FDA Approves First Drug to Treat Pediatric Neurofibromatosis Type 1

The Food and Drug Administration (FDA) approved the first drug, Koselugo (selumetinib), to treat pediatric patients aged 2 years or older with neurofibromatosis type 1 (NF1), a rare genetic disorder that causes tumors to grow on nerves, according to the agency.

NF1, which is usually diagnosed early in about one in every 3,000 infants, is a progressive disease. Between 30% and 50% of patients with NF1 have at least 1 or more plexiform neurofibromas (PNs) — tumors involving nerve sheaths that can grow throughout the body and affect organs.

The approval was based off a National Cancer Institute study involving 50 patients, who induced an overall response rate of 66%. All patients had a partial response, and no patients had a complete disappearance of the tumor.

Of these patients, 82% had a response lasting 12 months or longer.

Common side effects of Koselugo included vomiting, rash, abdominal pain, diarrhea, nausea, dry skin, fatigue, musculoskeletal pain (pain in the body affecting bones, muscles, ligaments, tendons and nerves), fever, acneiform rash (acne), stomatitis (inflammation of the mouth and lips), headache, paronychia (infection in the skin that surrounds a toenail or fingernail) and pruritus (itching).

“Everyone’s daily lives have been disrupted during the COVID-19 pandemic, and in 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,” Dr. Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Oncologic Diseases in the FDA’s Center for Drug Evaluation and Research, said in a press release. “We continue to expedite product development for these patients.”