The U.S. Food and Drug Administration (FDA) has accepted and granted priority review of Ipsen’s supplemental New Drug Application (sNDA) for Somatuline Depot
(lanreotide) 120mg injection in the treatment of gastroenteropancreatic neuroendocrine tumors (GEP-NETs).
The priority review status is given to drug candidates that have the potential to provide significant improvement in treatment compared to currently approved options.
Somatuline is a somatostatin analog that is currently approved to treat patients with acromegaly. Another somatostatin analog - Sandostatin LAR
(octreotide) is approved for treating symptoms (flushing, diarrhea) associated with carcinoid syndrome but the new sNDA by Somatuline will make it the first somatostatin analog to potentially be approved as anti-tumor therapy.
The sNDA is supported by the results of the CLARINET Phase III study which was a phase III, randomized, double-blind, placebo-controlled study in which 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 (120 mg; n=101) or placebo (n=103). At enrollment, primary tumor locations were pancreas (44%), midgut (36%), hindgut (7%) and unknown (13%).
The study found that after 96 weeks, the median progressive free survival (PFS) for Somatuline treated patients was not reached and 65.1% had not progressed or died. In contrast, the placebo-treated patients had a median PFS of 18.0 months and only 33.0% had not progressed or died at 96 weeks. That is a 53% reduction in risk of disease progression or death based on a hazard ratio of 0.47 (95% CI: 0.30–0.73). The treatment was found to be safe and well tolerated.
In a press release
, Marc de Garidel, Chairman and Chief Executive Officer of Ipsen stated: “We are pleased that the US regulatory authorities have accepted the filing for Somatuline in the treatment of GEP-NETs and that the dossier has been granted priority review. We are excited about the potential benefits Somatuline could bring to patients suffering from this debilitating disease”.
Cynthia Schwalm, President and CEO of Ipsen North America added “To date, somatostatin analogs are approved to manage symptoms of GEP NETs and are not approved as anti-tumor therapy. Somatuline is the first and only somatostatin analog to demonstrate a statistically significant improvement in progression free survival in GEP NET in a large, multinational study clinical trial."
About Gastroenteropancreatic Neuroendocrine Tumors (GEP-NETs)
GEP-NETs are rare heterogeneous tumors that usually begin in the gastric mucosa, pancreas, small and large intestine. As a result of their origin, symptoms often include diarrhea and flushing. The only orphan drugs currently approved for GEP-NETs or carcinoid syndromes are Afinitor
(everolimus) for treating progressive neuroendocrine tumors of pancreatic origin (PNETs) in patients with unresectable, locally advanced or metastatic disease and Sandostatin LAR
(octreotide) for treating symptoms (flushing, diarrhea) associated with carcinoid syndrome. Somatuline is approved for treating GEP-NETs in other countries but in the United States, Somatuline Depot
only has orphan drug approval for treating patients with acromegaly. However, Somatuline Depot has orphan designation status with the FDA for GEP-NETs and as of today, it has been given a priority review.
A decision from the FDA is expected in the first quarter 2015.