FDA approved a new treatment for non-small cell lung cancer (NSCLC), as well as drugs to treat chronic immune thrombocytopenia (ITP) and a rare form of childhood rickets.
FDA approved osimertinib (Tagrisso, AstraZeneca) for the first-line treatment of patients with metastatic non-small cell lung cancer (NSCLC) whose tumors have epidermal growth factor receptor (EGFR) mutations, as detected by an FDA-approved test.
The approval is based on results from a phase 3 trial, which were presented at the European Society of Medical Oncology 2017 Congress and published in the New England Journal of Medicine.
“The approval of osimertinib (Tagrisso) in the first-line setting represents a major advance in the treatment of patients with EGFR mutations and a significant change in the treatment paradigm. Osimertinib (Tagrisso) provides robust improvements in progression-free survival with no unexpected safety signals compared to the previous generation of EGFR inhibitors,” said Suresh S. Ramalingam, MD, principal investigator of the trial, from Winship Cancer Institute of Emory University, Atlanta, in a statement from AstraZeneca.
In addition, FDA approved ostamatinib disodium hexahydrate (Tavalisse, Rigel Pharmaceuticals) for the treatment of thrombocytopenia in adult patients with chronic immune thrombocytopenia (ITP) who have had an insufficient response to a previous treatment.
Tavalisse is an oral spleen tyrosine kinase (SYK) inhibitor that targets the underlying autoimmune cause of the disease by impeding platelet destruction.
"Related: Chronic ITP is challenging to treat because the heterogeneity of the disease makes it difficult to predict how an individual patient will respond to available treatments and not all patients can find a treatment that works well for them," said James Bussel, MD, professor emeritus of pediatrics at Weill Cornell Medicine and the principal study investigator on the FIT Phase 3 program, in a statement from Rigel. "The FDA approval of fostamatinib arms physicians with a new treatment option, which works via a novel mechanism.”
FDA also okayed burosumab-twza (Crysvita), the first drug approved to treat adults and children one year and older with x-linked hypophosphatemia (XLH), a rare, inherited form of rickets.
XLH is a serious disease affecting approximately 3,000 children and 12,000 adults in the United States.
“XLH differs from other forms of rickets in that vitamin D therapy is not effective,” stated Julie Beitz, MD, director of the Office of Drug Evaluation in FDA’s Center for Drug Evaluation and Research, in a FDA statement. “This is the first FDA-approved medication for the treatment of XLH and a real breakthrough for those living with this serious disease.”
The safety and efficacy of Crysvita were studied in 4 clinical trials. In the placebo-controlled trial, 94% of adults receiving Crysvita once a month achieved normal phosphorus levels compared to 8% of those receiving placebo. In children, 94% to 100% of patients treated with Crysvita every 2 weeks achieved normal phosphorus levels. In both children and adults, X-ray findings associated with XLH improved with Crysvita therapy, FDA said.