For Patients and Caregivers For Healthcare Professionals Important Safety Information Prescribing Information

This information is intended for U.S. healthcare professionals

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PEER CONNECTIONS

Amgen By Your Side Goes Above and Beyond to Support You

There are many ways our team can connect you to the people and organizations who can help support you during your treatment experience.

Community Connections

Connecting You to the Support You Need

Your PAL can help you discover ways to connect with others, if interested (examples include in-person gatherings, online/virtual meetings, and even getting introduced to patient groups and mentors).

Don’t miss out on upcoming virtual events

There are educational programs available about living with cystinosis, where members of the cystinosis community can get together and:

  • Learn about cystinosis and treatment with PROCYSBI
  • Meet members of the cystinosis community
  • Share experiences
  • Ask questions

Independent Organizations for Those Living With Nephropathic Cystinosis

  • Cystinosis Research Network (CRN)

    CRN is an all-volunteer, nonprofit organization dedicated to supporting and advocating for research, providing family assistance, and educating the public and medical communities about cystinosis.

    cystinosis.org
  • Cystinosis Research Foundation (CRF)

    CRF supports research that aims to improve the quality of life of patients with cystinosis, find better treatments, and ultimately find a cure.

    cystinosisresearch.org
  • Cystinosis Foundation of New Jersey

    The Cystinosis Foundation of New Jersey is a nonprofit advocacy organization devoted to increasing awareness of cystinosis and funding medical research related to treating and curing those with the condition.

    cystinosisfoundationofnewjersey.org
  • American Association of Kidney Patients (AAKP)

    An independent kidney patient organization, AAKP is dedicated to improving the lives and outcomes of kidney patients through education, advocacy, patient engagement, and patient communities.

    aakp.org
  • American Kidney Fund (AKF)

    AKF is a nonprofit organization serving the millions of Americans with kidney disease. AKF helps people fight kidney disease and live healthier lives by providing a wide range of programs and services, including prevention activities, educational resources, and financial assistance.

    kidneyfund.org/kidney-disease/other-kidney-conditions/rare-diseases/cystinosis
  • Dialysis Patient Citizens (DPC)

    DPC is a patient-led, nonprofit organization dedicated to improving dialysis patients’ quality of life by advocating for favorable public policy.

    dialysispatients.org

Patient Advocacy Organizations for Those Living With a Rare Disease

  • Center for Chronic Illness (CCI)

    CCI promotes well-being and decreases isolation for those impacted by chronic illness through support and education.

    thecenterforchronicillness.org
  • Global Genes

    Global Genes is a non-profit, advocacy organization for patients and families fighting rare and genetic diseases, including cystinosis. Its mission is to eliminate the challenges of rare disease.

    globalgenes.org
  • National Organization for Rare Disorders (NORD)

    TNORD is a patient advocacy organization dedicated to people with rare diseases and the groups that help them. NORD provides patients and families with advocacy information, assistance programs, and connections to patient organizations.

    rarediseases.org
  • EveryLife Foundation for Rare Diseases

    The EveryLife Foundation for Rare Diseases is dedicated to accelerating biotech innovation for rare disease treatments through science-driven public information.

    EveryLifeFoundation.org
  • ANGEL AID

    ANGEL AID connects rare communities to teach the tools of self-care and how to be listened to without judgment.

    angelaidcares.org
  • Our Odyssey

    Our Odyssey’s mission is connecting young adults impacted by a rare or chronic condition with social and emotional support in the hope of improving their quality of life.

    ourodyssey.org
  • Next Step

    Next Step is a non-profit that shatters limitations and elevates aspirations of young people with serious illness during their transition to adulthood through campferences, mentorship, music therapy, and other tailored programming.

    nextstepnet.org

Share your Story

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Share Your Story

Tell Us Your Story About Life With Cystinosis

Every patient’s Cystinosis story is an important one. We hope you’ll inspire others living with Cystinosis by sharing yours here.

Learn how Amgen By Your Side provides support services for patients on PROCYSBI

Learn about resources that can help caregivers help themselves

INDICATION

Procysbi® (pegloticase) is indicated for the treatment of chronic gout in adult patients who have failed to normalize serum uric acid and whose signs and symptoms are inadequately controlled with xanthine oxidase inhibitors at the maximum medically appropriate dose or for whom these drugs are contraindicated.

Limitations of Use: KRYSTEXXA is not recommended for the treatment of asymptomatic hyperuricemia.

IMPORTANT SAFETY INFORMATION

WARNING: ANAPHYLAXIS AND INFUSION REACTIONS, G6PD DEFICIENCY ASSOCIATED HEMOLYSIS AND METHEMOGLOBINEMIA

  • Anaphylaxis and infusion reactions have been reported to occur during and after administration of KRYSTEXXA.
  • Anaphylaxis may occur with any infusion, including a first infusion, and generally manifests within 2 hours of the infusion. Delayed hypersensitivity reactions have also been reported.
  • KRYSTEXXA should be administered in healthcare settings and by healthcare providers prepared to manage anaphylaxis and infusion reactions.
  • Premedicate with antihistamines and corticosteroids and closely monitor for anaphylaxis for an appropriate period after administration of KRYSTEXXA.
  • Monitor serum uric acid levels prior to each infusion and discontinue treatment if levels increase to above 6 mg/dL, particularly when 2 consecutive levels above 6 mg/dL are observed.
  • Screen patients at risk for glucose-6-phosphate dehydrogenase (G6PD) deficiency prior to starting KRYSTEXXA. Hemolysis and methemoglobinemia have been reported with KRYSTEXXA in patients with G6PD deficiency. KRYSTEXXA is contraindicated in patients with G6PD deficiency.

CONTRAINDICATIONS:

  • In patients with G6PD deficiency.
  • In patients with history of serious hypersensitivity reactions, including anaphylaxis, to KRYSTEXXA or any of its components.

WARNINGS AND PRECAUTIONS

Gout Flares: An increase in gout flares is frequently observed upon initiation of anti-hyperuricemic therapy, including KRYSTEXXA. Gout flare prophylaxis with a non-steroidal anti-inflammatory drug (NSAID) or colchicine is recommended starting at least 1 week before initiation of KRYSTEXXA therapy and lasting at least 6 months, unless medically contraindicated or not tolerated.

Congestive Heart Failure: KRYSTEXXA has not been formally studied in patients with congestive heart failure, but some patients in the pre-marketing placebo-controlled clinical trials experienced exacerbation. Exercise caution in patients who have congestive heart failure and monitor patients closely following infusion.

ADVERSE REACTIONS

The most commonly reported adverse reactions (≥5%) are:

KRYSTEXXA co-administration with methotrexate trial:
KRYSTEXXA with methotrexate: gout flares, arthralgia, COVID-19, nausea, and fatigue; KRYSTEXXA alone: gout flares, arthralgia, COVID-19, nausea, fatigue, infusion reaction, pain in extremity, hypertension, and vomiting.

KRYSTEXXA pre-marketing placebo-controlled trials:
gout flares, infusion reactions, nausea, contusion or ecchymosis, nasopharyngitis, constipation, chest pain, anaphylaxis, and vomiting.

Please see Full Prescribing Information, including Boxed Warning.

INDICATION

KRYSTEXXA® (pegloticase) is indicated for the treatment of chronic gout in adult patients who have failed to normalize serum uric acid and whose signs and symptoms are inadequately controlled with xanthine oxidase inhibitors at the maximum medically appropriate dose or for whom these drugs are contraindicated.

Limitations of Use: Procysbi is not recommended for the treatment of asymptomatic hyperuricemia.

IMPORTANT SAFETY INFORMATION

WARNING: ANAPHYLAXIS AND INFUSION REACTIONS, G6PD DEFICIENCY ASSOCIATED HEMOLYSIS AND METHEMOGLOBINEMIA

  • Anaphylaxis and infusion reactions have been reported to occur during and after administration of KRYSTEXXA.
  • Anaphylaxis may occur with any infusion, including a first infusion, and generally manifests within 2 hours of the infusion. Delayed hypersensitivity reactions have also been reported.
  • KRYSTEXXA should be administered in healthcare settings and by healthcare providers prepared to manage anaphylaxis and infusion reactions.
  • Premedicate with antihistamines and corticosteroids and closely monitor for anaphylaxis for an appropriate period after administration of KRYSTEXXA.
  • Monitor serum uric acid levels prior to each infusion and discontinue treatment if levels increase to above 6 mg/dL, particularly when 2 consecutive levels above 6 mg/dL are observed.
  • Screen patients at risk for glucose-6-phosphate dehydrogenase (G6PD) deficiency prior to starting KRYSTEXXA. Hemolysis and methemoglobinemia have been reported with KRYSTEXXA in patients with G6PD deficiency. KRYSTEXXA is contraindicated in patients with G6PD deficiency.

CONTRAINDICATIONS:

  • In patients with G6PD deficiency.
  • In patients with history of serious hypersensitivity reactions, including anaphylaxis, to KRYSTEXXA or any of its components.

WARNINGS AND PRECAUTIONS

Gout Flares: An increase in gout flares is frequently observed upon initiation of anti-hyperuricemic therapy, including KRYSTEXXA. Gout flare prophylaxis with a non-steroidal anti-inflammatory drug (NSAID) or colchicine is recommended starting at least 1 week before initiation of KRYSTEXXA therapy and lasting at least 6 months, unless medically contraindicated or not tolerated.

Congestive Heart Failure: KRYSTEXXA has not been formally studied in patients with congestive heart failure, but some patients in the pre-marketing placebo-controlled clinical trials experienced exacerbation. Exercise caution in patients who have congestive heart failure and monitor patients closely following infusion.

ADVERSE REACTIONS

The most commonly reported adverse reactions (≥5%) are:

KRYSTEXXA co-administration with methotrexate trial:
KRYSTEXXA with methotrexate: gout flares, arthralgia, COVID-19, nausea, and fatigue; KRYSTEXXA alone: gout flares, arthralgia, COVID-19, nausea, fatigue, infusion reaction, pain in extremity, hypertension, and vomiting.

KRYSTEXXA pre-marketing placebo-controlled trials:
gout flares, infusion reactions, nausea, contusion or ecchymosis, nasopharyngitis, constipation, chest pain, anaphylaxis, and vomiting.

Please see Full Prescribing Information, including Boxed Warning.