FDA gives orphan drug designation for Ga-68 DOTATOC

Gallium-68 DOTATOC is now blazing faster trail toward U.S. regulatory approval in the case of neuroendocrine cancer imaging, according to a Nov. 18 statement from the Society of Nuclear Medicine and Molecular Imaging (SNMMI).

The designation is given for the management of relatively rare diseases that affect fewer than 200,000 people or for applications that are narrow in scope. Ga-68 (DOTA0-Phel-Tyr3) octreotide (Ga-68 DOTATOC) is being fast-tracked for the management of neuroendocrine tumors, which has an estimated prevalence of about 110,000 people across the country. The orphan drug status is a result of an application for the designation submitted in August 2013 by SNMMI’s Clinical Trials Network, a focus group that facilitates the utilization of molecular imaging radiopharmaceuticals.

“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,” stated Michael Graham, PhD, MD, co-chair of the Clinical Trials Network and former SNMMI president, via release.

Orphan drug status could speed up regulatory approval by requiring fewer patients per clinical trial and releasing grant funds for its development. As an agent for neuroendocrine-tumor PET imaging, Ga-68 DOTATOC has the potential to cut down on imaging times and lead to an uptick in patient throughput. What used to take days with more conventional methods could be reduced to same-day imaging with a reduction in radiation dose. Further data is needed before the FDA can flash an official green light for commercial use.