Your Treatment Guide for Koselugo™ (selumetinib) · Your Treatment Guide for Koselugo™ (selumetinib) NF1 plexiform neurofibromas (PN) are benign tumors that can have an impact

Your Treatment Guide for Koselugo™ (selumetinib)NF1 plexiform neurofibromas (PN) are benign tumors that can have an impact on children. This treatment guide is designed to help you throughout your child’s treatment journey and is full of important information about NF1 PN and Koselugo, a breakthrough oral medicine proven to shrink PN tumors.

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.

IMPORTANT SAFETY INFORMATION

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

•have heart problems

•have eye problems

•are pregnant or plan to become pregnant. Koselugo can harm your unborn baby

— Females who could become pregnant and males with female partners who could become pregnant should use effective birth control (contraception) during treatment with Koselugo and for 1 week after your last dose

— Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Koselugo

•are breastfeeding or plan to breastfeed. It is not known if Koselugo passes into your breast milk

— Do not breastfeed during treatment with Koselugo and for 1 week after your last dose

— Talk to your healthcare provider about the best way to feed your baby during this time

Please see Important Safety Information on pages 14 and 15. Please click here for full Prescribing Information, including Patient Information.

LESSTUMOR SIZE

MORE— Cooper, age 8, actual patient

INTRODUCING the FIRST and ONLY FDA-approved treatment for pediatric patients aged 2 years and older who have neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PN) that cannot be completely removed by surgery

http://www.azpicentral.com/pi.html?product=koselugo

http://www.azpicentral.com/pi.html?product=koselugo&medguide=y

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UNDERSTANDING NF1 PLEXIFORM NEUROFIBROMAS (PN)

What are your concerns about your child’s NF1 plexiform neurofibromas? Write down any questions you may want to ask your child’s doctor and share them during your next visit.

What are plexiform neurofibromas (PN), or plexiform tumors?

Where do NF1 plexiform neurofibromas occur? These tumors can grow on any nerve in the body and can be large or small in size.

Head and neck

Back Legs, arms, and hands

Bowel and bladder

Chest and abdomen

Eyes

Airways

NF1=neurofibromatosis type 1.

of children with NF1 may develop benign (noncancerous) tumors called plexiform neurofibromas or plexiform tumors, which grow along nerves. 30% to 50%




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TREATMENT FOR NF1 PLEXIFORM NEUROFIBROMAS

Which doctors and healthcare specialists treat pediatric NF1 plexiform neurofibromas? NF1 plexiform neurofibromas are usually treated by a team of healthcare specialists, including neuro-oncologists, oncologists, neurologists, geneticists, surgeons, and others who specialize in treating symptoms related to this disease. The location of your child’s plexiform neurofibromas will help determine which doctors are best to see for treatment.

Why can’t some NF1 plexiform neurofibromas be completely removed with surgery?Surgery for NF1 plexiform neurofibromas may not be recommended by your child’s healthcare provider. One reason is plexiform neurofibromas may be closely intertwined with critical nerves and located near major organs. Also, PN may not be completely removed by surgery.

Are there any other ways to treat NF1 plexiform neurofibromas?

Your child’s doctor may prescribe therapies or medicines to help relieve symptoms of NF1 plexiform neurofibromas.

Previously, there have been no FDA-approved medications proven to reduce the size of NF1 plexiform neurofibromas.

Surgery has been a standard treatment for NF1 plexiform neurofibromas.

Now, there’s an FDA-approved oral treatment proven to shrink PN tumors




4

The FIRST and ONLY FDA-approved treatment shown to reduce the size of plexiform neurofibromas (PN)*

In a clinical study, Koselugo was proven to work for pediatric patients with NF1 and plexiform neurofibromas (PN):

* Patients saw a 20% or more decrease in the size of their plexiform neurofibromas, confirmed by 3D magnetic resonance imaging scan. NF1=neurofibromatosis type 1.

66% of patients saw their plexiform neurofibromas shrink* 82% of patients who responded continued to benefit for at least one year

66%

Possible side effectsAs with any medicine, it’s possible to experience side effects. Be sure to tell your child’s doctor if your child experiences any side effect that bothers them or does not go away.

Remember, the information on this page does not replace medical guidance or directions from your doctor.

Always talk to them about side effects and ways to manage them

IMPORTANT SAFETY INFORMATION (Cont’d)

Tell your healthcare provider about all the medicines you take, including prescription and 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 your risk of bleeding.

Koselugo has been shown to shrink tumors in patients aged 2 years and older




5

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 with Koselugo 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 •tiredness •shortness of breath •increased heart rate •swelling of your ankles and feet

Eye problems. Koselugo can cause eye problems that can lead to blindness. Your healthcare provider will check your vision before and during treatment with Koselugo. Tell your healthcare provider right away if your 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 with Koselugo. 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 •blisters •peeling skin

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, which may be a sign of muscle damage. Your healthcare provider should do a blood test to check your muscle enzyme levels 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 •dark, reddish urine •muscle spasms and weakness

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.

These are not all the possible side effects of Koselugo For more information, ask your doctor or pharmacist. Call your doctor for medical advice about side effects. You may report side effects to the FDA at 1-800-FDA-1088.

Vomiting

Stomach pain

Nausea

Dry skin

Feeling of tiredness, weakness, or lacking energy

Muscle and bone pain

Fever

Inflammation of the mouth

Headache

Redness around the fingernails

Itching

The most common side effects of Koselugo are:




About12 hours

apart

FIRST DOSE

SECOND DOSE

6

AT LEAST 2 HOURS BEFORE A DOSE

AT LEAST 1 HOUR AFTER A DOSE

Remember: Koselugo capsules must only be swallowed whole with water. They must not be chewed, dissolved, or opened. Taking Koselugo in a different way than instructed may affect how it works and could change the results you see from the treatment. The efficacy of the drug may be altered in such a way that overdosing or underdosing may occur.


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 with Koselugo 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 •tiredness

•shortness of breath •increased heart rate

•swelling of your ankles and feet

Eye problems. Koselugo can cause eye problems that can lead to blindness. Your healthcare provider will check your vision before and during treatment with Koselugo. 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

Doses should be taken—on an empty stomach—approximately the same time each day, about 12 hours apart Your child must take Koselugo on an empty stomach, which means your child should not eat 2 hours before each dose or 1 hour after each dose

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




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It’s important to GIVE KOSELUGO EXACTLY AS YOUR CHILD’S DOCTOR PRESCRIBES AND KEEP TO A DOSING SCHEDULE THAT FITS BEST WITH YOUR CHILD’S DAILY ROUTINE. The goal is to make it easy to stay on track to get the most out of treatment.

Do not change the dose or stop giving Koselugo unless directed by your child’s doctor

Your child’s doctor may change the dose, temporarily stop, or permanently stop treatment if your child has side effects

Your child’s doctor will decide on the right dose of Koselugo based on your child’s weight or size (body surface area), and how many capsules of Koselugo to take



Actual patient.

For resources about pill swallowing and managing the dosing schedule of Koselugo, the AstraZeneca Nurse Call Center is available Monday through Friday, 8 am–8 pm, ET. To learn more, visit www.NurseCallCenter.com or call 1-844-275-2360.




Sample dosing schedules to share with your child’s doctor

6:00 AM

6:00 PM 1

First Dose

Second Dose

Breakfast between 7AM–9 AM

Dinner between 7 PM–9 PM

7:00 AM

7:00 PM 2

First Dose

Second Dose

Breakfast between 8 AM–10 AM


8:00 AM

8:00 PM 3

First Dose

Second Dose

Breakfast between 9 AM–11 AM


8

You may want to use a dose-alert timer, or the alarm

on your smartphone, as a reminder to take the medicine

on time as part of your child’s daily dosing routine.


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 •blisters your body

•peeling skin

Timing may be everything Work with your child’s doctor to establish a dosing routine that can be easily followed. Taking each dose of Koselugo as directed, every day, is the best way to benefit from treatment.

These sample schedules are designed as suggestions only.




9


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, which may be a sign of muscle damage. Your healthcare provider should do a blood test to check your muscle enzyme levels 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 •dark, reddish urine

•muscle spasms and weakness

For some helpful tips to teach your child how to swallow pills, you may want to download the brochure Medicine & Your Child: A guide for parents on adherence and administration at https://ccr.cancer.gov/sites/default/files/medbooklet.pdf or visit www.kidshealth.org to see Teaching Your Child How to Swallow Pills for additional suggestions.Please note: These websites are maintained by a third party who is solely responsible for its contents. AstraZeneca is not responsible for the privacy policy of any third-party websites. We encourage you to read the privacy policy of every website you visit.

TIPS TO HELP YOUR CHILD TAKE MEDICATION

Convincing a child to take medicine can be a difficult task. Common reasons why children don’t want to take medicine may include:

Not understanding why they need to take it

Not liking the taste or texture

Having difficulty swallowing medication

It’s important that your child understands why it’s necessary to take 2 doses of Koselugo every day. Be sure to talk about it in a way that’s positive. And, as your child grows, you may need to change your approach to ensure that the dosing schedule is followed.

Suggestions to help give medicine to young children (3 to 12 years old) Role-playing can be helpful: Pretending to give a favorite doll or teddy bear some medicine

can make taking medicine more fun

Try to do a positive, fun activity after your child takes the medicine. This may help motivate your child to take the medicine again




10

Make a list of questions you’d like to ask your child’s doctor and information you’d like to share about your child:

Before taking Koselugo, tell your child’s doctor about all of your child’s medical conditions, including if they:

Have heart problems

Have eye problems

Are pregnant or plan to become pregnant. Koselugo can harm an unborn baby Your child’s healthcare provider should check to see if your child is pregnant before they begin treatment with Koselugo

Females who are able to become pregnant should use effective birth control (contraception) during treatment with Koselugo and for 1 week after the last dose

Males with female partners who are able to become pregnant should use effective birth control (contraception) during treatment with Koselugo and for 1 week after the last dose

Tell your child’s healthcare provider right away if your child becomes pregnant or thinks she may be pregnant during treatment with Koselugo

Are breastfeeding or plan to breastfeed It is not known if Koselugo passes into breast milk

Your child should not breastfeed during treatment with Koselugo and for 1 week after the last dose

Talk to your child’s healthcare provider about the best way for your child to feed her baby during this time

Your child will take 3 tests before taking Koselugo: A test using sound waves to painlessly create pictures of the heart (called an echocardiogram)

A blood test for certain enzymes and naturally occurring chemicals

An assessment of your child’s eyes

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

1Ask what tests are needed before and while on treatment

2Share details about pre-existing conditions

Provide names of other medicines your child may be taking

3




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HELPING YOUR CHILD WITH NF1 PN REQUIRES A FAMILY COMMITMENT

Plexiform neurofibromas (PN) can cause a lot of uncertainty. Creating daily routines for children can help them feel secure and give them a positive sense of control

Building a supportive home environment in which all family members are

treated equally with respect and acceptance can benefit not only the child with NF1 PN, but also siblings, who may feel they’re getting less attention

Making the transition from childhood to teenage years can be especially

difficult when living with NF1 PN. Helping your child gradually take more control of their health care, medications, and treatment decisions can encourage responsibility and help them feel empowered

NF1=neurofibromatosis type 1.

Actual patient.




•vomiting •fever

•stomach pain •inflammation of the mouth

•nausea •headache

•dry skin •redness around the fingernails

• feeling of tiredness, weakness, or •itching lacking energy

•muscle and bone pain

These are not all the possible side effects of Koselugo.




12

Your child is beginning treatment. It may be helpful to see what other patients experienced. The chart below is an overview of the first year of patients who took Koselugo in a clinical study. It notes when patients responded to treatment and when they experienced side effects.

It’s important to remember that everyone may respond to treatment differently, so your child may not experience the same efficacy results (tumor shrinkage) or the same side effects described below.

Please know that these are not all the possible efficacy results or side effects your child may experience during treatment with Koselugo.

Use the chart below to track your child’s progress and any side effects that may occur. Be sure to share your child’s experiences with their doctor.

* The median time to response was 7.2 months.†Koselugo can lower the amount of blood pumped by the heart. Your child’s doctor should do tests before and during treatment to check how well your child’s heart is working.‡Koselugo can cause eye problems. Tell your child’s doctor as soon as possible if your child’s vision blurs or if any other changes to their sight occur during treatment.


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.


An overview of the patient journey during the clinical study of Koselugo




13

Having the right support can make all the difference in your child’s treatment journey. Please see the back cover to learn about the AstraZeneca Case Managers who are ready to connect caregivers and patients to the personal assistance they need.

Things to remember

Track your child’s progress, and at their next doctor’s visit, talk about any changes your child has been experiencing. You might also want to ask about your child’s care as time goes by. Write down any guidance or recommendations you need to remember.

Actual patient.




14

IMPORTANT SAFETY INFORMATION

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

•have heart problems

•have eye problems

•are pregnant or plan to become pregnant. Koselugo can harm your unborn baby

— Females who could become pregnant and males with female partners who could become pregnant should use effective birth control (contraception) during treatment with Koselugo and for 1 week after your last dose

— Tell your healthcare provider right away if you become pregnant or think you may be pregnant during treatment with Koselugo

•are breastfeeding or plan to breastfeed. It is not known if Koselugo passes into your breast milk

— Do not breastfeed during treatment with Koselugo 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 and 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 your risk of bleeding.

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 with Koselugo 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 •tiredness

•shortness of breath •increased heart rate

•swelling of your ankles and feet

Eye problems. Koselugo can cause eye problems that can lead to blindness. Your healthcare provider will check your vision before and during treatment with Koselugo. 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





15

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 •blisters your body

•peeling skin

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, which may be a sign of muscle damage. Your healthcare provider should do a blood test to check your muscle enzyme levels 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 •dark, reddish urine

•muscle spasms and weakness



•vomiting •fever

•stomach pain •inflammation of the mouth

•nausea •headache

•dry skin •redness around the fingernails

• feeling of tiredness, weakness, or •itching lacking energy

•muscle and bone pain

These are not all the possible side effects of Koselugo.

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.





Koselugo and AstraZeneca Access 360 are trademarks of the AstraZeneca group of companies. All other trademarks are property of their respective owners.

©2020 AstraZeneca. All rights reserved. US-41339 Last Updated 6/20

HELPING YOUR PATIENTS AND CAREGIVERS ACCESS THE CARE THEY NEED

For more information, visit www.Koselugo.com.

PERSONALIZED KOSELUGO™ (selumetinib) SUPPORT IS HERE

If your doctor has prescribed Koselugo, you may have questions. This program is designed to help you navigate through the process and get the

most out of your child’s treatment.

To learn more or contact your personal Case Manager, please call 1-844-ASK-A360 (1-844-275-2360), Monday through

Friday, 8 am–8 pm ET, or visit www.MyAccess360.com


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.


PPlease see Important Safety Information on pages 14 and 15. Please click here for full Prescribing Information, including Patient Information.

Case Managers can provide personal support to answer questions about: Insurance coverage Out-of-pocket costs Access to patient assistance

Assistance from Nurse Advocates for patients and caregivers regarding: Disease education Product education Tips for your child’s treatment plan

AstraZeneca strives to achieve $0 co-pay target for all eligible patients*†

*The average monthly cost for Koselugo may be greater than $0 for some patients. Based on commercially insured patients for on-label use.†Based on current and previous dosage formulations and for all FDA-approved indications.



Documents

Your Treatment Guide for Koselugo™ (selumetinib) · Your Treatment Guide for Koselugo™ (selumetinib) NF1 plexiform neurofibromas (PN) are benign tumors that can have an impact