MONDAY, May 18, 2020 -- The U.S. Food and Drug Administration approved Qinlock (ripretinib) tablets as a fourth-line treatment for adults with advanced gastrointestinal stromal tumors (GISTs), the agency announced Friday.
The approval follows four other FDA-approved target therapies for GIST, including imatinib in 2002, sunitinib in 2006, regorafenib in 2013, and avapritinib in January. Qinlock is indicated for patients who have received at least three kinase inhibitor therapies, including imatinib.
MONDAY, May 11, 2020 -- Retevmo (selpercatinib) capsules have been approved to treat patients with non-small cell lung cancer (NSCLC), medullary thyroid cancer (MTC), and other thyroid cancers with an alteration in the "rearranged during transfection" (RET) gene, the U.S. Food and Drug Administration announced Friday.
Retevmo was approved for adults with metastatic NSCLC and patients aged 12 years and older with advanced or metastatic MTC requiring systemic therapy or advanced RET fusion-positive thyroid cancer requiring systemic therapy that has stopped responding to or is not appropriate for radioactive iodine (RAI) therapy. Before a patient initiates Retevmo treatment, presence of the RET gene alteration must be identified through laboratory testing.
WEDNESDAY, May 6, 2020 -- The U.S. Food and Drug Administration has granted approval for Farxiga (dapagliflozin) oral tablets to treat adults with heart failure with reduced ejection fraction, the agency announced Tuesday.
Farxiga is the first approved sodium-glucose cotransporter 2 inhibitor to treat adults with New York Heart Association functional class II to IV heart failure with reduced ejection fraction. The drug is indicated to reduce the risk for cardiovascular death and hospitalization for heart failure in this patient population. Farxiga has already been FDA-approved to improve glycemic control in patients with type 2 diabetes and to reduce the risk for hospitalization in patients with type 2 diabetes and known cardiovascular disease or other risk factors.
THURSDAY, April 23, 2020 -- Accelerated approval has been granted to Trodelvy (sacituzumab govitecan-hziy) to treat patients with metastatic triple-negative breast cancer who have received at least two previous therapies, the U.S. Food and Drug Administration announced Wednesday.
Approval of Trodelvy, a Trop-2-directed antibody and topoisomerase inhibitor drug conjugate, was based on clinical trial data from 108 breast cancer patients with metastatic triple-negative disease. Researchers found an overall response rate of 33.3 percent and a median response duration of 7.7 months. Response was maintained for six months or longer in 55.6 percent of patients who responded, and 16.7 percent of those who responded maintained response for one year or longer.
MONDAY, April 20, 2020 -- The U.S. Food and Drug Administration announced Friday the approval of Tukysa (tucatinib) in combination with trastuzumab and capecitabine for treatment of advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-positive breast cancer.
Tukysa, a kinase inhibitor, is indicated for patients who have already received treatment with at least one anti-HER2-based regimen in the metastatic setting. Approval was based on results of the HER2CLIMB clinical trial, which enrolled 612 patients with HER2-positive advanced unresectable or metastatic breast cancer who had previously received treatment with trastuzumab, pertuzumab, and ado-trastuzumab emtansine (T-DM1). Forty-eight percent of patients had brain metastases at trial start.
MONDAY, April 20, 2020 -- Pemazyre (pemigatinib) has been granted accelerated approval for the treatment of advanced cholangiocarcinoma, the U.S. Food and Drug Administration announced Friday.
The drug approval is indicated for previously treated patients with locally advanced or metastatic cholangiocarcinoma and tumors with a fusion or other rearrangement of fibroblast growth factor receptor 2 (FGFR2). Until now, the standard initial treatment for these patients has been a combination of chemotherapy.
TUESDAY, April 14, 2020 -- Koselugo (selumetinib) has received the first approval for treatment of neurofibromatosis type 1 (NF1) in children ages 2 years and older, the U.S. Food and Drug Administration announced Friday.
Approved specifically for patients with symptomatic, inoperable plexiform neurofibromas (PNs), Koselugo, a kinase inhibitor, works by blocking a key enzyme to help stop the tumor from growing. The FDA notes that NF1 occurs in one of every 3,000 infants, and 30 to 50 percent of patients born with NF1 develop at least one PN.
FRIDAY, March 20, 2020 -- Epclusa (sofosbuvir and velpatasvir) received approval for a supplemental application to treat hepatitis C virus (HCV) in children without cirrhosis or with mild cirrhosis, the U.S. Food and Drug Administration announced Thursday.
The supplemental application of Epclusa is indicated for children ages 6 years and older or weighing at least 37 lb who have HCV genotypes 1, 2, 3, 4, or 6. Epclusa combined with ribavirin is indicated for treating children 6 years and older or at least 37 lb with severe cirrhosis, and the FDA previously approved the drug to treat HCV in adults.
TUESDAY, March 10, 2020 -- Ofev (nintedanib) oral capsules have received the first approval for the treatment of patients with chronic fibrosing interstitial lung diseases (ILDs) with a progressive phenotype, the U.S. Food and Drug Administration announced Monday.
Ofev, a multitargeted tyrosine kinase inhibitor, was approved in 2014 to treat idiopathic pulmonary fibrosis and to slow pulmonary function decline among patients with ILD associated with systemic sclerosis or scleroderma.
MONDAY, March 9, 2020 -- Isturisa (osilodrostat) oral tablets have been approved to treat adults with Cushing disease, the U.S. Food and Drug Administration announced Friday.
Isturisa is indicated in adults with Cushing disease who are not candidates for pituitary gland surgery or who have had the surgery but still have the disease. The drug works by blocking the enzyme 11-beta-hydroxylase and preventing cortisol synthesis. Isturisa is taken orally twice a day, in the morning and evening. After treatment initiation, health care providers may reevaluate dosage based on the patient's response.