DANYELZA™ (naxilamab-gqgk) | Relapsed / Refractory Neuroblastoma 

Intended for US audiences only

Important Safety Information Prescribing Information For US Healthcare Professionals
Support – High Risk Neuroblastoma Treatment Site
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SUPPORT & RESOURCES

Learning that your child's high-risk neuroblastoma hasn't responded to treatment or has returned can be difficult. You are not alone. There are many helpful resources, programs, organizations, and individuals available to help you on your journey.

Y-mAbs ConnectTM may provide access and reimbursement support* for qualifying patients prescribed a Y-mAbs medication

Our dedicated team of case managers can provide information on:

  • Insurance coverage for DANYELZA®

  • Prior authorization and appeals requirements of insurer

  • Y-mAbs Connect financial support programs

  • Other financial or travel support programs provided by third-party organizations

If prescribed DANYELZA, here is how to enroll

If your child was prescribed DANYELZA, your healthcare provider can complete the Y-mAbs Connect enrollment form with you and return it by fax, mail, or email.* A Y-mAbs Connect case manager will contact you once you have been enrolled.

Note: 3rd party organizations are not associated with Y-mAbs Therapeutics, Inc.; specific details and eligibility requirements may vary by organization.

*To download the enrollment form, go to www.ymabsconnect.com. Email, mail, and fax contact information can be found on the enrollment form.

Learn more at www.ymabsconnect.com

Financial support may be available

Y-mAbs Connect Co-pay
Program†

For eligible individuals with commercial or private insurance

May help reduce out-of-pocket costs of DANYELZA to $0 for eligible patients

Y-mAbs Connect Patient Assistance Program (PAP)‡

For eligible individuals who are uninsured or are rendered underinsured for DANYELZA through their health plan

May be able to
provide DANYELZA
at no cost

You must be enrolled in Y-mAbs Connect for these programs.
Please visit www.ymabsconnect.com or speak to a case manager for additional eligibility criteria and terms and conditions for these programs.

†Eligibility criteria for the Co-pay Program include but are not limited to patients who have commercial or private insurance, are US or US territory residents, and are actively insured at time of treatment. Government- or publicly insured patients are not eligible.

‡For the Patient Assistance Program you must meet certain eligibility criteria.

Note: Y-mAbs Therapeutics, Inc. reserves the right at any time, and without notice, to modify or discontinue these programs and any support provided to the patient.

Speak with a Y-mAbs Connect case manager at 1-833-33YMABS (1-833-339-6227), option 2, Monday through Friday, 8 AM to 8 PM ET.

RESOURCES

While the diagnosis of high-risk neuroblastoma for your child produces serious questions for you, the right support can make all the difference. Below you will find important information to help navigate your treatment journey, including helpful questions to ask your child's doctor.

DANYELZA Fact Sheet

Provides important information you need to know on the efficacy and safety of DANYELZA®

Full Prescribing Information and Patient Information

A PDF of the full Product Information

Questions to ask your child’s doctor

When talking to your child’s doctor about DANYELZA, here are some questions that might be helpful.

Community groups for neuroblastoma patients

Below you can find various organizations that can give you and your family more information about neuroblastoma and connect you with the logistical and emotional support that can help navigate the challenges of the diagnosis.

Alex’s Lemonade Stand Foundation for Childhood Cancer
www.alexslemonade.org

Alex’s Lemonade Stand Foundation funds impactful research, raises awareness, and supports families in their quest to help cure childhood cancer.

Arms Wide Open Childhood Cancer Foundation
www.awoccf.org

The mission of Arms Wide Open is to help families, both financially and emotionally, who are suffering hardship due to their child’s diagnosis of cancer, providing them support through the most difficult time of their lives.

Band of Parents
www.bandofparents.org

Band of Parents acts as a support network for the newly diagnosed and their families as they go through treatment.

Braden’s Hope for Childhood Cancer
www.bradenshope.org/wp/

Named after Braden Hofen, who survived a diagnosis of Stage IV neuroblastoma, this organization is dedicated to funding research for new treatments of childhood cancers.

CAC2 Kids’ Cancer Research Foundation
www.cac2.org

Coalition Against Childhood Cancer (CAC2) is a collaborative network of international nonprofits, corporations, and individuals that effectively advance a variety of childhood cancer causes.

The Carrot Seed Foundation
www.carrotseedfoundation.org

The Carrot Seed Foundation helps fund pediatric cancer research, clinical trials, pediatric oncology nursing scholarships, and other causes that help families overcome the hardship of battling neuroblastoma.

The Children’s Oncology Group
www.childrensoncologygroup.org

The Children’s Oncology Group (COG) is the world’s largest organization devoted exclusively to childhood and adolescent cancer research. COG has nearly 100 active clinical trials open at any given time.

Children’s Neuroblastoma Cancer Foundation (CNCF)
www.cncfhope.org

The premier source for neuroblastoma information and resources, the CNCF is composed of families like yours committed to finding a cure for neuroblastoma through research, education, awareness, and advocacy.

The Children’s Oncology Group
www.clinicaltrials.gov

A database of privately and publicly funded clinical studies conducted around the world. Individuals can search key terms, such as “neuroblastoma”.

End Kids Cancer
www.endkidscancer.org

The mission of the End Kids Cancer Foundation, is to provide financial support for clinical research and clinical trials that will cure neuroblastoma.

The EVAN Foundation
www.theevanfoundation.org

Established in memory of Evan Lindberg, The EVAN Foundation spreads hope and drives change in the fight against neuroblastoma.

The James Fund/SickKids Foundation
www.sickkidsfoundation.com

SickKids fights to find new cures and provide world-class care to children with cancer.

Memorial Sloan Kettering Kids Neuroblastoma
www.mskcc.org/pediatrics/cancer-care/types/neuroblastoma

The neuroblastoma team at MSK Kids offers you leadership, expertise, reassurance, support, and compassion to give your child the best chance of a cure, a good quality of life, and the best chance for a promising future.

NANT
www.nant.org

NANT is a clinical trials consortium focused on developing new treatments and biomarkers for relapsed or refractory high-risk neuroblastoma through early phase clinical trials.

The Neuroblastoma Children’s Cancer Society
neuroblastomacancer.org

A dedicated group of volunteers, many of them caregivers to children with the disease, that offer support for families facing this disease. Their mission includes fundraising for research into neuroblastoma and raising awareness of the disease.

Solving Kids’ Cancer (SKC)
www.solvingkidscancer.org

Solving Kids’ Cancer focuses on aggressive childhood cancers with low survival rates, by helping accelerate new, next-generation treatments.

Commonly asked questions ABOUT DANYELZA

Here are some answers to the most commonly asked questions about DANYELZA®.

Who is DANYELZA for?

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.

DANYELZA is approved based on two clinical studies that looked at reducing tumor size. DANYELZA is still being studied to confirm the study results and the clinical benefit of treatment.

What is DANYELZA?

DANYELZA is a prescription medicine given with GM-CSF. DANYELZA is an anti-GD2 monoclonal antibody , which is a type of protein made in a lab that can bind to neuroblastoma tumor cells that have GD2 on their cell surfaces.

Is DANYELZA a type of chemotherapy?

No, DANYELZA is not a type of chemotherapy. It works with the body to help the immune system fight neuroblastoma.

How does DANYELZA work?

DANYELA works by attaching itself to a sensor on the cancer cell. It then signals the immune system to attack the cancer cell. The immune cells then kill the cancer cell.

How is DANYELZA given?

DANYELZA is given by intravenous injection by a healthcare professional. There are 3 infusions per treatment cycle. Treatment cycles are repeated every 4 weeks until complete response or partial response, followed by 5 additional cycles every 4 weeks. Subsequent cycles may be repeated every 8 weeks. The doctor will discontinue DANYELZA and GM-CSF in the event of disease progression or unacceptable toxicity. Your child's doctor will assess the patient's response to each treatment to determine if treatment should continue.

What should I tell my healthcare provider before receiving DANYELZA?

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

  • have high blood pressure

  • 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.

Does DANYELZA require an overnight stay in the hospital?

Your child may be able to receive DANYELZA in an outpatient setting and return home the same day. It is up to your child's doctor to determine if this option is right for you or your child.

Will my insurance cover DANYELZA?

Many do, but every insurance plan is different. Speak with a Y-mAbs Connect case manager to help confirm your eligibility by calling 1-833-33YMABS (1-833-339-6227), option 2, Monday through Friday (8AM to 8PM ET).

Glossary

Here is a glossary for various terms you may come across in neuroblastoma treatment

ANTIBODY

An antibody is a protein made by plasma cells (a type of white blood cell) in response to an antigen (a substance that causes the body to make a specific immune response). Each antibody can bind to only one specific antigen. The purpose of this binding is to help destroy the antigen. Antibody therapies use antibodies to help the body fight cancer, infection, or other diseases.

BONE MARROW

Bone marrow is the soft, sponge-like tissue in the center of most bones. It produces white blood cells, red blood cells, and platelets.

COMPLETE RESPONSE

A complete response, or CR, is when the patient showed no physical evidence of disease on examination or imaging tests after treatment (sometimes phrased as “no evidence of disease” and abbreviated NED).

GD2

GD2 is a molecule found on neuroblastoma cells and certain nerve cells.

GM-CSF

Granulocyte-macrophage colony-stimulating factor is a medication given with DANYELZA® to help activate the body’s immune system.

IMMUNE SYSTEM

The immune system is the network of cells, tissues, organs, and the substances they make that helps the body fight infections and other diseases. It includes white blood cells and organs and tissues of the lymph system, such as the thymus, spleen, tonsils, lymph nodes, lymph vessels, and bone marrow.

IMMUNOTHERAPY

A medicine that helps signal the body’s immune system to fight the disease.

INFUSION

An infusion is a way to put fluids, including drugs, into the bloodstream and is often called an intravenous infusion, which means an infusion into a vein.

IV (INTRAVENOUS)

IV is short for intravenous and usually refers to a way of giving a drug or other substance through a needle or tube inserted into a vein.

MRI

Magnetic resonance imaging allows a noninvasive scan to determine if a tumor has shrunk from treatment or has grown larger.

OUTPATIENT SETTING

A medical procedure that can be completed without a patient staying in a hospital overnight.

OVERALL RESPONSE RATE

Either a complete or partial response to treatment.

PARTIAL RESPONSE

A partial response, or PR, is when a patient had their cancer reduced by at least half.

PROPHYLACTIC

A medicine that is given before the onset of a disease or symptoms so that it can help prevent them.

REFRACTORY

This is a term used to describe high-risk neuroblastoma that does not respond completely to initial treatment.

RELAPSED

This is a term used to describe high-risk neuroblastoma that has returned following previous treatment.

SUBCUTANEOUS INJECTION

An injection that is delivered under the skin, usually into muscle, and not into a vein as in with an IV infusion.

TREATMENT RESPONSE

A term used to describe when treatment leads to a significant reduction in cancer burden in the body. This includes when treatment led to either a complete or partial response (overall response rate).

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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:
    1. swelling of your face, eyes, lips, mouth, or tongue
    2. itching
    3. redness on your face (flushing)
    4. skin rash or hives
    5. trouble breathing
    6. cough or wheezing
    7. noisy high-pitched breathing
    8. feeling faint or dizziness (low blood pressure)
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.

DANYELZA is approved based on two clinical studies that looked at reducing tumor size. DANYELZA is still being studied to confirm the study results and the clinical benefit of treatment.

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 infusion, including:
    1. swelling of your face, eyes, lips, mouth, or tongue
    2. itching
    3. redness on your face (flushing)
    4. skin rash or hives
    5. trouble breathing
    6. cough or wheezing
    7. noisy high-pitched breathing
    8. feeling faint or dizziness (low blood pressure)
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.

DANYELZA is approved based on two clinical studies that looked at reducing tumor size. DANYELZA is still being studied to confirm the study results and the clinical benefit of treatment.

  • 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
  • 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?”
  • 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

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.