Ignyta, a biotechnology company focused on precision medicine in oncology, announced that the US Food and Drug Administration (FDA) has granted a Breakthrough Therapy Designation (BTD) to entrectinib "for the treatment of NTRK fusion-positive, locally advanced or metastatic solid tumours in adult and paediatric patients who have either progressed following prior therapies or who have no acceptable standard therapies." Entrectinib is the company's investigational, orally available, CNS-active tyrosine kinase inhibitor targeting tumours that harbour NTRK1/2/3, ROS1, or ALK gene fusions.
The FDA's Breakthrough Therapy Designation is intended to expedite development and review timelines of potential new medicines for use in the treatment of a serious or life-threatening condition when preliminary clinical evidence indicates the drug may demonstrate substantial improvement over available therapies on one or more clinically significant endpoints.
"The granting of Breakthrough Therapy Designation enables us to continue high quality engagement with the FDA during the development of entrectinib, and we greatly appreciate the commitment by FDA to move this investigational drug forward," said Jonathan Lim, M.D., chairman and chief executive officer of Ignyta. "We believe this designation validates the broad potential of entrectinib as a novel treatment for patients, regardless of age, with TRK-positive tumors, a group of cancers for which there currently is no approved treatment and which represents a clear unmet medical need."
Entrectinib is a novel, orally available, CNS-active tyrosine kinase inhibitor targeting tumours that harbour activating alterations to NTRK1/2/3 (encoding TRKA/TRKB/TRKC), ROS1 or ALK. Entrectinib is the only TRK inhibitor with clinically demonstrated activity against primary and metastatic CNS disease, and does not have undesirable off-target activity. This product candidate is in a phase 2 clinical trial called STARTRK-2, which is the second of the "Studies of Tumor Alterations Responsive to Targeting Receptor Kinases." The trial is a global, multicenter, open label, potentially registration-enabling phase 2 clinical trial of entrectinib that utilizes a basket design with screening of patient tumour samples for the relevant targets. Such a basket design takes full advantage of entrectinib's demonstrated preliminary clinical activity across a range of different tumour types and molecular targets.