Sarcoma

FDA Grants Fast Track Designation to LSD1 Inhibitor SP-2577 for Ewing Sarcoma

December 17, 2019

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.

FDA Grants Regenerative Medicine Advanced Therapy Designation to ADP-A2M4 for Synovial Sarcoma

December 04, 2019

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.

FDA Grants Priority Review to Selumetinib for Neurofibromatosis Type 1 in Pediatric Patients

November 14, 2019

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.

Avapritinib NDA Being Split in Two, PDGFRA+ GIST Indication Continuing Review

October 28, 2019

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.

Cobimetinib Granted FDA Breakthrough Therapy Designation for Histiocytic Neoplasms

October 04, 2019

The FDA has granted cobimetinib, a MEK inhibitor, with a breakthrough therapy designation for the treatment of adult patients with histiocytic neoplasms who do not harbor a BRAF V600 mutation.

FDA Grants Breakthrough Designation to Nirogacestat for Desmoid Tumors

September 04, 2019

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 Demonstrates Benefit in Patients With Previously Treated GIST

August 13, 2019

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.

FDA Grants Priority Review to Avapritinib in PDGFRA+ and Fourth-Line GIST

August 07, 2019

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.

Tazemetostat Receives FDA Priority Review for Metastatic or Locally Advanced Epithelioid Sarcoma

July 25, 2019

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.

VEGFR Targeted Therapies and Combinations Emerge for the Treatment of Sarcomas

July 02, 2019

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.