Regulatory authorities decisions expected in early Q1, 2015 in US and in Q2, 2015 in Europe
September 1, 2014
Ipsen today announced that the U.S. Food and Drug Administration (FDA) has accepted and granted priority review of its supplemental New Drug Application (sNDA) for Somatuline® Depot® 120mg injection in the treatment of gastroenteropancreatic neuroendocrine tumors (GEP-NETs). The FDA designates priority review status to drug candidates that have the potential to offer a significant improvement in treatment compared to currently approved options. Decision is expected in early Q1, 2015.
In the European Union, the dossier of the national marketing authorization (MA) variations for Somatuline® Autogel® 120mg injection has been validated by all national 25 drug regulatory authorities. The first decisions are expected by Q2 2015.
The regulatory submissions and variations were supported by the results of the CLARINET® Phase III study, which demonstrated the antitumor effect of Somatuline® in the treatment of patients with GEP-NETs, and which was recently published in the July 17th issue of The New England Journal of Medicine2.
Marc de Garidel, Chairman and Chief Executive Officer of Ipsen stated: “We are pleased that the US and European regulatory authorities have accepted the filing for Somatuline® in the treatment of GEP-NETs and that the dossier in the US has been granted priority review. We are excited about the potential benefits Somatuline® could bring to patients suffering from this debilitating disease”.
The data from CLARINET® is purely investigational, as Somatuline® is not authorized for the indication of treatment of gastroenteropancreatic neuroendocrine tumors (GEP-NETs) in any market. In many countries where it is marketed as Somatuline® Autogel®, Somatuline® is approved for treatment of acromegaly and for the symptoms associated with neuroendocrine tumors, which can include the treatment of GEP-NET patients experiencing symptoms from carcinoid syndrome, and Somatuline® is approved in many countries for the treatment of acromegaly. Somatuline® Depot is approved in the US for the treatment of acromegaly but not for the treatment of GEP-NETs or the symptoms thereof.
About gastroenteropancreatic neuroendocrine tumors
Gastroenteropancreatic neuroendocrine tumors (GEP-NETs) are serious and rare types of cancer. They constitute an heterogeneous group of tumors most often arising from cells in the gastrointestinal tract and the pancreas; although rare, their incidence has been on the rise (4-6 fold increase in the last 30 years). They have the ability to secrete functional amines and peptides and based on the type and amount of these bioactive substances in circulation, they can or cannot result in an identifiable hormonal clinical syndrome. GEP-NETs can be clinically silent for long periods of time, delaying the diagnosis until late presentation with hormonal related symptoms or with symptoms related to tumor mass effect such as intestinal obstruction or abdominal pain.
The active substance in Somatuline® [(Somatuline® Autogel® / Somatuline® Depot) (lanreotide) Injection 120 mg (referred to as Somatuline®)] is lanreotide acetate, a somatostatin analogue that inhibits the secretion of several endocrine, exocrine and paracrine amines and peptides.. It has been shown to be effective in inhibiting the secretion of GH and certain hormones secreted by the digestive system. Somatuline® is marketed as Somatuline® Depot within the United States and as Somatuline® Autogel® in other countries where it has marketing authorization, including various EU Member States.
Somatuline® was initially developed and continues to be used for the treatment of acromegaly in many countries, including the United States, where it is indicated for the long-term treatment of patients with acromegaly who have had an inadequate response to or cannot be treated with surgery and/or radiotherapy. Somatuline® is not currently indicated as an antitumor agent for the treatment of GEP-NETs. Somatuline® is approved for the treatment of symptoms associated with neuroendocrine tumors in many markets, but is not approved within the United States for this indication.
Important Safety Information about Somatuline®
The most commonly reported adverse drug reactions following treatment with lanreotide are gastrointestinal disorders and cholelithiasis. In addition there have been reports of changes to glucose regulation, levels of liver enzymes, changes to heart rate, injection site and allergic reactions. The product information should be consulted for a complete list of undesirable effects, warnings and precautions and contraindications for use.
CLARINET® is an investigational, phase III, randomized, double-blind, placebo-Controlled study of Lanreotide’s Antiproliferative Response In patients with enteropancreatic Neuroendocrine Tumors (ClinicalTrials.gov NCT00353496). This 96-week multinational study was conducted in collaboration with the UK & Ireland Neuroendocrine Tumour Society (UKI NETS) and the European Neuroendocrine Tumour Society (ENETS).
A total of 204 patients from 48 centers across 14 countries with well or moderately differentiated non-functioning enteropancreatic neuroendocrine tumors and a proliferation index (Ki67) of <10%, were randomized to treatment with Somatuline® Autogel® 120 mg (n=101) or placebo (n=103). At enrollment, primary tumor locations were pancreas (44%), midgut (36%), hindgut (7%) and unknown (13%). Most patients had stable disease (96%) and were treatment-naïve (84%). Thirty percent of patients had a Ki67 of 3% to ≤10% (WHO grade 2) and 33% had an hepatic tumor load >25%.
The primary efficacy endpoint was time to either disease progression (centrally assessed using Response Evaluation Criteria In Solid Tumors, RECIST 1.0) or death. Two baseline computed tomography or magnetic resonance imaging scans (12 to 24 weeks) were performed, followed by additional scans at 12- week intervals during the first year and 24-week intervals during the second year up to 96 weeks.
The data from CLARINET® showed that investigational treatment with Somatuline® substantially prolonged time to disease progression or death versus placebo (hazard ratio 0.47, p=0.0002). Safety data generated from the CLARINET® study were consistent with the known safety profile of Somatuline®.
Ipsen is a global specialty-driven pharmaceutical company with total sales exceeding €1.2 billion in 2013. Ipsen’s ambition is to become a leader in specialty healthcare solutions for targeted debilitating diseases. Its development strategy is supported by 3 franchises: neurology, endocrinology and uro-oncology. Moreover, the Group has an active policy of partnerships. Ipsen’s R&D is focused on its innovative and differentiated technological platforms, peptides and toxins. In 2013, R&D expenditure totaled close to €260 million, representing more than 21% of Group sales. Moreover, Ipsen also has a significant presence in primary care. The Group has close to 4,600 employees worldwide. Ipsen’s shares are traded on segment A of Euronext Paris (stock code: IPN, ISIN code: FR0010259150) and eligible to the “Service de Règlement Différé” (“SRD”). The Group is part of the SBF 120 index. Ipsen has implemented a Sponsored Level I American Depositary Receipt (ADR) program, which trade on the over-the-counter market in the United States under the symbol IPSEY. For more information, visit www.ipsen.com.