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.

Rituxan Approved for Pediatric Patients With Rare Vasculitis Diseases

TUESDAY, Oct. 1, 2019 -- Rituxan (rituximab) injection was granted the first approval for a drug to treat granulomatosis with polyangiitis (GPA) and microscopic polyangiitis (MPA) in combination with glucocorticoids in children 2 years and older, the U.S. Food and Drug Administration announced Friday.

The agency noted that pediatric patients with GPA, previously known as Wegener's granulomatosis, and MPA have a safety profile that is consistent with that of the known safety profile of Rituxan in adults with autoimmune diseases, including GPA and MPA.

Mavyret Approved as 8-Week Treatment for Hep C, Compensated Cirrhosis

MONDAY, Sept. 30, 2019 -- Approval of Mavyret (glecaprevir and pibrentasvir) tablets has been expanded to eight-week treatment for treatment-naive patients aged 12 years and older with chronic hepatitis C virus (HCV) genotypes 1 through 6 and compensated cirrhosis, the U.S. Food and Drug Administration announced last week.

Mavyret is the first eight-week treatment approved for any treatment-naive HCV patients, regardless of cirrhosis status or treatment type. The drug is now approved for all treatment-naive adult patients and certain pediatric patients (weighing at least 99 pounds) without cirrhosis and with compensated cirrhosis. Standard treatment length for compensated cirrhosis was at least 12 weeks.

FDA Approves Vaccine for Prevention of Smallpox, Monkeypox

WEDNESDAY, Sept. 25, 2019 -- Jynneos Smallpox and Monkeypox Vaccine, Live, Non-Replicating, was approved by the U.S. Food and Drug Administration for prevention of smallpox and monkeypox disease in adults 18 years or older who are considered at high risk for infection, the agency announced this week.

Jynneos, which is administered in two doses four weeks apart, contains Modified Vaccinia Ankara, a form of the vaccinia virus closely related to the variola or monkeypox viruses but less harmful. To ensure it is accessible in the United States if needed, Jynneos is part of the Strategic National Stockpile, a national supply of pharmaceuticals and medical supplies for use in a public health emergency that depletes local supplies.

FDA Approves First Treatment for ILD With Systemic Sclerosis, Scleroderma

MONDAY, Sept. 9, 2019 -- The U.S. Food and Drug Administration granted the first approval for a drug to slow the decline of pulmonary function in interstitial lung disease associated with systemic sclerosis or scleroderma (SSc-ILD), the agency announced Friday.

Ofev (nintedanib) capsules were approved in 2014 to treat adults with idiopathic pulmonary fibrosis and are now approved for patients with SSc-ILD. The recommended dosage is 150 mg twice daily 12 hours apart.

Nourianz Approved to Treat 'Off' Episodes in Parkinson Disease

WEDNESDAY, Aug. 28, 2019 -- Nourianz (istradefylline) tablets have been approved as an add-on treatment to levodopa/carbidopa for adults with Parkinson disease experiencing "off" episodes, the U.S. Food and Drug Administration announced yesterday.

The drug is available in 20-mg or 40-mg doses, but the maximum recommended dosage in patients taking CYP3A4 inhibitors and those with moderate hepatic impairment is 20 mg once daily. The safety information for Nourianz states that use of the drug should be avoided in these patient populations.

Rozlytrek Approved to Treat Tumors With NTRK Gene Fusion

MONDAY, Aug. 19, 2019 -- Rozlytrek (entrectinib), a kinase inhibitor, has received approval to treat adults and adolescents with cancer that carries the genetic defect neurotrophic tyrosine receptor kinase (NTRK) gene fusion and has progressed following other treatment, the U.S. Food and Drug Administration announced last week.

The drug was also approved to treat adults with metastatic non-small cell lung cancer and ROS1-positive tumors. The recommended dose for adults with either indication -- NTRK gene fusion-positive tumors or ROS1-positive non-small cell lung cancer -- is 600 mg orally once daily. Dosage in adolescent patients aged 12 years and older is based on body surface area.

Implant Approved to Improve Symptoms in Advanced Heart Failure

MONDAY, Aug. 19, 2019 -- The Barostim Neo System was granted U.S. Food and Drug Administration approval to treat symptoms of patients with advanced heart failure who are not candidates for treatment with other devices such as cardiac resynchronization therapy, the agency announced on Friday.

The device includes a pulse generator implanted below a patient's clavicle and connected to a lead attached to the carotid artery. A physician tests and programs the device after implantation. The Barostim Neo System delivers electrical impulses to baroreceptors to ultimately inhibit production of stress-related hormones and reduce heart failure symptoms. Indications for treatment with the device include regular heart rhythm, not being a candidate for cardiac resynchronization therapy, and left ventricular ejection fraction of ≤35 percent. Contraindications include anatomy that could impair device implantation, certain nervous system disorders, uncontrolled and symptomatic slow heart rate, atherosclerosis, and a known allergy to silicone or titanium.

New Antibiotic Approved for Community-Acquired Bacterial Pneumonia

MONDAY, Aug. 19, 2019 -- Xenleta (lefamulin) has been approved to treat adults with community-acquired bacterial pneumonia, the U.S. Food and Drug Administration announced today.

Dosing of Xenleta is either an oral administration of 600 mg every 12 hours or an intravenous administration of 150 mg every 12 hours for five to seven days. Patients can be started on either intravenous or oral therapy or can transition from intravenous to oral therapy to accelerate hospital discharge.

FDA Approves Inrebic Capsules for Myelofibrosis

FRIDAY, Aug. 16, 2019 -- Inrebic (fedratinib) capsules have been approved to treat adults with intermediate-2 or high-risk primary or secondary myelofibrosis, making it the second drug approved to treat patients with this disease, the U.S. Food and Drug Administration announced today.

Approval of Inrebic for patients with intermediate-2 or high-risk primary or secondary (post-polycythemia vera or post-essential thrombocythemia) myelofibrosis was based on clinical trial data from 289 patients randomly assigned to 400 or 500 mg of oral Inrebic daily or placebo. Thirty-six percent of patients (35 of 96) treated with the label-recommended dose of 400 mg of Inrebic had experienced at least a 35 percent reduction in spleen volume from baseline to week 24 as measured by magnetic resonance imaging or computed tomography scan. Thirty-six patients treated with Inrebic had at least a 50 percent reduction in myelofibrosis-related symptoms, including night sweats, itching, abdominal discomfort, feeling full sooner than normal, rib pain on the left side, and bone or muscle pain.