Advanced Accelerator Applications S.A. (AAA) has announced that the Food and Drug Administration (FDA), in the United States, has accepted and considered complete the resubmission of the New Drug Application (NDA) for its investigational drug lutetium Lu 177 dotatate (Lutathera). Lutathera is the brand name for the radiologic used in Peptide Receptor Radionuclide Therapy (PRRT). The FDA has set an action date of Jan 26, 2018.
AAA is attempting to obtain U.S. marketing rights of Lutathera approved for the treatment of gastroenteropancreatic neuroendocrine tumors (GEP-NETs) in adults. Originally, they submitted their data to the FDA last year. In response, they received a letter from the FDA at the end of 2016 asking for more information on the way their data was formatted. Specifically, the FDA showed concerns about issues related to format, traceability, uniformity, and dataset completeness relating previous studies including NETTER-1 (phase III study that compared treatment using lutetium Lu 177 oxodotreotide with a double dose of Octreotide LAR) and Erasmus (Phase I/II study conducted in patients with a wide range of NET indications).
Further, the FDA requested subgroup analyses for gender, age and racial subgroups, as well as other stratification factors and important disease characteristics. The FDA’s 2016 response letter also noted that any observations made during inspections of manufacturing facilities supporting the NDA need to be resolved prior to approval. However, the FDA did not request for additional studies.
AAA resubmitted the application in July 2017 after taking into consideration the concerns expressed by the FDA. As part of this process, the company sent a test dataset to the FDA to confirm that the data and format were compatible and able to be analyzed. Prior to resubmission, AAA enlisted the aid of consultants experienced in FDA reviews to perform a rigorous critique of both the datasets and the major clinical sections of the dossier prior to resubmitting.
Earlier this summer, the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) issued a positive opinion recommending the marketing authorization of lutetium/LU 177 for the treatment of gastroenteropancreatic neuroendocrine tumors (GEP-NETs) in adults.
So, right now you are thinking…..
What does this mean for us in the NET Community? We wait a little longer. Again. Some of our first trials in the U.S. started in 2000, so, we’ve gotten good at the waiting part. The FDA has until January 26, 2018 to decide whether or not to approve Lutathera as a new treatment for neuroendocrine tumors. Pro: This is the closest we have ever been, so, we can only hope that it gets passed. Con: There is a possibility that the FDA could not approve it or ask for additional data.
We always recommend pursuing treatment over waiting. If you are a candidate for this kind of treatment and are at a precarious place, we are recommending two options. The first being medical tourism. There are a number of centers in the U.S. and abroad that offer PRRT. Remember that since this therapy is still not FDA approved, it may not be covered by your insurance. Please visit our list of centers offering PRRT. Second, please consider other treatments while we wait for this new therapy. We always look for the bridge to help us get to the next treatment. If an oral chemo or radiology procedure gives you another year or more of stability, it’s worth doing it while we wait for this next therapy.