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.

Brukinsa Approved for Relapsed, Refractory Mantle Cell Lymphoma

MONDAY, Nov. 18, 2019 -- Brukinsa (zanubrutinib), a kinase inhibitor, has been granted accelerated approval for the treatment of adults with mantle cell lymphoma who have received at least one previous therapy, the U.S. Food and Drug Administration announced last week.

The recommended dose of Brukinsa capsules is 160 mg orally twice daily or 320 mg orally once daily. The prescribing information lists warnings and precautions for hemorrhage, infections, cytopenias, second primary malignancies, cardiac arrhythmias, and embryo-fetal toxicity.

Fetroja Approved to Treat Complicated Urinary Tract Infections

FRIDAY, Nov. 15, 2019 -- Antibacterial drug Fetroja (cefiderocol) has been approved to treat patients aged 18 years and older with complicated urinary tract infections (cUTIs), the U.S. Food and Drug Administration announced yesterday.

The drug is indicated for patients with limited or no alternative treatment options for cUTI, including kidney infections caused by susceptible Gram-negative microorganisms. Dosage is 2 g of Fetroja every eight hours administered by intravenous infusion over three hours. Dose adjustments are required based on creatinine clearance. Prescription labeling on Fetroja includes a warning for a higher all-cause mortality rate in Fetroja-treated critically ill patients with multidrug-resistant Gram-negative bacterial infections.

Reblozyl Approved to Treat Anemia in Patients With Beta Thalassemia

WEDNESDAY, Nov. 13, 2019 -- Reblozyl (luspatercept-aamt) has been approved to treat anemia in adults with beta thalassemia who require regular red blood cell (RBC) transfusions, the U.S. Food and Drug Administration announced Friday.

Reblozyl is the first approved therapy that will help to reduce the number of blood transfusions for this patient population, 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, said in a statement. Reblozyl is administered through a subcutaneous injection with a recommended starting dose of 1 mg/kg once every three weeks.

Implant Approved for Patients With Phototoxic Reactions From Rare Disorder

WEDNESDAY, Oct. 9, 2019 -- The U.S. Food and Drug Administration has approved Scenesse (afamelanotide), a melanocortin-1 receptor agonist, for adult patients with a history of phototoxic reactions from erythropoietic protoporphyria, the agency announced yesterday.

Scenesse is a subcutaneous implant designed to increase pain-free light exposure in this rare patient population. The implant increases the production of eumelanin in the skin independent of exposure to sunlight or artificial light.