Pivotal phase 3 data demonstrated treatment with luspatercept resulted in statistically significant increased red blood cell transfusion independence compared with placebo.
Tazverik Receives FDA Approval as First Treatment Specifically for Metastatic or Locally Advanced Epithelioid Sarcoma
On January 23, 2020, the FDA granted accelerated approval to tazemetostat (Tazverik; Epizyme), a methyltransferase inhibitor, for the treatment of metastatic or locally advanced epithelioid sarcoma in adults and pediatric patients aged ≥16 years who are not eligible for complete resection.
On January 9, 2020, the FDA approved avapritinib (Ayvakit; Blueprint Medicines), a kinase inhibitor, for the treatment of adult patients with unresectable or metastatic gastrointestinal stromal tumor (GIST) harboring platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutations, including PDGFRA D842V mutations.
According to the FDA’s “Center for Drug Evaluation and Research (CDER) Annual Report: New Drug Therapy Approvals 2019,” the agency approved 48 novel drugs in 2019. Although this number does not approach the record of 59 approvals in 2018, it far surpasses the mere 22 approvals that occurred in 2016.
On December 12, 2019, the FDA issued draft guidance to implement amendments to the Federal Food, Drug, and Cosmetic (FD&C) Act that will facilitate early assessment of studies of molecularly targeted oncology drugs that may be effective in the treatment of pediatric cancers.
On December 3, 2019, the FDA granted Regenerative Medicine Advanced Therapy (RMAT) designation to the biopharmaceutical company Adaptimmune Therapeutics for ADP-A2M4 for the treatment of synovial sarcoma. Earlier this year, the FDA granted Orphan Drug designation to the agent for the treatment of soft tissue sarcomas.
- FDA Approves Ziextenzo, Third Biosimilar to Pegfilgrastim for Febrile Neutropenia
- Trump Will Nominate Dr Hahn as Next FDA Commissioner
- Researchers Report High Rates of E-Cigarette Use Among Children and Teens
- Xospata Extends Overall Survival in Patients with FLT3 Mutation–Positive Relapsed or Refractory Acute Myeloid Leukemia
- Published Results from KEYNOTE-048 Trial Show Extended Survival with Keytruda Advanced Head and Neck Cancers
- Discussing Costs of Genomic Testing with Patients
Reprogramming patients’ immune cells to treat their cancer has become the front line of cancer therapy, with chimeric antigen receptor (CAR) T-cell therapy now approved by the FDA for several blood cancers. But translating this success to solid tumors remains a challenge. At the 2019 American Society of Clinical Oncology (ASCO) Annual Meeting, Gianpietro Dotti, MD, Cancer Cellular Immunotherapy Program, Lineberger Comprehensive Cancer Center, University of North Carolina, Chapel Hill, discussed efforts to extend the application of CAR T-cell therapy to solid tumors.
FDA issues finalized guidelines for use of in vitro diagnostics in oncology drug trials and warnings on vaping; new findings report patients bear more costs when MBC treatment is not concordant with NCCN guidelines.