SOUTH PLAINFIELD, N.J., Nov. 18, 2020 /PRNewswire/ -- PTC Therapeutics, Inc. (NASDAQ: PTCT), today announced 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. 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.
"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
About PTC Therapeutics, Inc.
PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.
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1 Bessen, T., Caughey, G. E., Shakib, S., Potter, J. A., Reid, J., Farshid, G., Roder, D., & Neuhaus, S. J. (2019). A population-based study of soft tissue sarcoma incidence and survival in Australia: An analysis of 26,970 cases. Cancer epidemiology, 63, 101590. https://doi.org/10.1016/j.canep.2019.101590
2 Ries LAG, Young JL, Keel GE, Eisner MP, Lin YD, Horner M-J (editors). SEER Survival Monograph: Cancer Survival Among Adults: U.S. SEER Program, 1988-2001, Patient and Tumor Characteristics. National Cancer Institute, SEER Program, NIH Pub. No. 07-6215, Bethesda, MD, 2007.
3 Raabe, E. H., & Cohen, K. (n.d.). What is DIPG? Retrieved November 9, 2020, from https://www.dipg.org/dipg-facts/what-is-dipg/
4 Jansen, M. H., Veldhuijzen van Zanten, S. E., Sanchez Aliaga, E., Heymans, M. W., Warmuth-Metz, M., Hargrave, D., van der Hoeven, E. J., Gidding, C. E., de Bont, E. S., Eshghi, O. S., Reddingius, R., Peeters, C. M., Schouten-van Meeteren, A. Y., Gooskens, R. H., Granzen, B., Paardekooper, G. M., Janssens, G. O., Noske, D. P., Barkhof, F., Kramm, C. M., … van Vuurden, D. G. (2015). Survival prediction model of children with diffuse intrinsic pontine glioma based on clinical and radiological criteria. Neuro-oncology, 17(1), 160–166. https://doi.org/10.1093/neuonc/nou104
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