Ayvakit Approved to Treat GIST With PDGFRA Exon 18 Mutation

FRIDAY, Jan. 10, 2020 -- The kinase inhibitor Ayvakit (avapritinib) has been approved to treat adults with unresectable or metastatic gastrointestinal stromal tumors (GISTs) with a platelet-derived growth factor receptor alpha (PDGFRA) exon 18 mutation, the U.S. Food and Drug Administration announced Thursday.

The approval includes GISTs harboring a PDGFRA D842V mutation, the most common exon 18 mutation, and marks the first time a drug is specifically approved for GISTs harboring the PDGFRA exon 18 mutation, which is involved in about 10 percent of GIST cases.

Enhertu Approved for Unresectable, Metastatic HER2+ Breast Cancer

MONDAY, Dec. 23, 2019 -- Enhertu (fam-trastuzumab deruxtecan-nxki) has received accelerated approval for treatment of unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-positive breast cancer, the U.S. Food and Drug Administration announced Friday.

Enhertu, a HER2-directed antibody and topoisomerase inhibitor conjugate, is indicated for adults with unresectable or metastatic HER2-positive breast cancer who have received at least two previous anti-HER2-based regimens in the metastatic setting. The recommended dosage of Enhertu is an intravenous infusion of 5.4 mg/kg once every three weeks (21-day cycle) until disease progression or unacceptable toxicity.

FDA Approves Padcev for Treatment of Advanced Urothelial Cancer

THURSDAY, Dec. 19, 2019 -- Padcev (enfortumab vedotin-ejfv), a Nectin-4-directed antibody and microtubule inhibitor conjugate, has received accelerated approval for the treatment of locally advanced or metastatic urothelial cancer, the U.S. Food and Drug Administration announced yesterday.

Padcev is indicated for adults whose disease has progressed while receiving standard treatment with a programmed death receptor-1 (PD-1) or programmed death ligand 1 (PD-L1) inhibitor and a platinum-based therapy.

FDA Authorizes Marketing of Automated Insulin Dosing Controller

THURSDAY, Dec. 19, 2019 -- The U.S. Food and Drug Administration recently approved marketing of an interoperable automated glycemic controller device, the Tandem Diabetes Care Control-IQ Technology, the agency announced last week.

The device automatically adjusts insulin delivery through connection to an alternate controller-enabled insulin pump (ACE pump) and integrated continuous glucose monitor (iCGM). According to the FDA, this controller is the first that can be used with other diabetes devices that are meant to be integrated into a diabetes management system for automated insulin delivery. With this marketing authorization, the FDA notes iCGMs and ACE pumps can be used with an interoperable automated glycemic controller as a complete automated insulin dosing (AID) system. Patients with type 1 diabetes can use the Control-IQ Technology controller with compatible iCGMs and ACE pumps to automatically increase, decrease, and suspend delivery of basal insulin based on insulin delivery history, iCGM readings, and predicted glucose values. The controller can also automatically deliver insulin when the glucose value is predicted to exceed a predefined number.

FDA Approves Oxbryta for Treatment of Sickle Cell Disease

TUESDAY, Nov. 26, 2019 -- Oxbryta (voxelotor) has been granted accelerated approval for the treatment of sickle cell disease in patients ages 12 years and older, the U.S. Food and Drug Administration announced Monday.

Oxbryta is the first approved treatment for sickle cell disease that directly inhibits the root cause of the disease, sickle hemoglobin polymerization, according to a company press release. Nonclinical studies of Oxbryta have shown the drug inhibits red blood cell sickling and improves red blood cell deformability and the ability of blood to flow. The recommended dosage of Oxbryta is 1,500 mg orally once daily with or without food. For severe hepatic impairment, the recommended dosage is 1,000 mg orally once daily.

FDA Approves First System to Insert Ear Tubes Under Local Anesthesia

TUESDAY, Nov. 26, 2019 -- The U.S. Food and Drug Administration has approved the first tympanostomy tube delivery system that can be performed under local anesthesia in a physician's office, the agency announced Monday.

The Tubes Under Local Anesthesia (Tula) System is indicated for inserting tympanostomy tubes for the treatment of otitis media in adults and children ages 6 months and older. The system includes the anesthetic Tymbion, Tusker Medical tympanostomy tubes, and devices needed for delivery of the ear tubes and the anesthetic into the ear drum. The Tula System uses a small electrical current to deliver the local anesthetic into the ear drum before tube insertion.

FDA Approves XCOPRI for Treatment of Partial-Onset Seizures

MONDAY, Nov. 25, 2019 -- XCOPRI (cenobamate) has received approval for the treatment of partial-onset seizures in adults, the U.S. Food and Drug Administration announced last week.

Following a titration period, the recommended maintenance dose of XCOPRI is 200 mg by mouth daily, but some patients may need an additional titration to the maximum recommended dose of 400 mg daily based on their individual clinical response and tolerance, the FDA noted.

Calquence Approved to Treat CLL, SLL

FRIDAY, Nov. 22, 2019 -- The U.S. Food and Drug Administration has granted supplemental approval to Calquence (acalabrutinib) for treatment of chronic lymphocytic leukemia (CLL) or small lymphocytic lymphoma (SLL), the agency announced Thursday.

Calquence was initially approved in 2017 for treatment of adults with previously treated mantle cell lymphoma. It is now approved as a new treatment option or as an initial or subsequent therapy for adults with CLL or SLL. The latest approval of Calquence is part of Project Orbis, a collaboration among the FDA, the Australian Therapeutic Goods Administration, and Health Canada. Through Project Orbis, oncology drug applications can be concurrently submitted for review among the FDA and its international partners, according to Richard Pazdur, M.D., director of the FDA Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA Center for Drug Evaluation and Research.

Givlaari Approved for Acute Hepatic Porphyria

FRIDAY, Nov. 22, 2019 -- On Wednesday, the U.S. Food and Drug Administration announced the approval of Givlaari (givosiran) for the treatment of acute hepatic porphyria.

Previous treatment options for this genetic disorder have only provided partial relief from the pain of porphyria attacks, according to Richard Pazdur, M.D., director of the FDA Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA Center for Drug Evaluation and Research. Givlaari helps to reduce the number of attacks. The recommended dose of Givlaari is a subcutaneous injection of 2.5 mg/kg once monthly.

FDA Approves First Contact Lens That Slows Myopia Progression

MONDAY, Nov. 18, 2019 -- MiSight, the first contact lens indicated to slow the progression of myopia in children ages 8 to 12 years, has been approved by the U.S. Food and Drug Administration, the agency announced Friday.

The single-use, disposable, soft contact lenses should be discarded after one-day use and are not intended to be worn overnight. They are indicated to correct and slow progression of myopia in children with healthy eyes, the FDA noted. Like a standard corrective lens, one part of the MiSight contact lens corrects the refractive error to improve distance vision. Concentric peripheral rings in the lens also focus part of the light in front of the retina to reduce the stimulus causing myopia progression.