The US Food and Drug Administration (FDA) granted orphan drug designation today to TPX-0005 — a small molecule kinase inhibitor — for the treatment of non–small cell lung adenocarcinoma with an ALK, ROS1, or NTRK mutation.1
Previous studies evaluating different agents for the treatment of non–small cell lung cancer (NSCLC) established the clinical benefit of targeting ALK-, ROS1-, and NTRK-positive malignancies, but a frequent challenge is drug resistance.
TPX-0005 demonstrates potent inhibitory effects against solvent mutations after multiple treatments, including ALK G1202R, ROS1 G2032R, TRKA G595R, and TRKC G623R.
The FDA based its approval on the findings from the ongoing TRIDENT-1 trial (ClinicalTrial.gov Identifier: NCT03093116), a “phase 1/2, open-label, multi-center, first-in-human study of the safety, tolerability, pharmacokinetics, and anti-tumor activity of TPX-0005 in patients with advanced solid tumors harboring ALK, ROS1, or NTRK1-3 rearrangements.”
Orphan drug status is granted to drugs and biologics that target rare diseases, defined as a condition that affects fewer than 200,000 people in the US, or affects more than 200,000 people but are not expected to recoup the costs of research, development, and marketing.
TPX-0005 will be granted market exclusivity for 7 years if approved.
- TP therapeutics announces FDA orphan drug designation granted to TPX-0005 for treatment of non-small cell lung adenocarcinomas harboring ALK, ROS1, or NTRK oncogenic rearrangements [news release]. San Diego, CA: Business Wire; June 27, 2017. http://www.businesswire.com/news/home/20170627005421/en/TP-Therapeutics-Announces-FDA-Orphan-Drug-Designation. Accessed June 27, 2017.