Journey To Danyelza

Treatment With DANYELZA

Every child's HRNB experience is different, but common treatment steps include:

With DANYELZA, feel more confident about what's next

If your child has an incomplete response to induction therapy (chemotherapy +/- surgery) or relapse therapy (if the disease comes back), HRNB may still be in their bone and/or bone marrow. It’s important to know what to look for, where to look, and when your child’s treatment plan needs to change.

Common ways to measure the disease

Neuroblastoma in the bone marrow can be measured by
taking samples using bone biopsies and aspiration

Neuroblastoma in the bone is measured by imaging tests,
such as MIBG^? scans and/or PET scans

MIBG^? scans are used to calculate a Curie score, which helps your
care team assess your child's HRNB.

What Is a Curie Score?

Knowing you child's Curie score could inform treatment planning

In the simplest terms, the Curie score^? is a way for doctors to measure how much neuroblastoma has spread in the body. To get a Curie score, your care team will look at MIBG^? scans and assess the amount of disease in specific areas of the body.

Curie score measures the extent of HRNB in the bone or soft tissue

10 total areas

are scored from 0-3 (9 bone & 1 soft-tissue)

Total scores can range from

0-30

A higher score means there is more neuroblastoma in the bone and/or soft tissue

How do Curie scores help?

Knowing your child's Curie score over time can provide a clearer picture of how much cancer is in their body and how it has changed with treatment. This information may help guide upcoming treatment decisions.

A complete response means the child also
has a Curie score of 0

The Curie score after induction therapy may help
determine if a change in treatment is needed

Planning next steps? Knowledge is your power—get the facts on Curie
scores at your child's next appointment.

DANYELZA Treatment Cycle

Know what to expect, and when, during DANYELZA treatment

Your child will receive DANYELZA^® intravenously (IV) by healthcare professionals on days 1, 3, and 5 of a 28-day cycle.

Five days before receiving their first infusion of DANYELZA, your child will also start another medicine, granulocyte-macrophage colony-stimulating factor (GM-CSF). GM-CSF can be given at home and helps the body make white blood cells so the immune system is stronger during treatment. Starting 5 days before treatment with DANYELZA, your care team may also prescribe a 12-day course of gabapentin to help reduce pain during treatment, which can also be given at home.

Since every child is different, the number of treatment cycles will depend on your child’s response. Once your care team notices an initial response to treatment, DANYELZA will be given for at least another 5 cycles.

Managing Side Effects

Understanding side effects and how they're handled

Based on the clinical studies of DANYELZA^®, some side effects can be expected. Your child’s care team will monitor your child closely throughout treatment and will have a plan to help manage any side effects, as needed.

Pain

Pain is common with DANYELZA and can be severe. Your child will likely experience pain during the infusion in places such as the belly, bone, neck, legs, or arms. In Study 201, severe pain resolved within an hour in the majority of patients who experienced it.

Nervous system problems

Severe pain from nerves (neuropathic pain)
Inflammation of the spinal cord
Reversible Posterior Leukoencephalopathy Syndrome (RPLS - also known as Posterior Reversible Encephalopathy Syndrome - PRES), which is a condition of the brain
Numbness, tingling, or burning sensation in the arms or legs
Nervous system problems of the eye
Problems urinating or emptying the bladder (prolonged urinary retention)

The following medicines may help with pain:

Preventive pain medicine (eg, gabapentin)
Taken by mouth for 12 days starting 5 days before the first DANYELZA infusion in each cycle
Other pain medicine (eg, opioids)
Given approximately an hour before the infusion and may be administered by IV as needed for additional pain during the infusion
Anesthetic (eg, ketamine)
Given if the pain is not adequately controlled by opioids. This may make your child sleepy during the infusion

Infusion-related reactions and how they may be managed

DANYELZA can cause serious infusion-related reactions, which are common with this treatment, that may require immediate medical attention.

Signs and symptoms of infusion-related reactions during or after DANYELZA treatment include:

Swelling of the face, eyes, lips, mouth, or tongue
Itching
Redness of the face
Skin rash or hives
Trouble breathing
Coughing or wheezing
Noisy, high-pitched breathing
Feeling faint or dizzy

DANYELZA can also cause fever, nausea, or vomiting during or after the infusion.

Speak with your care team right away if your child experiences any side effects while on DANYELZA.

It's possible to prepare for infusion-related reactions. Based on how your child is feeling, their care team may offer the following medicines:

Steroids (eg, methylprednisolone): Given by IV between 30 minutes and 2 hours before the first DANYELZA infusion. If your child had a severe reaction before, these medicines may be given at the next infusion or cycle.

Antihistamines (eg, diphenhydramine): Given 30 minutes before the infusion.

Fever reducers (eg, acetaminophen): Given 30 minutes before the infusion.

Medicine for nausea/vomiting (eg, antiemetics and H2 antagonists): Given 30 minutes before the infusion to help reduce nausea and vomiting.

Your child's care team is more than just doctors and nurses; it also includes a variety of specialists and therapists dedicated to supporting your whole journey.

WHAT IS DANYELZA?

DANYELZA is a prescription medicine used in combination with a medicine called granulocyte-macrophage colony-stimulating factor (GM-CSF) to treat children 1-year of age and older and adults with high-risk neuroblastoma in the bone or bone marrow that:

has come back (relapsed) or that did not respond to previous treatment (refractory), and
has shown a partial response, minor response, or stable disease to prior therapy.

It is not known if DANYELZA is safe and effective in children younger than 1 year of age.

IMPORTANT SAFETY INFORMATION

What is the most important information I should know about DANYELZA?
DANYELZA may cause serious side effects, including:

Serious infusion-related reactions. DANYELZA can cause serious infusion-related reactions that require immediate medical attention. Infusion-related reactions are common with DANYELZA. Tell your healthcare provider right away if you get any signs or symptoms during or after your DANYELZA infusion, including:

swelling of your face, eyes, lips, mouth, or tongue
itching
redness on your face (flushing)
skin rash or hives
trouble breathing
cough or wheezing
noisy high-pitched breathing
feeling faint or dizziness (low blood pressure)

Nervous system problems. Talk to your healthcare provider right away if you have new symptoms or worsening of nervous system problems, including:

Severe pain from nerves (neuropathic pain), including pain in the belly (abdomen), bone, neck, legs, or arms. Pain is common with DANYELZA and can be severe

Inflammation of the spinal cord. Signs or symptoms may include:

weakness in your legs or arms
bladder and bowel problems
pain in back, legs, or stomach (abdomen)
numbness
tingling
burning sensation

Reversible Posterior Leukoencephalopathy Syndrome (RPLS – also known as Posterior Reversible Encephalopathy Syndrome - PRES). PRES is a condition that affects the brain. Your healthcare provider will monitor your blood pressure and check for any neurologic symptoms after your DANYELZA infusion. Signs or symptoms of PRES may include:

severe headache
vision changes
changes in mental status, such as confusion, disorientation, or decreased alertness
difficulty speaking
weakness in your arms or legs
seizures

Numbness, tingling, or burning sensation in the arms or legs.

Nervous system problems of the eye.Signs or symptoms may include:

unequal pupil size
blurred vision
trouble focusing your eyes
larger pupil size (dilated)
decreased ability to see
sensitivity to light

Problems urinating or emptying your bladder (prolonged urinary retention).

Do not receive DANYELZA if you have had a severe allergic reaction to naxitamab-gqgk (the active ingredient in DANYELZA). Ask your healthcare provider if you are not sure.

Before receiving DANYELZA, tell your healthcare provider about all your medical conditions, including if you:

have high blood pressure
have heart disease
are pregnant or plan to become pregnant. DANYELZA may harm your unborn baby.
- your healthcare provider will do a pregnancy test before you start treatment with DANYELZA.
- females who are able to become pregnant should use effective birth control (contraception) during treatment and for 2 months after your last dose of DANYELZA. Talk to your healthcare provider about birth control choices that may be right for you during this time.
- tell your healthcare provider right away if you become pregnant or think you might be pregnant during treatment with DANYELZA.
are breastfeeding or plan to breastfeed. It is not known if DANYELZA passes into your breast milk. Do not breastfeed during treatment and for 2 months after your last dose of DANYELZA.

Tell your healthcare provider about all the medicines you take, including prescription and over-the-counter medicines, vitamins, and herbal supplements.

What are the possible side effects of DANYELZA?
DANYELZA may cause serious side effects, including:

See “What is the most important information I should know about DANYELZA?”

Swelling of the heart (myocarditis). Myocarditis has happened in adolescents ages 12-18 within days of receiving DANYELZA. Tell your healthcare provider if you get any signs or symptoms of myocarditis, including:

chest pain
shortness of breath
irregular heartbeat or feel like your heart is racing

High blood pressure (hypertension). High blood pressure is common in people who receive DANYELZA. Your blood pressure will be monitored during your DANYELZA infusion, and at least each day on Days 1 to 8 of each DANYELZA treatment cycle. Tell your healthcare provider right away if you get any signs or symptoms of high blood pressure, including:

headaches
seizures
nausea or vomiting
chest pain
dizziness
visual changes
shortness of breath
feeling that your heart is pounding or racing (palpitations)
nose bleeds

Decreased blood pressure (orthostatic hypotension) that can be severe and require hospitalization. You may feel dizzy, lightheaded, or pass out (faint) when you rise too quickly from a sitting or lying position. Your healthcare provider will monitor your blood pressure before you start and during treatment with DANYELZA.

The most common side effects of DANYELZA include:

fast heart rate
vomiting
cough
nausea
decreased white blood cell, red blood cell, and platelet counts
diarrhea
decreased appetite
tiredness
skin rashes
decreased level of potassium, sodium, and phosphate in the blood
hives
fever
headache
injection site reaction
swelling of the body or only in one part of the body
anxiety
irritability
increased liver function blood tests
decreased blood sugar level
decreased calcium levels in the blood
decreased protein levels (albumin) in the blood

These are not all of the possible side effects of DANYELZA. Call your doctor for medical advice about side effects. You may report side effects to FDA at 1-800-FDA-1088.

Please click for full Prescribing Information and Patient Information for DANYELZA including Boxed Warning on serious infusion-related reactions and nervous system problems, and talk to your doctor.