On November 18, 2020 PTC Therapeutics, Inc. (NASDAQ: PTCT), reported that the United States (U.S.) Food and Drug Administration (FDA) has granted PTC596 both Orphan Drug Designation and Fast Track designation for the potential treatment of leiomyosarcoma (LMS), a rare type of cancer that affects smooth muscle tissue (Press release, PTC Therapeutics, NOV 18, 2020, ry-designations-for-ptc596-to-advance-treatment-of-two-rare-oncology-indications-301175743.html [SID1234571377]). Furthermore, the FDA has also granted PTC596 a Rare Pediatric Disease designation and Orphan Drug Designation for the potential treatment of Diffuse Intrinsic Pontine Glioma (DIPG), an ultra-rare childhood glioma. PTC596 is currently being studied in clinical trials in LMS and DIPG.

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"We are very pleased with the FDA’s decisions to grant PTC596 these designations," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics. "This brings us one step closer to providing truly novel therapeutic approaches to patients with devastating rare cancers seen in children and adults that do not have good treatment options."

PTC596 is an orally bioavailable small molecule tubulin binding agent that arrests tumor cells in G2/M phase, including cancer stem cells, through the action of inhibiting tubulin polymerization. It is currently in a Phase 1b study for LMS, which accounts for approximately 10 to 28% of all soft tissue sarcomas.1 Approximately 4000 patients are diagnosed with LMS annually in the US, the risk of developing metastases is approximately 40%, and the 5-year survival rate is estimated to be 13.6% for metastatic LMS.2 PTC596 is also currently in a clinical study for DIPG, an ultra-rare glioma arising in the brainstem that makes up 10 to 15% of all brain tumors in children.3 Approximately 300 patients are diagnosed with DIPG annually in the US, and the median overall survival with the current standard of care of radiation therapy, is approximately 9 months with a two-year overall survival rate of less than 10%.4