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FDA OKs First Drug for Pediatric Neurofibromas

WASHINGTON -- The FDA approved selumetinib (Koselugo) on Friday for the treatment of plexiform neurofibromas in children with neurofibromatosis type 1 (NF1).

NF1 is a genetic condition that leads to the growth of tumors throughout the body, that while benign can affect patients' organs and be painful and debilitating. Selumetinib, a mitogen-activated protein kinase (MAPK) kinase (MEK) inhibitor, is indicated for children 2 years and older with symptomatic and inoperable plexiform neurofibromas, tumors of the nerve sheaths.

"[I]n this critical time we want patients to know that the FDA remains committed to making patients with rare tumors and life threatening diseases, and their unique needs, a top priority," Richard Pazdur, MD, the FDA's top oncology/hematology official, said in a statement. "For the first time, pediatric patients now have an FDA-approved drug to treat plexiform neurofibroma, a rare tumor associated with NF1."

Approval was based on a phase II study of 50 NF1 patients with inoperable plexiform neurofibromas who received the drug's recommended oral dose (25 mg/m2 twice daily) until disease progression or unacceptable toxicity. Two-thirds of patients achieved a partial response to selumetinib, defined as a 20% reduction in tumor volume on MRI at 6 months, and 82% of these patients had responses lasting a year or more.

Patient-reported quality-of-life scores also improved for 29 patients during treatment, as did parent-reported scores for 45 patients.

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COVID-19 ICU Drug Shortages Redressed, But Only in Part

As the federal government took steps to ease shortages of some critical sedatives and other drugs used in the ICU, pharmacy and anesthesiology groups say more needs to be done.

On Tuesday, the Drug Enforcement Administration loosened restrictions on controlled substances needed for the ICU treatment of COVID-19, increasing by 15% the allowed production volumes of fentanyl, morphine, hydromorphone, codeine, ephedrine, pseudoephedrine, and certain intermediates for their production. The agency also boosted the amount of ketamine, diazepam, midazolam, lorazepam, and phenobarbital that can be imported.

More action is being sought, though.

The American Society of Anesthesiologists (ASA), for example, is calling for the FDA to work with manufacturers to extend expiration dates of critical products.

The ASA threw the spotlight most urgently on the ICU sedative propofol. "With the increased demand for this drug and the number of COVID-19 positive patients needing ventilators, we can only anticipate that this will soon be in shortage," the group said in an open letter to the FDA.

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First Report of COVID-19 Neurologic Symptoms in China

More than a third of 214 confirmed COVID-19 cases in China had neurologic symptoms, researchers said.

Acute cerebrovascular events, impaired consciousness, and muscle injury were seen in 36.4% of patients and were more common (45.5%) in patients with severe infection who required mechanical ventilation, reported Bo Hu, MD, PhD, of Union Hospital and Huazhong University of Science and Technology in Wuhan, and colleagues.

Neurologic symptoms included central nervous system (CNS) manifestations such as dizziness, headache, impaired consciousness, acute cerebrovascular disease, ataxia, or seizure; peripheral nervous system manifestations such as taste and smell impairment, vision impairment, or nerve pain; and skeletal muscular injury manifestations.

"For those with severe COVID-19, rapid clinical deterioration or Worsening could be associated with a neurologic event such as stroke, which would contribute to its high mortality rate," the team wrote in JAMA Neurology. "During the epidemic period of COVID-19, when seeing patients with these neurologic manifestations, clinicians should consider SARS-CoV-2 [the virus that causes COVID-19] infection as a differential diagnosis to avoid delayed diagnosis or misdiagnosis and prevention of transmission."

COVID-19 and severe acute respiratory syndrome (SARS), which first appeared in China in late 2002, are similar in many ways clinically, noted S. Andrew Josephson, MD, of the University of California San Francisco, and colleagues, in an accompanying editorial.

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