Why Koselugo

Sam, age 10, living with NF1 PN.
Sam is a Koselugo patient.

What is Koselugo?

KOSELUGO® (selumetinib) is the FIRST and ONLY FDA-approved oral medication proven to shrink neurofibromatosis type 1 (NF1) plexiform neurofibromas (PN) when PN cannot be completely removed by surgery.

You may know Koselugo as selumetinib. Koselugo is the brand name for selumetinib. When you see either of these names, it's important to know that they are referring to the same medication.

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Can Koselugo help shrink plexiform neurofibromas?

In the SPRINT study, Koselugo was proven to shrink PN in children 2 years and older with NF1 PN that couldn't be completely removed by surgery.*

The SPRINT study was conducted by the National Cancer Institute (NCI) to test Koselugo in NF1 patients who could not have their PN removed by surgery.

In this clinical study, 66% (33/50) of children treated with Koselugo saw their PN shrink by at least 20%.** Almost all children taking Koselugo saw their tumor shrink or stay about the same.† This study is still ongoing and continues to collect data on the long-term safety and effectiveness of Koselugo.

Stat

In the SPRINT study, 66% (33/50) of children treated with Koselugo saw their NF1 PN shrink by at least 20%**

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Of the children who responded to treatment with Koselugo, 97% (32/33) saw results within one year.**

For most children who saw their PN shrink, Koselugo kept working for more than 3 years.‡

Of the children whose NF1 PN shrank 20% or more with Koselugo:

79%

(26/33) maintained their results for at least 2 years‡

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64%

(21/33) maintained their results for at least 3 years‡

Some children maintained their results for more than 4.5 years.§

Your child's doctor may refer to this as “duration of response,” which is the amount of time that patients in the study who achieved ≥20% tumor shrinkage maintained that shrinkage.

*Confirmed by 3D magnetic resonance imaging (MRI) scan.
**Data cutoff June 2018. The median time to onset of response was 7.2 months (range: 3.3 months to 1.6 years).
†"Stay about the same" is defined as no more than a 20% increase in tumor volume.
‡Data cutoff March 2021.
§Data cutoff February 2021. This information is from the SPRINT long-term follow-up study.

Has the safety of Koselugo been studied?

The safety of Koselugo was studied for up to 7.7 years‖¶

Side effects are well-known, can be manageable, and may not require stopping or delaying treatment with Koselugo.

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In the SPRINT Phase 1 and 2 studies,‖ half of patients took Koselugo for more than 4.4 years. Some patients in the study are still taking Koselugo and the longest treatment period is yet to be determined.

In the SPRINT Phase 2 Stratum 1 study, as of June 2018, children experienced mostly mild or moderate side effects while taking Koselugo.

76%

(38/50) of children were able to stay on a full dose of Koselugo without having to reduce their dose

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80%

(40/50) of children required a dosing pause but were able to avoid stopping treatment

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12%

(6/50) of children permanently stopped treatment due to side effects

    ‖

    Data cutoff February 2021. Based on the long-term follow-up of the SPRINT studies, the evaluation of Koselugo is run by the National Cancer Institute and is designed to test the efficacy and safety of Koselugo. These data reflect exposure to Koselugo in 74 pediatric patients with NF1 PN that could not be removed by surgery without risk of issues because of their location or size. These patients received a dosage ranging from 20 mg/m2 to 30 mg/m2 orally twice daily in SPRINT.

    The median duration of Koselugo exposure is 4.4 years (range: 28 days to 7.7 years). Median is defined as the middlemost point in a dataset. In this case, 50% of patients received Koselugo treatment for less than 4.4 years, while the other 50% received Koselugo treatment for more than 4.4 years.

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What are the most common side effects of Koselugo?

What are the most common side effects of Koselugo?

The most common side effects include:


  • 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
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These are not all the possible side effects of Koselugo. Your child’s doctor may change your child’s dose, temporarily stop, or permanently ask your child to stop taking Koselugo if they experience certain side effects.

Remember, the information on this page does not replace medical guidance or directions from your child’s doctor. Always talk to them about side effects and ways to manage them.

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Is there a support program to help my child access treatment?

Koselugo comes with a team to support you at every step.

Learn about OneSource

Discover more about Koselugo including tips and resources when starting treatment.

Find out more

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important safety information

What are the possible side effects of Koselugo?

Koselugo may cause serious side effects, including:

  • Heart problems. Koselugo can lower the amount of blood pumped by your heart, which is common and can also be severe. Your healthcare provider will do tests before and during treatment to check how well your heart is working. Tell your healthcare provider right away if you get any of the following signs or symptoms: persistent coughing or wheezing, shortness of breath, swelling of your ankles and feet, tiredness, increased heart rate.
  • Eye problems. Koselugo can cause eye problems that can lead to blindness. Your healthcare provider will check your vision before and during treatment. Tell your healthcare provider right away if you get any of the following signs or symptoms: blurred vision, loss of vision, dark spots in your vision (floaters), other changes to your vision.
  • Severe diarrhea. Diarrhea is common with Koselugo and can also be severe. Tell your healthcare provider right away the first time that you get diarrhea during treatment. Your healthcare provider may give you medicine to help control your diarrhea and may tell you to drink more fluids.
  • Skin rash. Skin rashes are common with Koselugo and can also be severe. Tell your healthcare provider if you get any of the following signs or symptoms: rash that covers a large area of your body, peeling skin, blisters.
  • Muscle problems (rhabdomyolysis). Muscle problems are common with Koselugo and can also be severe. Treatment with Koselugo may increase the level of a muscle enzyme in your blood called creatine phosphokinase (CPK) and may be a sign of muscle damage. Your healthcare provider should do a blood test to check your muscle enzyme levels of CPK before you start taking Koselugo and during treatment. Tell your healthcare provider right away if you get any of the following signs or symptoms: muscle aches or pain; muscle spasms and weakness; dark, reddish urine.

Before taking Koselugo, tell your healthcare provider about all your medical conditions, including if you:

  • have heart problems.
  • have eye problems.
  • have liver problems.
  • are pregnant or plan to become pregnant. Koselugo can harm your unborn baby. Your healthcare provider should verify if you/your partner are pregnant before beginning treatment. Ensure you/your partner use effective birth control (contraception) during treatment and for 1 week after your last dose if there is possibility pregnancy could occur. Tell your healthcare provider right away if you/your partner think you may be pregnant.
  • are breastfeeding or plan to breastfeed. It is not known if Koselugo passes into your breast milk. Do not breastfeed during treatment and for 1 week after your last dose. Talk to your healthcare provider about the best way to feed your baby during this time.

Tell your healthcare provider about all the medicines you take, including prescription, over-the-counter medicines, vitamins, or herbal supplements. Especially tell your healthcare provider if you are taking aspirin, blood thinners, or other medicines to treat blood clots. Koselugo contains vitamin E, which may increase risk of bleeding.

What should I avoid while taking Koselugo?

Do not drink grapefruit juice, eat grapefruit, or take supplements with grapefruit or St. John’s Wort during treatment.

Most common side effects include: 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.

These are not all the possible side effects of Koselugo. Call your healthcare provider for medical advice about side effects. Your healthcare provider may change your dose, temporarily stop, or permanently ask you to stop taking Koselugo if you have any of these side effects. You may report side effects to AstraZeneca at 1-800-236-9933 or at https://
us-aereporting.astrazeneca.com or FDA at
1-800-FDA-1088 or www.fda.gov/medwatch.

Indication

What is Koselugo?

Koselugo is a prescription medicine that is used to treat children 2 years of age and 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.

Please see Patient Information in the full Prescribing Information for Koselugo (selumetinib).

All families shown in this website have been compensated by Alexion, unless otherwise noted.