Cost Savings and Support

FINDING HELP ALONG THE CYSTINOSIS JOURNEY

Horizon Patient Services

Horizon Patient Services is dedicated to improving the lives of those living with cystinosis. This service provides ongoing individualized support and education for you and your family.

Your Patient Access Manager (PAM) leads your dedicated Horizon Patient Services team that is here to help you, from answering questions as you start taking PROCYSBI and through every step along the way. Your PAM is available in person or by email, text, or phone with information and guidance.

To contact a PAM, call 1-833-830-RARE (1-833-830-7273). These comprehensive services are built around three components: coordinate, connect, and champion.

Your Horizon Patient Services team will:

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  • Work with you and your doctor's office to simplify the process of getting started on your medicine
  • Help address financial barriers by researching your insurance benefits, finding out what your insurance will cover, explaining your insurance options, and connecting you to financial assistance options
  • Assist in connecting with your specialty pharmacy to schedule the shipment of your medicine so that you avoid running out of supply

Cystinosis Community Icon
  • Connect you and your family to others living with cystinosis through live events and online resources
  • Provide helpful tools and resources to manage the day-to-day challenges of living with cystinosis
  • Introduce you and your family to cytinosis advocacy groups for more support and inform you of cystinosis events in your area

Cystinosis Prescription Help Icon
  • Serve as your dedicated personal resource and the main point of contact for your ongoing PROCYSBI logistical needs
  • Provide cystinosis education and answer nontreatment-related questions
  • Help you through changes along the way that may impact your treatment
  • Respond to your questions and concerns, and connect you with the right resources to get answers

To get started with Horizon Patient Services, call 855-888-4004

Cystinosis Cost Savings Icon

Patient Access Managers: Because it’s personal

    Your Patient Access Manager (PAM) is available to support you as a personal resource. Your PAM can help with insurance, travel and financial assistance, life transitions, and helping to link you to the cystinosis community. Your PAM is there for regular talks to answer questions and provide support to you and your family.

    HEAR HOW PAMS HELP PEOPLE WITH CYSTINOSIS

    Read video transcript

    Our PAM plays a big role in our support system. She helps us any time we need help with medicine issues, delivery problems, insurance issues.

    They can also travel to you if you’re a new diagnosis, to your home, and teach you how to properly administer your PROCYSBI through your child’s G-tube. They can help you in many ways.

    We had an issue with one of our shipments that didn’t come in. And so, we reached out to our patient access manager and we got it delivered.

    A PAM, to me, is someone that helps you learn how to be an advocate for yourself.

    A while back, I lost my insurance, which can be very stressful dealing with cystinosis. You need your medications to live really. And, she’s helped me to figure out how to pay for these medications while we’re dealing with these appeals and things like that.

    When my dad changed jobs our insurance no longer covered PROCYSBI. So my PAM was super influential in helping us find another provider for PROCYSBI.

    She’s been very influential in me getting my cystine level drawn. She’s been very helpful in that process and making sure the lab is ready and knows that I’m coming.

    When we sent our first kit in, it didn’t process properly. I reached out to my PAM and she basically was able to track the kit down and get it corrected for the next time we tested.

    The involvement I’ve had with the cystinosis community through the years has been awesome. My PAM has helped me get connections to these cystinosis events by getting me connected to the right people.

    My PAM has helped us connect with all kinds of outside resources and organizations to help my family and I navigate through this entire process of having cystinosis.

    Our PAM is more than just a PAM to us. She’s, she’s a friend.

    She’s my friend. We draw with her. Play tic-tac-toe. When Mom is done talking to her, I ask her if she wants to play with me.

    My PAM means a lot to me. I’d honestly be lost without my PAM.

    She always wants to know not just about me but how my mom is doing, how my dad is doing. I love that she’s genuinely intrigued in our family and invested.

    I think that it’s very important to have somebody cheering you on and being on your team that’s outside of your immediate family, outside of your immediate friend group. And it’s, I know it’s uplifted me.

    I would advise everybody to reach out and know their PAM.

    If you have been prescribed PROCYSBI® (cysteamine bitartrate) delayed-release capsules or are interested in learning more, you can connect with your PAM at 1-855-888-4004 or HPSPRO@horizontherapeutics.com.

    APPROVED USES and IMPORTANT SAFETY INFORMATION for PROCYSBI
    What is PROCYSBI?

    PROCYSBI (cysteamine bitartrate) delayed-release capsules is a prescription medicine used to treat a medical condition called nephropathic cystinosis in adults and children 1 year of age and older. It is not known if PROCYSBI is safe and effective in children under 1 year of age.

    IMPORTANT SAFETY INFORMATION
    What is the most important safety information I should know about PROCYSBI?
    PROCYSBI can cause serious side effects, including:
    • Skin, bone, and joint problems. People treated with high doses of cysteamine bitartrate may develop abnormal changes of their skin and bones, such as stretch marks, bone injuries (such as fractures), bone deformities, and joint problems. Check your skin while taking PROCYSBI. Tell your doctor if you notice any skin changes or problems with your bones or joints. Your doctor will check you for these problems.
    • Skin rash. Skin rash is common with cysteamine bitartrate and may sometimes be severe. Your dose of PROCYSBI may need to be decreased until the rash goes away. If the rash is severe, your doctor may tell you to stop taking PROCYSBI. Tell your doctor right away if you get a skin rash.
    • Stomach and bowel (intestinal) problems. Some people who take other medicines that contain cysteamine bitartrate develop ulcers and bleeding in their stomach or bowel. Tell your doctor right away if you get stomach-area pain, nausea, vomiting, loss of appetite, or vomit blood.
    • Central nervous system symptoms. Some people who take other medicines that contain cysteamine bitartrate develop seizures, depression, and become very sleepy. The medicine may affect how your brain is working (encephalopathy). Tell your doctor right away if you develop any of these symptoms.
    • Low white blood cell count and certain abnormal liver function blood tests. Your doctor should check you for these problems.
    • Benign intracranial hypertension (pseudotumor cerebri) has happened in some people who take immediate-release cysteamine bitartrate. This is a condition where there is high pressure in the fluid around the brain. Your doctor should do eye examinations to find and treat this problem early. Tell your doctor right away if you develop any of the following symptoms while taking PROCYSBI: headache, buzzing or "whooshing" sound in the ear, dizziness, nausea, double vision, blurry vision, loss of vision, pain behind the eye, or pain with eye movement.
    Who should not take PROCYSBI?

    Do not take PROCYSBI if you are allergic to penicillamine or cysteamine.

    What should I tell my doctor before taking PROCYSBI?
    Tell your doctor if you have any other medical conditions, including if you:
    • drink alcohol.
    • have a skin rash or bone problems.
    • have or have had stomach or bowel (intestinal) problems including ulcers or bleeding.
    • have a history of seizures, lack of energy, unusual sleepiness, depression, or changes in your ability to think clearly.
    • have liver or blood problems.
    • are pregnant or plan to become pregnant. It is not known if PROCYSBI will harm your unborn baby. Tell your doctor right away if you think that you are pregnant. Talk with your doctor about the benefits and risks of taking PROCYSBI during pregnancy.
    • are breastfeeding or plan to breastfeed. You should not breastfeed during treatment with PROCYSBI. Talk with your doctor about the best way to feed your baby if you take PROCYSBI.
    What should I avoid while taking PROCYSBI?
    • Do not drive or operate machinery until you know how PROCYSBI affects you. PROCYSBI can make you sleepy or less alert than normal.
    • Do not drink alcohol if you take PROCYSBI. Drinking alcohol while taking PROCYSBI may change how PROCYSBI works and may cause an increase in the amount of PROCYSBI in your blood that may cause serious side effects.
    What are the possible side effects of PROCYSBI?
    • See “What is the most important information I should know about PROCYSBI?”

    The most common side effects of PROCYSBI include: vomiting, nausea, stomach (abdominal) pain, breath odor, diarrhea, skin odor, tiredness, skin rash, headache, problems with body salts or electrolytes.

    These are not all of the possible side effects of PROCYSBI. Call your doctor for medical information about side effects.

    For additional important safety information, see the Patient Information and Instructions for Use available at PROCYSBI.com and discuss with your doctor.

    You are encouraged to report negative side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch or call 1-800-FDA-1088.

    Your PAM is there to:

    • Guide you along your treatment journey
    • Answer your questions
    • Connect you with resources
    • Help prepare you for treatment

    To get in touch with your PAM, just call 844-830-CARE (844-830-2273), and PAMs are also available via email or text, or in person.

    You can also sign up to connect with a PAM

    Mortar

    Helping with your PROCYSBI prescription

      Horizon Patient Services can help get you started on PROCYSBI and will help you connect with the specialty pharmacy. PROCYSBI is only available through a specialty pharmacy and service provider for patients.

      Your PROCYSBI prescription is filled and sent to your home through a package delivery service. PAMs can help keep patients on track with refills and answer any questions you have.

      Talk to your doctor about a PROCYSBI prescription. The Prescription Enrollment Form found here is required, and your doctor will assist you in filling it out.

      To avoid a gap in treatment, plan ahead. Mark your calendar to contact the specialty pharmacy to schedule a timely delivery of your refill.

      Ordering refills

      When to reorder PROCYSBI:

      • Before you run out
      • Before the discard date written by your pharmacist

      When you fill your prescription for PROCYSBI, there should be exactly enough capsules in the bottle to last until your next refill arrives. Do not use expired PROCYSBI.

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      Impact Programs

        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

          Find a list of Impact Programs taking place in cities across the country this year.


          Show me upcoming Impact Programs

          Choose the location that works best for you and your family:

          • Please save this link and check back for updated event dates in the future.

          For more information on Impact Programs or to suggest a city near you for a future program, contact us at impact@horizontherapeutics.com or call 602-953-2552.

          Cystinosis resources and support

          Visit the following for more support:

          APPROVED USES and IMPORTANT SAFETY INFORMATION for PROCYSBI

          What is PROCYSBI?

          PROCYSBI (cysteamine bitartrate) delayed-release capsules is a prescription medicine used to treat a medical condition called nephropathic cystinosis in adults and children 1 year of age and older. It is not known if PROCYSBI is safe and effective in children under 1 year of age.

          IMPORTANT SAFETY INFORMATION

          What is the most important safety information I should know about PROCYSBI?

          PROCYSBI can cause serious side effects, including:

          • Skin, bone, and joint problems. People treated with high doses of cysteamine bitartrate may develop abnormal changes of their skin and bones, such as stretch marks, bone injuries (such as fractures), bone deformities, and joint problems. Check your skin while taking PROCYSBI. Tell your doctor if you notice any skin changes or problems with your bones or joints. Your doctor will check you for these problems.
          • Skin rash. Skin rash is common with cysteamine bitartrate and may sometimes be severe. Your dose of PROCYSBI may need to be decreased until the rash goes away. If the rash is severe, your doctor may tell you to stop taking PROCYSBI.  Tell your doctor right away if you get a skin rash.
          • Stomach and bowel (intestinal) problems. Some people who take other medicines that contain cysteamine bitartrate develop ulcers and bleeding in their stomach or bowel. Tell your doctor right away if you get stomach-area pain, nausea, vomiting, loss of appetite, or vomit blood.
          • Central nervous system symptoms. Some people who take other medicines that contain cysteamine bitartrate develop seizures, depression, and become very sleepy. The medicine may affect how your brain is working (encephalopathy). Tell your doctor right away if you develop any of these symptoms.
          • Low white blood cell count and certain abnormal liver function blood tests. Your doctor should check you for these problems.
          • Benign intracranial hypertension (pseudotumor cerebri) has happened in some people who take immediate-release cysteamine bitartrate. This is a condition where there is high pressure in the fluid around the brain. Your doctor should do eye examinations to find and treat this problem early. Tell your doctor right away if you develop any of the following symptoms while taking PROCYSBI: headache, buzzing or "whooshing" sound in the ear, dizziness, nausea, double vision, blurry vision, loss of vision, pain behind the eye, or pain with eye movement.

          Who should not take PROCYSBI?

          Do not take PROCYSBI if you are allergic to penicillamine or cysteamine.

          What should I tell my doctor before taking PROCYSBI?

          Tell your doctor if you have any other medical conditions, including if you:

          • drink alcohol.
          • have a skin rash or bone problems.
          • have or have had stomach or bowel (intestinal) problems including ulcers or bleeding.
          • have a history of seizures, lack of energy, unusual sleepiness, depression, or changes in your ability to think clearly.
          • have liver or blood problems.
          • are pregnant or plan to become pregnant. It is not known if PROCYSBI will harm your unborn baby. Tell your doctor right away if you think that you are pregnant. Talk with your doctor about the benefits and risks of taking PROCYSBI during pregnancy.
          • are breastfeeding or plan to breastfeed. You should not breastfeed during treatment with PROCYSBI. Talk with your doctor about the best way to feed your baby if you take PROCYSBI.

          What should I avoid while taking PROCYSBI?

          • Do not drive or operate machinery until you know how PROCYSBI affects you. PROCYSBI can make you sleepy or less alert than normal.
          • Do not drink alcohol if you take PROCYSBI. Drinking alcohol while taking PROCYSBI may change how PROCYSBI works and may cause an increase in the amount of PROCYSBI in your blood that may cause serious side effects.

          What are the possible side effects of PROCYSBI?

          • See “What is the most important information I should know about PROCYSBI?”

          The most common side effects of PROCYSBI include: vomiting, nausea, stomach (abdominal) pain, breath odor, diarrhea, skin odor, tiredness, skin rash, headache, problems with body salts or electrolytes.

          These are not all of the possible side effects of PROCYSBI. Call your doctor for medical information about side effects.

          For additional important safety information, click here for the Full Prescribing Information, 
          Patient Information, and Instructions for Use and discuss with your doctor.

          You are encouraged to report negative side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch or call 1-800-FDA-1088.

          APPROVED USES and IMPORTANT SAFETY INFORMATION for PROCYSBI

          What is PROCYSBI?

          PROCYSBI (cysteamine bitartrate) delayed-release capsules is a prescription medicine used to treat a medical condition called nephropathic cystinosis in adults and children 1 year of age and older. It is not known if PROCYSBI is safe and effective in children under 1 year of age.

          IMPORTANT SAFETY INFORMATION

          What is the most important safety information I should know about PROCYSBI?

          PROCYSBI can cause serious side effects, including:

          • Skin, bone, and joint problems. People treated with high doses of cysteamine bitartrate may develop abnormal changes of their skin and bones, such as stretch marks, bone injuries (such as fractures), bone deformities, and joint problems. Check your skin while taking PROCYSBI. Tell your doctor if you notice any skin changes or problems with your bones or joints. Your doctor will check you for these problems.
          • Skin rash. Skin rash is common with cysteamine bitartrate and may sometimes be severe. Your dose of PROCYSBI may need to be decreased until the rash goes away. If the rash is severe, your doctor may tell you to stop taking PROCYSBI.  Tell your doctor right away if you get a skin rash.
          • Stomach and bowel (intestinal) problems. Some people who take other medicines that contain cysteamine bitartrate develop ulcers and bleeding in their stomach or bowel. Tell your doctor right away if you get stomach-area pain, nausea, vomiting, loss of appetite, or vomit blood.
          • Central nervous system symptoms. Some people who take other medicines that contain cysteamine bitartrate develop seizures, depression, and become very sleepy. The medicine may affect how your brain is working (encephalopathy). Tell your doctor right away if you develop any of these symptoms.
          • Low white blood cell count and certain abnormal liver function blood tests. Your doctor should check you for these problems.
          • Benign intracranial hypertension (pseudotumor cerebri) has happened in some people who take immediate-release cysteamine bitartrate. This is a condition where there is high pressure in the fluid around the brain. Your doctor should do eye examinations to find and treat this problem early. Tell your doctor right away if you develop any of the following symptoms while taking PROCYSBI: headache, buzzing or "whooshing" sound in the ear, dizziness, nausea, double vision, blurry vision, loss of vision, pain behind the eye, or pain with eye movement.

          Who should not take PROCYSBI?

          Do not take PROCYSBI if you are allergic to penicillamine or cysteamine.

          What should I tell my doctor before taking PROCYSBI?

          Tell your doctor if you have any other medical conditions, including if you:

          • drink alcohol.
          • have a skin rash or bone problems.
          • have or have had stomach or bowel (intestinal) problems including ulcers or bleeding.
          • have a history of seizures, lack of energy, unusual sleepiness, depression, or changes in your ability to think clearly.
          • have liver or blood problems.
          • are pregnant or plan to become pregnant. It is not known if PROCYSBI will harm your unborn baby. Tell your doctor right away if you think that you are pregnant. Talk with your doctor about the benefits and risks of taking PROCYSBI during pregnancy.
          • are breastfeeding or plan to breastfeed. You should not breastfeed during treatment with PROCYSBI. Talk with your doctor about the best way to feed your baby if you take PROCYSBI.

          What should I avoid while taking PROCYSBI?

          • Do not drive or operate machinery until you know how PROCYSBI affects you. PROCYSBI can make you sleepy or less alert than normal.
          • Do not drink alcohol if you take PROCYSBI. Drinking alcohol while taking PROCYSBI may change how PROCYSBI works and may cause an increase in the amount of PROCYSBI in your blood that may cause serious side effects.

          What are the possible side effects of PROCYSBI?

          • See “What is the most important information I should know about PROCYSBI?”

          The most common side effects of PROCYSBI include: vomiting, nausea, stomach (abdominal) pain, breath odor, diarrhea, skin odor, tiredness, skin rash, headache, problems with body salts or electrolytes.

          These are not all of the possible side effects of PROCYSBI. Call your doctor for medical information about side effects.

          For additional important safety information, click here for the Full Prescribing Information, 
          Patient Information, and Instructions for Use and discuss with your doctor.

          You are encouraged to report negative side effects of prescription drugs to the FDA. Visit http://www.fda.gov/medwatch or call 1-800-FDA-1088.