Signifor från Novartis godkänt i Europa för behandling av acromegali

Novartis announced today that the European Commission has approved Signifor® (pasireotide) as a new long acting release formulation for once monthly intramuscular injection 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 somatostatin analogue (SSA). Next-generation SSA Signifor offers the first alternative treatment option directly targeting the pituitary tumor for patients whose acromegaly remains inadequately controlled on currently available SSAs[5].

Acromegaly affects an estimated one to two in every 10,000 people in the EU[6]. In the majority of acromegaly cases, a non-cancerous tumor in the pituitary gland leads to excess production of growth hormone (GH) and, ultimately, insulin-like growth factor-1 (IGF-1) in the body[3]. Prolonged exposure to excess GH and IGF-1 may cause patients to experience significant physical changes including the enlargement of hands, feet, facial features, and internal organs[3]. Moreover, acromegaly patients who do not achieve biochemical control of their disease, as measured by GH and IGF-1 levels, may face serious health consequences such as heart disease, hypertension, diabetes, arthritis, colon cancer leading to an increased risk of death[3]. According to recent research, 45% of acromegaly patients fail to achieve the recommended levels of GH or normalized IGF-1 on current therapies[2].

”Acromegaly that is not properly controlled can have a devastating impact on the long-term health of patients living with this serious pituitary disorder,” said Bruno Strigini, President, Novartis Oncology. ”This first approval of Signifor in acromegaly marks a much needed advance in the treatment of this rare disease and we are working hard to bring this therapy to this underserved patient population worldwide in the near future.”

The approval is based on data from two multicenter Phase III studies, C2402 and C2305, which respectively evaluated patients with inadequately controlled acromegaly on first-generation SSAs, and medically naïve patients who were post-surgery, or newly diagnosed patients for whom surgery is contraindicated. Both studies showed Signifor to have superior efficacy in providing biochemical control, as measured by both GH and IGF-1 levels, compared to a first-generation SSA[5].

The EU approval follows a positive opinion adopted by the Committee for Medicinal Products for Human Use (CHMP) in September 2014 for Signifor for the treatment of acromegaly and applies to all 27 EU member states, plus Iceland and Norway[7]. Additional regulatory applications for the new long acting release formulation of Signifor have been filed worldwide for the treatment of acromegaly, including an application currently filed in the United States. In the EU, Signifor has orphan drug designation for acromegaly[8]. Orphan drugs are those that treat a condition which affects no more than five in 10,000 people in the EU[9].

References
[1]Holdaway M et al. Factors Influencing Mortality in Acromegaly. J Clin Endocrin Metab. 2004; 89(2): 667-674.
[2]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.
[3]Acromegaly. National Institutes of Health. National Institute of Diabetes and Digestive and Kidney Diseases. 2008 May; 8(3924): 1-10.
[4]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.
[5]Signifor® (pasireotide) Summary of Product Characteristics. Basel, Switzerland: Novartis Pharma AG; September 2014.
[6]RI Health Solutions. ”Epidemiology Report for an Orphan Drug Application in the European Union.” 2012: 1-17.
[7]European Medicines Agency. ”Summary Opinion (Post Authorisation) for Signifor Pasireotide.” http://www.ema.europa.eu/docs/en_GB/document_library/Summary_of_opinion/human/002052/WC500173649.pdf. Accessed November 2014.
[8]European Medicines Agency. ”Public Summary of Positive Opinion of Orphan Designation of Pasireotide for the Treatment of Acromegaly.” Available at: http://www.ema.europa.eu/docs/en_GB/document_library/Orphan_designation/2009/10/WC500006177.pdf. Accessed November 2014.
[9]European Medicines Agency. ”Orphan Designation.” Available at: http://www.ema.europa.eu/ema/index.jsp?curl=pages/regulation/general/general_content_000029.jsp. Accessed November 2014.
[10]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.
[11]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.
[12]Colao, et. al. Systemic Complications of Acromegaly: Epidemiology, Pathogenesis, and Management. Endocrine Reviews. 2004; 25:102-152.

* Lanreotide Autogel (Somatuline®Autogel®) is a registered trademark of Ipsen.