November 20, 2013 — The Society of Nuclear Medicine and Molecular Imaging (SNMMI) announced that the U.S. Food and Drug Administration (FDA) has designated the radiopharmaceutical Gallium-68 (DOTA0-Phel-Tyr3) octreotide (Ga-68 DOTATOC) as an orphan drug for the management of neuroendocrine tumors (NET). This designation may lead to faster approval of the agent, which would greatly benefit NET patients in the United States. Currently, there are only a few small U.S.-based clinical trials for Ga-68 labeled NET positron emission tomography (PET) agents available for patients, who must otherwise travel out of the country if the scan is required to manage their disease.
In August 2013, SNMMI’s Clinical Trials Network (CTN) submitted an application to the FDA for orphan drug designation for Ga-68 DOTATOC under the Orphan Drug Act. This act provides for granting special status to a drug or biological product to treat a rare disease or condition. For a drug to qualify for orphan designation, the disease or condition must affect fewer than 200,000 people. With a prevalence of approximately 110,000 patients in the United States with NETs, Ga-68 DOTATOC meets this requirement.
With this new orphan drug designation, Ga-68 DOTATOC will be directed down a unique pathway within the FDA. On its road to approval, fewer patients will likely be required for clinical trials, and funds may be available for studies through the Office of Orphan Products Development (OOPD) grant program to support the clinical development of products where the proposed product will be superior to the existing product (Octreoscan).
“The next step in this process will be to meet with FDA officials to discuss the data needed for regulatory approval, factoring in the extensive literature on the safety and efficacy of this agent and the patients currently enrolled in an ongoing prospective trial,” said Michael Graham, Ph.D., M.D. and co-chair, CTN.
SNMMI’s CTN will remain involved in the clinical trials for Ga-68-labeled DOTA agents as new studies begin. CTN has developed a comprehensive repository of materials to aid in the initiation and conduct of clinical trials including release criteria, an imaging manual, data collection forms and a template investigational new drug (IND) application for Ga-68 agents to help increase investigational use.
“I think I speak for all NET patients when I say this is a great first step towards approval of this agent in the United States,” said Josh Mailman, chairman, SNMMI’s Patient Advocacy Advisory Board and president, NorCalCarciNET. “Ga-68-labeled NET PET radiopharmaceuticals will reduce the time it takes to image a NET patient from 2-3 days to just a few hours. It has also been shown in early studies in the U.S. and those overseas to change treatment paths for a number of patients. Lastly, the exposed dose of radiation is lower than the current standard of care. ”
For more information: www.snmmi.org
July 30, 2024 
