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

Resources to Support You and Your Patients Every Step of the Way

Discover helpful resources to share with your office, patient, or their caregivers. These digital resources can help them get started or learn more about the support available while on PROCYSBI® (cysteamine bitartrate) delayed-release capsules or delayed-release oral granules. If you are a patient, you can learn more from these important tools or by visiting the PROCYSBI page for patients. page for patients..

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Patient Access Liaison (PAL)

The Patient Access Liaison (PAL) is a dedicated support partner who helps investigate, explain, and educate on each step of the treatment experience. They will be your patient’s point of contact and a constant champion in helping accomplish their treatment goals. Learn more about contacting a PAL and why you should connect with one.

Hear what an Amgen PAL has to say about how she helps patients

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PAL-Video
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Carol: When I'm going to be meeting with the patient and their family, explaining what my role is, I want them to understand that I will be a point person for them

First and foremost, I'm there to listen to what their story is and also let them know that I will be there to support them and ultimately to empower them.

I look at my role as being able to take as much off their plate as I can as they navigate that process, but also helping them understand in layman's terms where they are in the process and what they need to do on their end.

We don't want patients to feel that they're just out there alone and floating in the space of the unknown. We want patients to know they can reach out to us at any time or a family member or a caregiver, we're always available to them.

Amgen is definitely a company that puts patients first.

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

Independent 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)

    NORD 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 policy.

    everylifefoundation.org
  • ANGEL AID

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

    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

Once you decide PROCYSBI is right for your patient, it’s easy to initiate their enrollment in Amgen By Your Side

Familiarize yourself with important forms and downloadable resources that explain the process

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INDICATION and IMPORTANT SAFETY INFORMATION

INDICATION

PROCYSBI (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules is a cystine-depleting agent indicated for the treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

  • Patients with serious hypersensitivity reaction, including anaphylaxis to penicillamine or cysteamine.

WARNINGS AND PRECAUTIONS

  • Ehlers-Danlos-like Syndrome: Skin and bone lesions that resemble clinical findings for Ehlers-Danlos-like syndrome have been reported in patients treated with high doses of immediate-release cysteamine bitartrate or other cysteamine salts. Monitor patients for development of skin or bone lesions and reduce PROCYSBI dosing if patients develop these lesions.
  • Skin Rash: Severe skin rashes such as erythema multiforme bullosa or toxic epidermal necrolysis have been reported in patients receiving immediate-release cysteamine bitartrate. Discontinue use if severe skin rash occurs.
  • Gastrointestinal (GI) Ulcers and Bleeding: GI ulceration and bleeding have been reported in patients receiving immediate-release cysteamine bitartrate. Monitor for GI symptoms and consider decreasing the dose if severe symptoms occur.
  • Fibrosing Colonopathy: Fibrosing colonopathy has been reported with postmarketing use of PROCYSBI. Evaluate patients with severe, persistent, and/or worsening abdominal symptoms for fibrosing colonopathy. If the diagnosis is confirmed, permanently discontinue PROCYSBI and switch to immediate-release cysteamine bitartrate capsules.
  • Central Nervous System (CNS) Symptoms: CNS symptoms such as seizures, lethargy, somnolence, depression, and encephalopathy have been associated with immediate-release cysteamine. Monitor for CNS symptoms; interrupt or reduce the dose for severe symptoms or those that persist or progress.
  • Leukopenia and/or Elevated Alkaline Phosphatase Levels: Cysteamine has been associated with reversible leukopenia and elevated alkaline phosphatase levels. Monitor white blood cell counts and alkaline phosphatase levels; decrease or discontinue the dose until values revert to normal.
  • Benign lntracranial Hypertension: Benign intracranial hypertension (pseudotumor cerebri; PTC) and/or papilledema has been reported in patients receiving immediate-release cysteamine bitartrate treatment. Monitor for signs and symptoms of PTC; interrupt or reduce the dose for signs/symptoms that persist, or discontinue if diagnosis is confirmed.

ADVERSE REACTIONS

The most common adverse reactions reported in PROCYSBI clinical trials (≥ 5%) were:

 

  • Patients 2 years of age and older previously treated with cysteamine: vomiting, nausea, abdominal pain, headache, conjunctivitis, influenza, gastroenteritis, nasopharyngitis, dehydration, ear infection, upper respiratory tract infection, fatigue, arthralgia, cough, and pain in extremity.
  • Patients 1 year of age and older naïve to cysteamine treatment: vomiting, gastroenteritis/viral gastroenteritis, diarrhea, breath odor, nausea, electrolyte imbalance, headache.

DRUG INTERACTIONS

  • Drugs that increase gastric pH may alter the pharmacokinetics of cysteamine due to the premature release of cysteamine from PROCYSBI and increase WBC cystine concentration. Monitor WBC cystine concentration with concomitant use.
  • Consumption of alcohol with PROCYSBI may increase the rate of cysteamine release and/or adversely alter the pharmacokinetic properties, as well as the effectiveness and safety of PROCYSBI.
  • PROCYSBI can be administered with electrolyte (except bicarbonate) and mineral replacements necessary for management of Fanconi Syndrome as well as vitamin D and thyroid hormone.

USE IN SPECIFIC POPULATIONS

  • Lactation: Because of the potential risk for serious adverse reactions in breastfed children from cysteamine, breastfeeding is not recommended during treatment with PROCYSBI.

Please see Full Prescribing Information.

INDICATION and IMPORTANT SAFETY INFORMATION

INDICATION

PROCYSBI (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules is a cystine-depleting agent indicated for the treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

  • Patients with serious hypersensitivity reaction, including anaphylaxis to penicillamine or cysteamine.

WARNINGS AND PRECAUTIONS

  • Ehlers-Danlos-like Syndrome: Skin and bone lesions that resemble clinical findings for Ehlers-Danlos-like syndrome have been reported in patients treated with high doses of immediate-release cysteamine bitartrate or other cysteamine salts. Monitor patients for development of skin or bone lesions and reduce PROCYSBI dosing if patients develop these lesions.
  • Skin Rash: Severe skin rashes such as erythema multiforme bullosa or toxic epidermal necrolysis have been reported in patients receiving immediate-release cysteamine bitartrate. Discontinue use if severe skin rash occurs.
  • Gastrointestinal (GI) Ulcers and Bleeding: GI ulceration and bleeding have been reported in patients receiving immediate-release cysteamine bitartrate. Monitor for GI symptoms and consider decreasing the dose if severe symptoms occur.
  • Fibrosing Colonopathy: Fibrosing colonopathy has been reported with postmarketing use of PROCYSBI. Evaluate patients with severe, persistent, and/or worsening abdominal symptoms for fibrosing colonopathy. If the diagnosis is confirmed, permanently discontinue PROCYSBI and switch to immediate-release cysteamine bitartrate capsules.
  • Central Nervous System (CNS) Symptoms: CNS symptoms such as seizures, lethargy, somnolence, depression, and encephalopathy have been associated with immediate-release cysteamine. Monitor for CNS symptoms; interrupt or reduce the dose for severe symptoms or those that persist or progress.
  • Leukopenia and/or Elevated Alkaline Phosphatase Levels: Cysteamine has been associated with reversible leukopenia and elevated alkaline phosphatase levels. Monitor white blood cell counts and alkaline phosphatase levels; decrease or discontinue the dose until values revert to normal.
  • Benign lntracranial Hypertension: Benign intracranial hypertension (pseudotumor cerebri; PTC) and/or papilledema has been reported in patients receiving immediate-release cysteamine bitartrate treatment. Monitor for signs and symptoms of PTC; interrupt or reduce the dose for signs/symptoms that persist, or discontinue if diagnosis is confirmed.

ADVERSE REACTIONS

The most common adverse reactions reported in PROCYSBI clinical trials (≥ 5%) were:

  • Patients 2 years of age and older previously treated with cysteamine: vomiting, nausea, abdominal pain, headache, conjunctivitis, influenza, gastroenteritis, nasopharyngitis, dehydration, ear infection, upper respiratory tract infection, fatigue, arthralgia, cough, and pain in extremity.
  • Patients 1 year of age and older naïve to cysteamine treatment: vomiting, gastroenteritis/viral gastroenteritis, diarrhea, breath odor, nausea, electrolyte imbalance, headache.

DRUG INTERACTIONS

  • Drugs that increase gastric pH may alter the pharmacokinetics of cysteamine due to the premature release of cysteamine from PROCYSBI and increase WBC cystine concentration. Monitor WBC cystine concentration with concomitant use.
  • Consumption of alcohol with PROCYSBI may increase the rate of cysteamine release and/or adversely alter the pharmacokinetic properties, as well as the effectiveness and safety of PROCYSBI.
  • PROCYSBI can be administered with electrolyte (except bicarbonate) and mineral replacements necessary for management of Fanconi Syndrome as well as vitamin D and thyroid hormone.

USE IN SPECIFIC POPULATIONS

  • Lactation: Because of the potential risk for serious adverse reactions in breastfed children from cysteamine, breastfeeding is not recommended during treatment with PROCYSBI.

Please see Full Prescribing Information.