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 a Urea Cycle Disorder (UCD). 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 RAVICTI® (glycerol phenylbutyrate) Oral Liquid page for patients.

PAL Icon

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.

Personalized Support

Hear what a Horizon PAL has to say about how she helps patients

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Additional Patient Resources

UCD Mentors

When you or a loved one gets diagnosed with a UCD, it can feel like your whole world has been turned upside down. Questions come up like: What now? How is this going to affect my life? Where do I go for advice? Who can I talk to? You’ll be glad to know there is a community of people with UCDs who have been in your shoes and are happy to talk to you. If you’d like to talk with another UCD patient or caregiver, sign up to speak with one of our UCD Mentors.

Sign Up

RAVICTI Facebook Group

A page for anyone who has a UCD or cares for someone who does, and is taking, considering, or wants to learn more about RAVICTI.

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Patient Advocacy Organizations for Those Living With a Urea Cycle Disorder (UCD)
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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

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

thecenterforchronicillness.org

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 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

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

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

angelaidcares.org

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 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

National PKU News provides resources and support for individuals, families, and clinicians managing inborn errors of metabolism.

pkunews.org

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

Program Enrollment

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

Forms and Downloads
Enroll Your Patient
Patient Enrollment Form

Download PDF

Learn more about patient enrollment

INDICATION and IMPORTANT SAFETY INFORMATION

INDICATION

RAVICTI (glycerol phenylbutyrate) Oral Liquid is indicated for use as a nitrogen-binding agent for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g. essential amino acids, arginine, citrulline, protein-free calorie supplements).

LIMITATIONS OF USE

  • RAVICTI is not indicated for the treatment of acute hyperammonemia in patients with UCDs because more rapidly acting interventions are essential to reduce plasma ammonia levels.
  • The safety and efficacy of RAVICTI for the treatment of N-acetylglutamate synthase (NAGS) deficiency has not been established.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

  • Patients with known hypersensitivity to phenylbutyrate: Reactions include wheezing, dyspnea, coughing, hypotension, flushing, nausea, and rash.

WARNINGS AND PRECAUTIONS

  • Neurotoxicity: Phenylacetate (PAA), the major metabolite of RAVICTI, may be toxic at levels of 500 micrograms/mL or greater. If symptoms of vomiting, nausea, headache, somnolence, or confusion, are present in the absence of high ammonia or other intercurrent illness which explains these symptoms, consider the potential for PAA neurotoxicity which may need reduction in the RAVICTI dosage.
  • Pancreatic Insufficiency or Intestinal Malabsorption: Low or absent pancreatic enzymes or intestinal disease resulting in fat malabsorption may result in reduced or absent digestion of RAVICTI and/or absorption of phenylbutyrate and reduced control of plasma ammonia. Monitor ammonia levels closely.

ADVERSE REACTIONS

The most common adverse reactions reported in clinical trials (at least 10% of patients) were:

  • Adult patients: diarrhea, flatulence, and headache occurred during 4-week treatment (n=45) with RAVICTI; nausea, vomiting, diarrhea, decreased appetite, dizziness, headache, and fatigue occurred during 12-month treatment (n=51) with RAVICTI.
  • Pediatric patients ages 2 to 17 years: upper abdominal pain, rash, nausea, vomiting, diarrhea, decreased appetite, and headache occurred during 12-month treatment (n=26) with RAVICTI.
  • Pediatric patients ages 2 months to less than 2 years: neutropenia, vomiting, constipation, diarrhea, pyrexia, hypophagia, cough, nasal congestion, rhinorrhea, rash, and papule occurred during 12-month treatment (n=17) with RAVICTI.
  • Pediatric patients less than 2 months of age: vomiting, rash, gastroesophageal reflux, increased hepatic enzymes, feeding disorder (decreased appetite, hypophagia), anemia, cough, dehydration, metabolic acidosis, thrombocytosis, thrombocytopenia, neutropenia, lymphocytosis, diarrhea, flatulence, constipation, pyrexia, lethargy, and irritability/agitation occurred during 24-month treatment (n=16) with RAVICTI.

DRUG INTERACTIONS

  • Corticosteroids, valproic acid, or haloperidol may increase plasma ammonia level. Monitor ammonia levels closely.
  • Probenecid may affect renal excretion of metabolites of RAVICTI, including phenylacetylglutamine (PAGN) and PAA.
  • CYP3A4 substrates with narrow therapeutic index (eg, alfentanil, quinidine, cyclosporine): RAVICTI may decrease exposure to the concomitant drug.
  • Midazolam: Use of RAVICTI decreased exposure of midazolam with concomitant use.

USE IN SPECIFIC POPULATIONS

  • Pregnancy: RAVICTI should be used with caution in patients who are pregnant or planning to become pregnant. Based on animal data, RAVICTI may cause fetal harm. Report pregnancies to Horizon at 1‐866‐479‐6742.
  • Lactation: breastfeeding is not recommended during treatment with RAVICTI. There are no data on the presence of RAVICTI in human milk, the effects on the breastfed infant, nor the effects on milk production.

Please see Full Prescribing Information.

INDICATION and IMPORTANT SAFETY INFORMATION

INDICATION

RAVICTI (glycerol phenylbutyrate) Oral Liquid is indicated for use as a nitrogen-binding agent for chronic management of patients with urea cycle disorders (UCDs) who cannot be managed by dietary protein restriction and/or supplementation alone. RAVICTI must be used with dietary protein restriction and, in some cases, dietary supplements (e.g. essential amino acids, arginine, citrulline, protein-free calorie supplements).

LIMITATIONS OF USE

  • RAVICTI is not indicated for the treatment of acute hyperammonemia in patients with UCDs because more rapidly acting interventions are essential to reduce plasma ammonia levels.
  • The safety and efficacy of RAVICTI for the treatment of N-acetylglutamate synthase (NAGS) deficiency has not been established.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

  • Patients with known hypersensitivity to phenylbutyrate: Reactions include wheezing, dyspnea, coughing, hypotension, flushing, nausea, and rash.

WARNINGS AND PRECAUTIONS

  • Neurotoxicity: Phenylacetate (PAA), the major metabolite of RAVICTI, may be toxic at levels of 500 micrograms/mL or greater. If symptoms of vomiting, nausea, headache, somnolence, or confusion, are present in the absence of high ammonia or other intercurrent illness which explains these symptoms, consider the potential for PAA neurotoxicity which may need reduction in the RAVICTI dosage.
  • Pancreatic Insufficiency or Intestinal Malabsorption: Low or absent pancreatic enzymes or intestinal disease resulting in fat malabsorption may result in reduced or absent digestion of RAVICTI and/or absorption of phenylbutyrate and reduced control of plasma ammonia. Monitor ammonia levels closely.

ADVERSE REACTIONS

The most common adverse reactions reported in clinical trials (at least 10% of patients) were:

  • Adult patients: diarrhea, flatulence, and headache occurred during 4-week treatment (n=45) with RAVICTI; nausea, vomiting, diarrhea, decreased appetite, dizziness, headache, and fatigue occurred during 12-month treatment (n=51) with RAVICTI.
  • Pediatric patients ages 2 to 17 years: upper abdominal pain, rash, nausea, vomiting, diarrhea, decreased appetite, and headache occurred during 12-month treatment (n=26) with RAVICTI.
  • Pediatric patients ages 2 months to less than 2 years: neutropenia, vomiting, constipation, diarrhea, pyrexia, hypophagia, cough, nasal congestion, rhinorrhea, rash, and papule occurred during 12-month treatment (n=17) with RAVICTI.
  • Pediatric patients less than 2 months of age: vomiting, rash, gastroesophageal reflux, increased hepatic enzymes, feeding disorder (decreased appetite, hypophagia), anemia, cough, dehydration, metabolic acidosis, thrombocytosis, thrombocytopenia, neutropenia, lymphocytosis, diarrhea, flatulence, constipation, pyrexia, lethargy, and irritability/agitation occurred during 24-month treatment (n=16) with RAVICTI.

DRUG INTERACTIONS

  • Corticosteroids, valproic acid, or haloperidol may increase plasma ammonia level. Monitor ammonia levels closely.
  • Probenecid may affect renal excretion of metabolites of RAVICTI, including phenylacetylglutamine (PAGN) and PAA.
  • CYP3A4 substrates with narrow therapeutic index (eg, alfentanil, quinidine, cyclosporine): RAVICTI may decrease exposure to the concomitant drug.
  • Midazolam: Use of RAVICTI decreased exposure of midazolam with concomitant use.

USE IN SPECIFIC POPULATIONS

  • Pregnancy: RAVICTI should be used with caution in patients who are pregnant or planning to become pregnant. Based on animal data, RAVICTI may cause fetal harm. Report pregnancies to Horizon at 1‐866‐479‐6742.
  • Lactation: breastfeeding is not recommended during treatment with RAVICTI. There are no data on the presence of RAVICTI in human milk, the effects on the breastfed infant, nor the effects on milk production.

Please see Full Prescribing Information.