FDA approves Signifor LAR to treat patients with acromegaly, a rare and life-threatening hormonal disorder

January 15, 2015

FDA has approved long-acting release (LAR) pasireotide (Signifor, Novartis) for injectable suspension, for intramuscular use, for the treatment of patients with acromegaly who have had an inadequate response to surgery and/or for whom surgery is not an option.

FDA has approved long-acting release (LAR) pasireotide (Signifor, Novartis) for injectable suspension, for intramuscular use, for the treatment of patients with acromegaly who have had an inadequate response to surgery and/or for whom surgery is not an option.

Signifor LAR, a next-generation somatostatin analog (SSA) is administered intramuscularly once-monthly. The drug has been found effective in both medically naïve patients with acromegaly who have had prior surgery or for whom surgery was not an option, as well as patients whose disease is not fully controlled on first-generation SSAs.

FDA's approval was based on 2 multicenter phase 3 studies, C2305 and C2402, which 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. In both studies, higher rates of full biochemical control (defined as mean GH level <2.5 µg/L and normal IGF-1 levels) were achieved with Signifor LAR compared to a first-generation SSA

Acromegaly is a hormonal disorder that most commonly occurs in middle-aged men and women. The prevalence of acromegaly is approximately 4,676 cases per million population and the incidence is 116.9 new cases per million per year, according to Acomegaly.org.

It is caused by the excess production of growth hormone (GH) and insulin-like growth factor-1 (IGF-1]. In the majority of cases, the disease is caused by a non-cancerous tumor on the pituitary gland. Prolonged exposure to GH and IGF-1 may cause patients to experience extreme physical changes including the enlargement of hands, feet and facial features. Acromegaly is also associated with 2- to 3-fold increased mortality rates and serious health complications, including heart disease, hypertension, diabetes, arthritis and colon cancer. In fact, heart disease is responsible for approximately 60% of deaths among people with acromegaly.

There have been significant advances in the treatment of acromegaly in the past 20 years. Signifor LAR is the first new product to be approved in 7 years, according to Eric Althoff, head global media relations, Novartis International AG.

“Signifor LAR helps to address a critical unmet need among acromegaly patients who are unable to be treated by surgery or have not otherwise achieved disease control on current therapy,” Althoff said. “We are still working on developing the pricing and formularies for this product, which will reflect the value it brings to patients with acromegaly and society, the scientific innovation it represents and the necessary investment in clinical studies to support development of this innovative medicine.”

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On average, patients experience a delayed diagnosis of 6 to 10 years from disease onset. Once diagnosed, the primary objective when treating acromegaly is to achieve biochemical control of the disease, as measured by both the reduction of GH levels and normalization of IGF-1 levels. Notably, a recent meta-analysis using more sensitive assays and more stringent evaluation criteria showed that 45% of patients with acromegaly fail to achieve recommended levels of GH or normalized levels of IGF-1. Reduction of tumor volume and minimization of clinical manifestations are other important treatment goals.

Signifor LAR has orphan drug designation in the United States for acromegaly.

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