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

The resources below are here to help you support your patients and their families living with urea cycle disorders (UCDs). If your patient has a UCD, these resources can assist in managing your patient’s day-to-day challenges. If you are a patient, you can learn more from these important tools or by visiting the BUPHENYL®(sodium phenylbutyrate) Tablets and Powder 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.

Patient Advocacy Organizations for Those Living With UCD

  • National UCD Foundation (NUCDF)

    NUCDF drives innovative research to improve quality of life and leads the search for a cure. A lifeline to UCD patients, families, and medical professionals worldwide seeking information, support, and hope.

    nucdf.org
  • Connecting Families UCD Foundation (CFUCDF)

    CFUCDF has a vision to connect UCD families together, providing support and critical tools to enhance quality of life.

    ucdfamily.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
  • Remember the Girls

    The mission of Remember the Girls is to raise awareness of the many issues facing female carriers of X-linked genetic disorders; to provide a forum for X-linked females to share their stories, ask questions, provide and receive emotional support; and to advocate for increased attention of the medical community to the physical and emotional issues of females who carry X-linked disorders.

    rememberthegirls.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 BUPHENYL 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

BUPHENYL® (sodium phenylbutyrate) Tablets for oral administration and BUPHENYL® (sodium phenylbutyrate) Powder for oral, nasogastric, or gastrostomy tube administration are indicated as adjunctive therapy in the chronic management of patients with urea cycle disorders (UCDs) involving deficiencies of carbamoyl phosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).

BUPHENYL is indicated in all patients with neonatal-onset deficiency (complete enzymatic deficiency, presenting within the first 28 days of life). It is also indicated in patients with late-onset disease (partial enzymatic deficiency, presenting after the first month of life) who have a history of hyperammonemic encephalopathy.

BUPHENYL must be used with dietary protein restriction and, in some cases, essential amino acid supplementation.

Any episode of acute hyperammonemia should be treated as a life-threatening emergency.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

  • Acute hyperammonemia: BUPHENYL should not be used to manage acute hyperammonemia, which is a medical emergency.

WARNINGS AND PRECAUTIONS

BUPHENYL should not be administered to patients with known hypersensitivity to sodium phenylbutyrate or any component of this preparation.

  • Use caution with administering BUPHENYL to patients with:
    • Congestive heart failure or severe renal insufficiency, and in clinical states in which there is sodium retention with edema.
    • Hepatic or renal insufficiency or inborn errors of beta oxidation.
  • Probenecid may affect renal excretion of the conjugated product of BUPHENYL as well as its metabolite.
  • Use of corticosteroids may cause the breakdown of body protein and increase plasma ammonia levels.
  • There have been published reports of hyperammonemia being induced by haloperidol and by valproic acid.

ADVERSE REACTIONS

  • The most common adverse reactions (≥3%) reported in BUPHENYL clinical trials were decreased appetite, body odor, bad taste or taste aversion.
  • In female patients, amenorrhea/menstrual dysfunction (irregular menstrual cycles) occurred in 23% of the menstruating patients.
  • Neurotoxicity was reported in cancer patients receiving intravenous phenylacetate. Manifestations were predominately somnolence, fatigue, and lightheadedness; with less frequent headache, dysgeusia, hypoacusis, disorientation, impaired memory, and exacerbation of a pre-existing neuropathy.
  • Laboratory adverse events occurring in >2% of UCD patients by body system were:
    • Metabolic: acidosis, alkalosis, hyperchloremia, and hypophosphatemia
    • Nutritional: hypoalbuminemia and decreased total protein
    • Hepatic: increased alkaline phosphatase and increased liver transaminases
    • Hematologic: anemia, leukopenia, leukocytosis, and thrombocytopenia

USE IN SPECIFIC POPULATIONS

  • Pregnancy: BUPHENYL should be used with caution in patients who are pregnant or planning to become pregnant. Animal reproduction studies have not been conducted with BUPHENYL. It is not known whether BUPHENYL can cause fetal harm when administered to a pregnant woman or can affect reproduction capacity.
  • Lactation: breastfeeding is not recommended during treatment with BUPHENYL. There are no data on the presence of BUPHENYL in human milk.

Please see Full Prescribing Information.

INDICATION and IMPORTANT SAFETY INFORMATION

INDICATION

BUPHENYL® (sodium phenylbutyrate) Tablets for oral administration and BUPHENYL® (sodium phenylbutyrate) Powder for oral, nasogastric, or gastrostomy tube administration are indicated as adjunctive therapy in the chronic management of patients with urea cycle disorders (UCDs) involving deficiencies of carbamoyl phosphate synthetase (CPS), ornithine transcarbamylase (OTC), or argininosuccinic acid synthetase (AS).

BUPHENYL is indicated in all patients with neonatal-onset deficiency (complete enzymatic deficiency, presenting within the first 28 days of life). It is also indicated in patients with late-onset disease (partial enzymatic deficiency, presenting after the first month of life) who have a history of hyperammonemic encephalopathy.

BUPHENYL must be used with dietary protein restriction and, in some cases, essential amino acid supplementation.

Any episode of acute hyperammonemia should be treated as a life-threatening emergency.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

  • Acute hyperammonemia: BUPHENYL should not be used to manage acute hyperammonemia, which is a medical emergency.

WARNINGS AND PRECAUTIONS

BUPHENYL should not be administered to patients with known hypersensitivity to sodium phenylbutyrate or any component of this preparation.

  • Use caution with administering BUPHENYL to patients with:
    • Congestive heart failure or severe renal insufficiency, and in clinical states in which there is sodium retention with edema.
    • Hepatic or renal insufficiency or inborn errors of beta oxidation.
  • Probenecid may affect renal excretion of the conjugated product of BUPHENYL as well as its metabolite.
  • Use of corticosteroids may cause the breakdown of body protein and increase plasma ammonia levels.
  • There have been published reports of hyperammonemia being induced by haloperidol and by valproic acid.

ADVERSE REACTIONS

  • The most common adverse reactions (≥3%) reported in BUPHENYL clinical trials were decreased appetite, body odor, bad taste or taste aversion.
  • In female patients, amenorrhea/menstrual dysfunction (irregular menstrual cycles) occurred in 23% of the menstruating patients.
  • Neurotoxicity was reported in cancer patients receiving intravenous phenylacetate. Manifestations were predominately somnolence, fatigue, and lightheadedness; with less frequent headache, dysgeusia, hypoacusis, disorientation, impaired memory, and exacerbation of a pre-existing neuropathy.
  • Laboratory adverse events occurring in >2% of UCD patients by body system were:
    • Metabolic: acidosis, alkalosis, hyperchloremia, and hypophosphatemia
    • Nutritional: hypoalbuminemia and decreased total protein
    • Hepatic: increased alkaline phosphatase and increased liver transaminases
    • Hematologic: anemia, leukopenia, leukocytosis, and thrombocytopenia

USE IN SPECIFIC POPULATIONS

  • Pregnancy: BUPHENYL should be used with caution in patients who are pregnant or planning to become pregnant. Animal reproduction studies have not been conducted with BUPHENYL. It is not known whether BUPHENYL can cause fetal harm when administered to a pregnant woman or can affect reproduction capacity.
  • Lactation: breastfeeding is not recommended during treatment with BUPHENYL. There are no data on the presence of BUPHENYL in human milk.

Please see Full Prescribing Information.