries outside the US or the EU in patients with acromegaly.
On the NYSE, Novartis shares closed Monday's trading at $91.64, down 2.01 percent.
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