Welcome!

News Feed Item

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 radiopharmacy unit.

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 few years."

"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 Gérard 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-[(4,7,10-Tricarboxymethyl-1,4,7,10-tetraazacyclododec-1-yl)acetyl]-D-phenylalanyl-L-cysteinyl-L-tyrosyl-D-tryptophanyl-L-lysyl-L-threoninyl-L-cysteinyl-L-threonine-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 €56.6 million (+27% vs. 2012) and EBITDA of €14 million (+49% vs. 2012). For more information please visit: http://www.adacap.com

SOURCE Advanced Accelerator Applications

More Stories By PR Newswire

Copyright © 2007 PR Newswire. All rights reserved. Republication or redistribution of PRNewswire content is expressly prohibited without the prior written consent of PRNewswire. PRNewswire shall not be liable for any errors or delays in the content, or for any actions taken in reliance thereon.

Latest Stories
"Storpool does only block-level storage so we do one thing extremely well. The growth in data is what drives the move to software-defined technologies in general and software-defined storage," explained Boyan Ivanov, CEO and co-founder at StorPool, in this SYS-CON.tv interview at 16th Cloud Expo, held June 9-11, 2015, at the Javits Center in New York City.
As Marc Andreessen says software is eating the world. Everything is rapidly moving toward being software-defined – from our phones and cars through our washing machines to the datacenter. However, there are larger challenges when implementing software defined on a larger scale - when building software defined infrastructure. In his session at 16th Cloud Expo, Boyan Ivanov, CEO of StorPool, provided some practical insights on what, how and why when implementing "software-defined" in the datacent...
Blockchain. A day doesn’t seem to go by without seeing articles and discussions about the technology. According to PwC executive Seamus Cushley, approximately $1.4B has been invested in blockchain just last year. In Gartner’s recent hype cycle for emerging technologies, blockchain is approaching the peak. It is considered by Gartner as one of the ‘Key platform-enabling technologies to track.’ While there is a lot of ‘hype vs reality’ discussions going on, there is no arguing that blockchain is b...
Blockchain is a shared, secure record of exchange that establishes trust, accountability and transparency across business networks. Supported by the Linux Foundation's open source, open-standards based Hyperledger Project, Blockchain has the potential to improve regulatory compliance, reduce cost as well as advance trade. Are you curious about how Blockchain is built for business? In her session at 21st Cloud Expo, René Bostic, Technical VP of the IBM Cloud Unit in North America, discussed the b...
ChatOps is an emerging topic that has led to the wide availability of integrations between group chat and various other tools/platforms. Currently, HipChat is an extremely powerful collaboration platform due to the various ChatOps integrations that are available. However, DevOps automation can involve orchestration and complex workflows. In his session at @DevOpsSummit at 20th Cloud Expo, Himanshu Chhetri, CTO at Addteq, will cover practical examples and use cases such as self-provisioning infra...
You know you need the cloud, but you’re hesitant to simply dump everything at Amazon since you know that not all workloads are suitable for cloud. You know that you want the kind of ease of use and scalability that you get with public cloud, but your applications are architected in a way that makes the public cloud a non-starter. You’re looking at private cloud solutions based on hyperconverged infrastructure, but you’re concerned with the limits inherent in those technologies.
Is advanced scheduling in Kubernetes achievable?Yes, however, how do you properly accommodate every real-life scenario that a Kubernetes user might encounter? How do you leverage advanced scheduling techniques to shape and describe each scenario in easy-to-use rules and configurations? In his session at @DevOpsSummit at 21st Cloud Expo, Oleg Chunikhin, CTO at Kublr, answered these questions and demonstrated techniques for implementing advanced scheduling. For example, using spot instances and co...
A strange thing is happening along the way to the Internet of Things, namely far too many devices to work with and manage. It has become clear that we'll need much higher efficiency user experiences that can allow us to more easily and scalably work with the thousands of devices that will soon be in each of our lives. Enter the conversational interface revolution, combining bots we can literally talk with, gesture to, and even direct with our thoughts, with embedded artificial intelligence, whic...
The use of containers by developers -- and now increasingly IT operators -- has grown from infatuation to deep and abiding love. But as with any long-term affair, the honeymoon soon leads to needing to live well together ... and maybe even getting some relationship help along the way. And so it goes with container orchestration and automation solutions, which are rapidly emerging as the means to maintain the bliss between rapid container adoption and broad container use among multiple cloud host...
The cloud era has reached the stage where it is no longer a question of whether a company should migrate, but when. Enterprises have embraced the outsourcing of where their various applications are stored and who manages them, saving significant investment along the way. Plus, the cloud has become a defining competitive edge. Companies that fail to successfully adapt risk failure. The media, of course, continues to extol the virtues of the cloud, including how easy it is to get there. Migrating...
Imagine if you will, a retail floor so densely packed with sensors that they can pick up the movements of insects scurrying across a store aisle. Or a component of a piece of factory equipment so well-instrumented that its digital twin provides resolution down to the micrometer.
The need for greater agility and scalability necessitated the digital transformation in the form of following equation: monolithic to microservices to serverless architecture (FaaS). To keep up with the cut-throat competition, the organisations need to update their technology stack to make software development their differentiating factor. Thus microservices architecture emerged as a potential method to provide development teams with greater flexibility and other advantages, such as the abili...
In his keynote at 18th Cloud Expo, Andrew Keys, Co-Founder of ConsenSys Enterprise, provided an overview of the evolution of the Internet and the Database and the future of their combination – the Blockchain. Andrew Keys is Co-Founder of ConsenSys Enterprise. He comes to ConsenSys Enterprise with capital markets, technology and entrepreneurial experience. Previously, he worked for UBS investment bank in equities analysis. Later, he was responsible for the creation and distribution of life settle...
Product connectivity goes hand and hand these days with increased use of personal data. New IoT devices are becoming more personalized than ever before. In his session at 22nd Cloud Expo | DXWorld Expo, Nicolas Fierro, CEO of MIMIR Blockchain Solutions, will discuss how in order to protect your data and privacy, IoT applications need to embrace Blockchain technology for a new level of product security never before seen - or needed.
As DevOps methodologies expand their reach across the enterprise, organizations face the daunting challenge of adapting related cloud strategies to ensure optimal alignment, from managing complexity to ensuring proper governance. How can culture, automation, legacy apps and even budget be reexamined to enable this ongoing shift within the modern software factory? In her Day 2 Keynote at @DevOpsSummit at 21st Cloud Expo, Aruna Ravichandran, VP, DevOps Solutions Marketing, CA Technologies, was jo...