SP-2577, a potent reversible LSD1 inhibitor, has been granted Fast Track Designation by the FDA for the treatment of relapsed/refractory patients with Ewing sarcoma, Salarius Pharmaceuticals, Inc. developers of the drug, announced in a press release.
The FDA has granted a Regenerative Medicine Advanced Therapy designation to ADP-A2M4 for the treatment of patients with synovial sarcoma, according to a press release from Adaptimmune Therapeutics, developers of the agent.
A New Drug Application has been accepted and granted priority review by the FDA to the investigational agent selumetinib for the treatment of patients ≥3 years old with neurofibromatosis type 1 and symptomatic, inoperable plexiform neurofibromas, according to a press release from AstraZeneca and Merck.
The FDA plans to split up the submitted New Drug Application for avapritinib into 2 separate NDAs in the treatment of 2 different populations of patients with gastrointestinal stromal tumors, according to a press release from Blueprint Medicines.
The FDA has granted nirogacestat, an investigational gamma-secretase inhibitor, with a breakthrough therapy designation for the treatment of adult patients with progressive, unresectable, recurrent or refractory desmoid tumors or deep fibromatosis.
Ripretinib demonstrated a significantly improved progression-free survival compared with placebo in patients with gastrointestinal stromal tumors being treated in the fourth-line setting or beyond, according to topline results from the phase III INVICTUS trial. An NDA for ripretinib is planned for the first quarter of 2020.
The FDA has granted a priority review to a New Drug Application for avapritinib as a treatment for adult patients with PDGFRA exon 18–mutant gastrointestinal stromal tumors, regardless of prior therapy, and in the fourth-line setting for GIST.
A new drug application for tazemetostat, an EZH2 inhibitor, has been granted a priority review by the FDA for the treatment of patients with metastatic or locally advanced epithelioid sarcoma not eligible for curative surgery based on data from the epithelioid sarcoma cohort of a phase II trial.
Based on the ongoing and positive data observed with VEGFR targeted therapies, novel tyrosine kinase inhibitors and those already approved for other disease indications may become available to patients with bone and soft tissue sarcomas.