AAA Receives Orphan Drug Designation from FDA and EMA for Gallium-68 DOTATATE for Use in Patients with Gastro-Entero-Pancreatic Neuroendocrine Tumors
SAINT-GENIS-POUILLY, France, March 4, 2014 /PRNewswire/ –
Advanced Accelerator Applications (AAA), a fast growing international player in
Molecular Nuclear Medicine (MNM), announced today that they have received orphan drug
designation status for their radiopharmaceutical, Gallium-68 DOTATATE. The orphan drug
designation has been granted by both the U.S. Food and Drug Administration (FDA) and the
European Medicines Agency (EMA) for use of Gallium-68 DOTATATE as a diagnostic agent for
the management of Gastro-Entero-Pancreatic Neuroendocrine Tumors (GEP-NETs). The
designation should foster rapid development of the agent for the benefit of GEP-NET
patients in the U.S. and Europe.
Gallium-68 DOTATATE is a radiopharmaceutical used in PET/CT imaging of GEP-NETs. The
product will be prepared using AAA’s patented kit, which is reconstituted in hospital
radiopharmacies without the use of a radiochemistry module, thus making the product
available to all hospitals, even those who do not have a fully equipped GMP production
Existing data show that the Gallium-68-labeled PET radiopharmaceutical should
represent a major improvement compared to the current standard. Available data indicates
that Gallium-68 DOTATATE not only has greater sensitivity and specificity for tumor
detection than the current standard, but it is also expected to significantly reduce
radiation doses received by patients.
Stefano Buono, Chief Executive Officer of AAA, commented: “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
“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,” added AAA’s Chief Operating Officer Gerard Ber.
The EMA’s orphan medicinal product designation is designed to promote the development
of drugs that may provide significant benefit to patients suffering from rare,
life-threatening diseases. In addition to ten years of market exclusivity, the orphan drug
designation also provides special incentives for sponsors including eligibility for
protocol assistance and possible exemptions or reductions in certain regulatory fees
during development or at the time of application for marketing approval.
Similarly, FDA orphan drug designation is intended to encourage companies to develop
therapies for the treatment of diseases that affect fewer than 200,000 individuals in the
U.S. This designation could provide AAA with seven years of marketing exclusivity for
AAA’s Gallium-68 DOTATATE as a diagnostic agent for the management of GEP-NETs if it
receives first approval by the FDA. Prior to FDA approval, orphan designation by the FDA
provides the opportunity to obtain grant funding to help finance costs of clinical trial
expenses, tax credits for clinical research expenses and potential waiver of the FDA’s
application user fees.
About Gallium-68 DOTATATE kit
Gallium-68 DOTATATE or Gallium [Ga-68]-N-[(
hreonine-cyclic(2-7)disulfide is a diagnostic PET imaging agent currently under
development. The pre-formulated GMP kit is used to prepare the final product, which is a
ready-to-inject 68Ga-somatostatin analogue peptide solution.
Gallium-68 DOTATATE is a radiopharmaceutical agent used to help diagnose
Gastro-Entero-Pancreatic Neuroendocrine cancers via PET/CT imaging. Existing data show
that AAA’s Ga-68-labeled GEP-NET PET radiopharmaceutical should represent a major
improvement compared to the existing standard.
About Gastro-Entero-Pancreatic Neuroendocrine Tumors
Gastro-Entero-Pancreatic Neuroendocrine Tumors (GEP-NETs) are a rare group of tumors
that start in the cells of the neuroendocrine system, a network of endocrine glands and
cells throughout the body. GEP-NETs develop in the digestive system, usually starting in
the stomach, bowel or pancreas and constitute a life-threatening disease.
About Molecular Nuclear Medicine (MNM)
Molecular Nuclear Medicine is a medical specialty which uses trace amounts of active
substances, called radiopharmaceuticals, to create images of organs and lesions and to
treat various diseases, like cancer. The technique works by injecting into the patient’s
body targeted radiopharmaceuticals that accumulate in the organs or lesions that reveal
specific biochemical processes.
Molecular Nuclear Diagnostics employ a variety of imaging devices and
radiopharmaceuticals. PET (Positron Emission Tomography) and SPECT (Single Photon Emission
Tomography) are highly sensitive imaging technologies that enable physicians to diagnose
different types of cancer, cardiovascular diseases, neurological disorders and other
diseases in their early stages.
PET (Positron Emission Tomography) is a MNM imaging technique used in diagnosis and
biomedical research. In PET, a chemical compound labeled with a short-lived
positron-emitting radionuclide of carbon, oxygen, nitrogen, or fluorine is injected into
the body. The activity of such a radiopharmaceutical is quantitatively measured throughout
the target organs. Data are analyzed and reconstructed by means of a computer to produce
images of the organs being scanned.
About Advanced Accelerator Applications
Advanced Accelerator Applications (AAA) is a European pharmaceutical company founded
in 2002 to develop innovative diagnostic and therapeutic products. AAA’s main focus is in
the field of Molecular Imaging and targeted, individualized therapy for the management of
patients with serious conditions (Personalized Medicine). AAA currently has 17 production
and R&D facilities able to manufacture both diagnostics and therapeutic MNM products, and
has over 290 employees in 11 countries (France, Italy, UK, Germany, Switzerland, Spain,
Poland, Portugal, Israel, U.S. and Canada). In 2013 AAA is expecting to reach revenues of
EUR56.6 million (+27% vs. 2012) and EBITDA of EUR14 million (+49% vs. 2012). For more
information please visit: http://www.adacap.com
SOURCE Advanced Accelerator Applications