FDA And EMA Grant Orphan Drug Status To AAA's Gallium-68 DOTATATE

Advanced Accelerator Applications (AAA) has won orphan drug status from both the U.S. Food and Drug Administration (FDA) and the European Medicines Agency (EMA) for its Gallium-68 DOTATATE as a diagnostic agent for the management of Gastro-Entero-Pancreatic Neuroendocrine Tumors (GEP-NETs).

The orphan drug designation grants the company a range of benefits from both regulatory authorities. The EMA’s orphan medicinal product designation awards ten years of marketing exclusivity along with special incentives such as eligibility for protocol assistance and possible exemptions or reductions in fees during the time of product development or application of marketing authorization.                                                      

The FDA’s orphan drug status designation could also provide AAA’s Gallium-68 DOTATATE with seven years of marketing exclusivity, if approved. The designation itself provides sponsor companies with opportunity for grant funding to aid in support costs of expenses due to clinical trials, tax credits for clinical research costs, and potential waiver of the FDA’s fees for application users. The designation is intended to encourage companies to promote development of drugs to treat patients suffering from rare, life-threatening diseases.

Gérard Ber, AAA's Chief Operating Officer, said, “Receiving orphan drug designation for Gallium-68 DOTATATE is an important step in the overall approval process. It reinforces our position and interest in NETs, both on the diagnostic and treatment front. Strong literature evidence already exists about the efficacy of Gallium-68 DOTATATE, which we believe reduces our development risks for this product.”

Gallium-68 DOTATATE is a diagnostic PET imaging agent being developed by AAA to help diagnose Gastro-Entero-Pancreatic Neuroendocrine cancers using PET/CT imaging. The company said Ga-68-labeled GEP-NET PET radiopharmaceutical potentially represents a step forward compared to current standards due to greater sensitivity and specificity for tumor detection. The product could also significantly reduce radiation doses received by patients, the company said.

GEP-NETs are a rare group of tumors which originate in the cells of the neuroendocrine system. The tumors then develop in parts of the digestive system such as the stomach, bowel, or pancreas, and progress into a life-threatening disease.

Chief Executive Officer of AAA, Stefano Buono, said, “GEP-NETs constitute a life-threatening disease and effective patient management requires accurate diagnostic tools. The orphan drug designation of AAA's Gallium-68 DOTATATE will accelerate the development of this agent and hopefully allow it to be available to patients in the next few years.”