NF1 is a rare genetic disorder caused by a mutation or flaw in a particular gene. Some children with NF1 develop benign (noncancerous) tumors called plexiform neurofibromas (PN), which can grow along nerves. Because these PN are caused by NF1, they are referred to as NF1 PN.

The location and symptoms of your child's PN on the body will help determine which doctors are best to see for treatment. Oncologists, or medical doctors who specialize in treating cancerous and noncancerous tumors, can treat NF1 PN or refer you and your child to other NF1 PN specialists. These specialists may include geneticists, scientists who specialize in the study of genes and heredity; neurologists, medical doctors who specialize in treating disease of the nervous system; ophthalmologists, medical doctors who treat eyes and vision; orthopedists, medical doctors who treat bone and mobility issues; and others. Work with your child's doctor to determine the right set of specialists for your child's care team.

Koselugo is the first and only prescription medicine proven to shrink NF1 PN in pediatric patients 2 years of age or older with neurofibromatosis type 1 (NF1) who have plexiform neurofibromas that cannot be completely removed by surgery. It is not known if Koselugo is safe and effective in children under 2 years of age. Learn more about taking Koselugo.

Koselugo is not chemotherapy. Koselugo is an oral, targeted treatment option proven to shrink PN in children 2 years of age and older with NF1 who have plexiform neurofibromas that cannot be completely removed by surgery.

After prescribing Koselugo, your child’s doctor will monitor their response to treatment and perform periodic testing and will determine the appropriate length of therapy. Treatment duration of Koselugo can be different for every patient, so it’s important to carefully follow the instructions provided by your child’s doctor.

It is available only through specialty pharmacies and is not available at your local retail pharmacy. For more specific information about specialty pharmacies and other distribution channels that carry Koselugo and to learn more about insurance coverage and out-of-pocket costs, visit www.alexiononesource.com.

The Alexion OneSource™ program can provide information about financial support and resources that may be available for eligible patients. To learn more, please visit www.alexiononesource.com.

Your child’s doctor will decide if Koselugo is right for your child. Koselugo is a prescription medicine that is used to treat children 2 years of age and older with NF1 who have plexiform neurofibromas that cannot be completely removed by surgery. It is not known if Koselugo is safe and effective in children under 2 years of age. Learn about Koselugo.

Koselugo is an oral medication taken twice daily, about 12 hours apart. Koselugo capsules must only be swallowed whole with water. Koselugo can be taken on an empty stomach or with food. They must not be chewed, dissolved, or opened. Taking Koselugo in a different way than instructed may affect how it works. Learn more about taking Koselugo.

Tell your doctor if your child has trouble swallowing capsules. To learn more or to contact your dedicated Case Manager, please call 1-888-765-4747, Monday through Friday, 8:30 AM–8 PM ET, or visit www.alexiononesource.com/kos. To learn how to help your child take capsules more easily, visit our Dosing Information page.

No, Koselugo capsules must be swallowed whole with water. Do not let your child chew, dissolve, or open a capsule. Tell your child’s doctor if your child has trouble swallowing a capsule. To learn more, visit the Taking Koselugo page.

The side effects of Koselugo are well-known, can be manageable, and may not require stopping or delaying treatment. The most common side effects of Koselugo are vomiting, stomach-area pain, nausea, dry skin, muscle and bone pain, feeling of tiredness or lacking energy, fever, sores in your mouth, headache, redness around the fingernails, itching. Please visit our Safety page to learn more.

Of the 33 patients that responded to Koselugo treatment in the clinical trial, 97% (32/33) saw their tumor volume shrink by at least 20% within 1 year. The time to onset of response ranged from 3.3 months to 1.6 years. It’s important to know that results are different for each patient. Your child’s experience may be different. Your child’s doctor will determine if Koselugo is working for your child. Learn the results of pediatric patients taking Koselugo in a clinical study.

If your child misses a dose, they should take it as soon as you remember. If it is less than 6 hours before the next scheduled dose, give the next dose at the regular time. Do not make up for the missed dose. If your child vomits at any time after taking Koselugo, they should not take an additional dose. The next dose should be taken at the regular time. To learn more about taking Koselugo, visit our taking Koselugo page.

Your child can take Koselugo with or without food. To learn more about taking Koselugo, visit our Taking Koselugo page.