Novartis gains FDA approval for Signifor LAR to treat patients with acromegaly, a rare and life-threatening hormonal disorder
Novartis has announced that the US Food and Drug Administration (FDA) has approved Signifor® long-acting release (LAR)* (pasireotide) 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. The approval of Signifor LAR, a next-generation somatostatin analog (SSA), helps address a critical unmet need among the acromegaly patient population. Signifor LAR has been studied and 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 SSAs2.
Acromegaly is a rare, debilitating endocrine disorder caused by the excess production of growth hormone (GH) and insulin-like growth factor-1 (IGF-1)1,3. 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 features1. Acromegaly is also associated with two- to three-fold increased mortality rates and serious health complications, including heart disease, hypertension, diabetes, arthritis and colon cancer1,4,5. In fact, heart disease is responsible for approximately 60% of deaths among people with acromegaly6.
“Treating acromegaly can be extremely challenging and the consequences of inadequate normalization of hormone levels can be serious for patients,” said Dr. Monica Gadelha, Professor, Federal University of Rio de Janeiro and pivotal trial study author. “With the approval of Signifor LAR, physicians now have a new acromegaly therapy that provides an enhanced mechanism to address elevated hormone levels. This is a significant achievement and much welcomed news for patients with acromegaly.”
Worldwide, the prevalence of acromegaly is estimated to be 60 cases per million, with an annual incidence of 3 to 4 new cases per million1. However, recent studies suggest that pituitary adenomas may be more prevalent than previously thought, and that the prevalence of acromegaly may be between 115 and 295 cases per million3. On average, patients experience a delayed diagnosis of 6 to 10 years from disease onset7. 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 levels8. 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-19. Reduction of tumor volume and minimization of clinical manifestations are other important treatment goals8.
1. Acromegaly. National Institutes of Health. National Institute of Diabetes and Digestive and Kidney Diseases. 2008 May; 8(3924): 1-10.
2. Signifor® LAR Prescribing Information. East Hanover, New Jersey, USA: Novartis Pharmaceuticals Corporation; December 2014.
3. Rosario P.W. Frequency of Acromegaly in Adults with Diabetes or Glucose Intolerance and Estimated Prevalence in the General Population. Pituitary. 2011; 14: 217-221.
4. Holdaway I.M. et al. Factors Influencing Mortality in Acromegaly. J Clin Endocrin Metab. 2004; 89(2): 667- 674.
5. Holdaway I.M. et al. A Meta-Analysis of the Effect of Lowering Serum Levels of GH and IGF-1 on Mortality in Acromegaly. European Journal of Endocrinology. 2009; 159: 89-95.
6. Colao, et. al. Systemic Complications of Acromegaly: Epidemiology, Pathogenesis, and Management. Endocrine Reviews. 2004; 25:102-152.
7. Schneider H et al. A Novel Approach to the Detection of Acromegaly: Accuracy of Diagnosis by Automatic Face Classification. J Clin Endocrin Metab. 2011; 96: 2074-2080.
8. Katznelson L et al. Acromegaly: An Endocrine Society Clinical Practice Guideline. J Clin Endocrinol Metab. 2014; 99(11):3933-3951.
9. Carmichael J.D. et al. Acromegaly Clinical Trial Methodology Impact on Reported Biochemical Efficacy Rates of Somatostatin Receptor Ligan Treatments – a Meta-analysis. J Clin Endocrin Metab. 2014; 99:1825- 1833.
10. FDA Website. “How to Apply for Orphan Product Designation.” Available at: http://www.fda.gov/ForIndustry/DevelopingProductsforRareDiseasesConditions/HowtoapplyforOrphanProductDesignation/ucm364750.htm. Accessed December 2014.
11. FDAWebsite.“PasireotideOrphanDesignation.”Availableat: http://www.accessdata.fda.gov/scripts/opdlisting/oopd/OOPD_Results_2.cfm?Index_Number=288609 ??Accessed December 2014.
* In the European Union, the long-acting release formulation of pasireotide for the treatment of acromegaly has been approved under the trade name Signifor®.
Source: Novartis Pharmaceuticals