Drugs

AstraZeneca’s Koselugo OK’d for Pediatric Neurofibromatosis

FDA has approved an AstraZeneca NDA for Koselugo (selumetinib) for treating of pediatric patients with neurofibromatosis type 1 (NF1), a nervous system genetic disorder that causes tumors to grow on nerves. FDA says that Koselugo is approved specifically for patients who have symptomatic, inoperable plexiform neurofibromas (PN), which are tumors involving the nerve sheaths that can grow anywhere in the body.

NF1 is a mutation or flaw in a particular gene, and it is usually diagnosed in early childhood and appears in an estimated 1 out of every 3,000 infants, FDA says. It approved Koselugo based on a clinical trial conducted by the National Cancer Institute involving 50 patients who had routine evaluations of changes in tumor size and tumor-related morbidities during the trial. The overall response rate was 66% and all patients had a partial response, meaning that no patients had complete disappearance of the tumor, the agency says, adding that “82% had a response lasting 12 months or longer. Other clinical outcomes for patients during Koselugo treatment including changes in PN-related disfigurement, symptoms and functional impairments. Although the sample sizes of patients assessed for each PN-related morbidity (such as disfigurement, pain, strength and mobility problems, airway compression, visual impairment and bladder or bowel dysfunction) were small, there appeared to be a trend of improvement in PN-related symptoms or functional deficits during treatment.”

On the safety side, FDA says common side effects observed were vomiting, rash, abdominal pain, diarrhea, nausea, dry skin, fatigue, musculoskeletal pain, fever, acneiform rash, stomatitis, headache, paronychia and pruritus. Because the drug can cause serious side effects including heart failure and ocular toxicity, the agency recommends that patients should have cardiac and ophthalmic assessments performed prior to initiating Koselugo and at regular treatment intervals.

Gupta Raises Concerns About Overton’s Independence, Ideology

FDA commissioner nominee Heidi Overton’s responses on vaccines, contraception and abortion medication during her 9/24 Senate confirmation hearing raise concerns that ideology could influence her decision-making if she takes over the agency, MSNBC senior medical analyst Dr. Vin Gupta said Friday. Gupta was particularly critical of Overton’

Overton Confirmation Remains Uncertain After Senate Hearing

FDA commissioner nominee Heidi Overton emerged from her Senate confirmation hearing Thursday (see earlier story) without resolving questions about whether she has enough Republican support to advance — and with a rapidly narrowing window for confirmation before the midterm elections. The Senate Health, Education, Labor and Pensions Committee hearing provided few

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA has classified Boston Scientific's recall of its Imager II Angiographic Catheters as Class I because a manufacturing problem could cause catheter tips to degrade and detach, potentially resulting in life-threatening complications. The agency announced the Class I classification 9/25, updating an Early Alert it issued

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome

FDA has approved Roche’s Gazyva (obinutuzumab) for reducing the risk of relapse in adults and children aged 2 and older with frequently relapsing or steroid-dependent, childhood-onset idiopathic nephrotic syndrome who are in remission. The expanded approval covers the use of the CD20-directed antibody beyond its existing

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Gupta Raises Concerns About Overton’s Independence, Ideology

Overton Confirmation Remains Uncertain After Senate Hearing

FDA Classifies Boston Scientific Imager II Catheter Recall as Class I

FDA Approves Gazyva for Childhood-Onset Idiopathic Nephrotic Syndrome