How
Koselugo
can help

Sam, a 10-year-old who takes Koselugo for NF1 PN, playing the keyboard

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

What is Koselugo?

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

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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.

Koselugo 10 mg and 25 mg pill bottles

Koselugo is proven to shrink plexiform neurofibromas

The SPRINT study was conducted by the National Cancer Institute (NCI) to test Koselugo in children and teens who could not have their plexiform neurofibromas removed by surgery.*

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In 66% (33/50) of children and teens, Koselugo shrank plexiform neurofibromas by at least 20%**

In the SPRINT study

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96% (48/50) of children and teens saw their PN shrink or stay about the same.**†

~2.5 years

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In the SPRINT long-term follow-up

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74% (37/50) of children and teens continued to see shrinkage or stability over 2.5 years later.‡

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Sam, a 10-year-old who takes Koselugo for NF1 PN
Sam, a 10-year-old who takes Koselugo for NF1 PN

97% (32/33) of children and teens who responded to Koselugo achieved 20% or greater PN shrinkage between 3.3 months and 1 year into treatment‡

In the majority of children and teens who responded, plexiform neurofibromas remained stable for at least 3 years.

79%

(26/33) maintained a PN reduction response for at least 2 years§

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64%

(21/33) maintained a PN reduction response for at least 3 years§ 

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

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Your child's doctor may refer to this as “duration of response,” which is the amount of time that children and teens in the study achieved 20% or greater tumor shrinkage and maintained that shrinkage.

 

Hear from a real Koselugo caregiver and her child

Watch a video about Kim and her son, Quentin, as they discuss his experience with Koselugo.

4+ YEARS

Koselugo has been prescribed at top NF1 centers across the country for over 4 years

*Confirmed by 3D magnetic resonance imaging (MRI) scan.
**The best response recorded as of June 2018 data cutoff. The median time to onset of response was 7.2 months (range: 3.3 months to 1.6 years). Median is defined as the middlemost point in a dataset.
†"Stay about the same" is defined as no more than a 20% increase or decrease in tumor volume.
‡Data cutoff February 2021. This information is from the SPRINT long-term follow-up study. The most recent response at each patient’s last visit prior to the data cutoff is as follows: 50% (n=25) saw their PN shrink by at least 20%, 24% (n=12) saw their PN stay about the same, 22% (n=11) saw their PN grow, and 4% (n=2) were not evaluated.
§One more person achieved 20% or greater PN shrinkage after about a year and a half.
‖Data cutoff March 2021.

Koselugo has 7.7 years of established safety studied in people with NF1 plexiform neurofibromas

In clinical trials, most children and teens had manageable side effects that did not require permanently stopping treatment.

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

In the Koselugo long-term follow-up study, no new side effects were identified.***

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

76%

(38/50) were able to stay on a full dose of Koselugo

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80%

(40/50) were able to avoid stopping treatment by temporarily pausing dosing

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88%

(44/50) were able to stay on treatment without permanently stopping due to a 
side effect

    Data cutoff February 2021. Based on the long-term follow-up of the SPRINT Phase 1 and Phase 2 Stratum 1 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 children and teens with NF1 PN that could not be removed by surgery without risk of issues because of their location or size. These children and teens 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 children and teens received Koselugo treatment for less than 4.4 years, while the other 50% received Koselugo treatment for more than 4.4 years.

    *** Known side effects were reported. These data include 74 children and teens who received a Koselugo dose of 20 mg/m2 to 30 mg/m2 twice daily for up to 7.7 years with no new safety signals reported.

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

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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STAY INFORMED ABOUT NF1 PLEXIFORM NEUROFIBROMAS

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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.