TranscendRare™, a HorizonCares™ program, created with input from people with nephropathic cystinosis and their caregivers and healthcare providers who understand the challenges unique to living with and treating this disease. The TranscendRare program provides valuable services to support you and your patients on the journey to manage cystinosis.

For the general nephropathic cystinosis community, TranscendRare offers important information about nephropathic cystinosis, as well as valuable tools to help patients better manage the challenges they may face along the way.

For eligible patients on PROCYSBI (cysteamine bitartrate) delayed-release capsules, TranscendRare offers access and reimbursement support to help them stay on track with treatment.

To get started with TranscendRare, call 855-888-4004
Case managers are available from 8 a.m. to 7 p.m. CST
Nurses are available from 8 a.m. to 7 p.m. CST

TranscendRare: Supporting you and your patients every step of the way

Services and support offered through TranscendRare include:

  • Reimbursement assistance for insured patients
    • TranscendRare advocates on patients’ behalf to help ensure coverage for PROCYSBI and to resolve contested claims
    • Copay assistance will minimize out-of-pocket expenses for eligible patients
  • Reimbursement assistance for underinsured patients
    • TranscendRare helps find available alternate or supplemental insurance if needed
    • Qualified patients may be eligible for financial assistance to help cover premiums
    • If no insurance coverage can be secured, the TranscendRare program may provide PROCYSBI to eligible patients
  • Travel assistance
  • White blood cell (WBC) cystine testing assistance
    • TranscendRare also offers reimbursement assistance to cover the cost associated with WBC cystine testing for eligible patients

Patient Access Managers

Patient Access Managers (PAM) are available to support your patients as a personal resource. A dedicated PAM can help with:

  • Insurance*
    • Review and discuss insurance challenges
    • Troubleshoot problems with insurance payments and prescription refills
    • Connect patients with copay assistance or co-insurance programs
  • Travel and financial assistance*
    • Discuss options for patients who need to travel to see their cystinosis doctor
    • Discuss support for out-of-pocket costs for white blood cell (WBC) cystine testing
  • Life transitions
    • Help your patients stay on track when they are going through life changes, such as:
      • Starting therapy
      • Moving to a new city
      • Changing jobs or going off to college
      • Switching schools or teachers
      • Transitioning to adult care
      • Changing doctors
  • Linking patients to the cystinosis community
    • Connect your patients with other people living with cystinosis
    • Inform your patients of cystinosis events in their area
    • Introduce your patients to cystinosis advocacy groups

*Assistance may include the cost of transportation, lodging, or meals. Services provided by the PAM are for eligible and qualified patients only. Please contact a PAM to determine eligibility.

To learn more, connect with a PAM at 844-830-CARE (2273)
PAMs are also available via e-mail or text or in person

Accredo Specialty Pharmacy

PROCYSBI® (cysteamine bitartrate) delayed-release capsules is available only through Accredo Specialty Pharmacy. TranscendRare and Accredo work together to make sure eligible patients have access to PROCYSBI.

TranscendRare™ Program**
Works directly with your patient’s insurance
Keeps in touch with you and your patients
Refers your patients to other options for financial support
Accredo Specialty Pharmacy
Provides you and your patients with a dedicated contact person
Reminds your patients when it’s time to schedule and ship refills
Delivers medication to your patient’s door
Provide your patient with a PROCYSBI prescription
Return calls and keep in touch with TranscendRare
Remind your patients to return Accredo Specialty Pharmacy’s phone calls

**TranscendRare provides personalized support for eligible patients and caregivers in the United States who are prescribed PROCYSBI. Call for eligibility for assistance with the WBC testing and travel related to cystinosis care.

Cystinosis organizations, advocacy groups, and other resources

Visit these helpful websites for additional information and support for living with cystinosis.


INDICATIONS AND USAGE: PROCYSBI® (cysteamine bitartrate) delayed-release capsules is a cystine depleting agent indicated for the treatment of nephropathic cystinosis in adult and pediatric patients 1 year of age and older.


  • Serious hypersensitivity reaction, including anaphylaxis to penicillamine or cysteamine.


  • 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.
    • These include molluscoid pseudotumors (purplish hemorrhagic lesions), skin striae, bone lesions (including osteopenia, compression fractures, scoliosis and genu valgum), leg pain, and joint hyperextension.
    • One patient on immediate-release cysteamine bitartrate with serious skin lesions subsequently died of acute cerebral ischemia with marked vasculopathy.
    • Monitor patients for development of skin or bone lesions and interrupt PROCYSBI dosing if patients develop these lesions. PROCYSBI may be restarted at a lower dose under close supervision, then slowly increase to the appropriate therapeutic dose.
  • Skin Rash: Severe skin rashes such as erythema multiforme bullosa or toxic epidermal necrolysis have been reported in patients receiving immediate-release cysteamine bitartrate. If severe skin rashes develop, permanently discontinue use of PROCYSBI.
  • Gastrointestinal Ulcers and Bleeding: Gastrointestinal (GI) ulceration and bleeding have been reported in patients receiving immediate-release cysteamine bitartrate.
    • GI tract symptoms including nausea, vomiting, anorexia and abdominal pain, sometimes severe, have been associated with cysteamine. If severe GI tract symptoms develop, consider decreasing the dose of PROCYSBI.
  • Central Nervous System Symptoms: Central Nervous System (CNS) symptoms such as seizures, lethargy, somnolence, depression, and encephalopathy have been associated with immediate-release cysteamine.
    • Neurological complications have also been described in some patients with cystinosis who have not been treated with cysteamine.
    • Carefully evaluate and monitor patients who develop CNS symptoms. Interrupt medication or adjust the dose as necessary for patients with severe symptoms or with symptoms that persist or progress.
    • Inform patients that PROCYSBI may impair their ability to perform tasks such as driving or operating machinery.
  • 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. If tests values remain elevated, consider decreasing the dose or discontinuing the drug until values revert to normal.
  • Benign Intracranial Hypertension: Benign intracranial hypertension (pseudotumor cerebri; PTC) and/or papilledema has been reported in patients receiving immediate-release cysteamine bitartrate treatment.
    • Monitor patients for signs and symptoms of PTC, including headache, tinnitus, dizziness, nausea, diplopia, blurry vision, loss of vision, pain behind the eye or pain with eye movement. If signs/symptoms persist, interrupt dosing or decrease the dose and refer the patient to an ophthalmologist. If the diagnosis is confirmed, permanently discontinue use of PROCYSBI.


The most common adverse reactions (≥5%) in patients treated in clinical trials are vomiting, nausea, gastroenteritis / viral gastroenteritis, abdominal pain, breath odor, diarrhea, skin odor, fatigue, rash, headache, and electrolyte imbalance.

To report SUSPECTED ADVERSE REACTIONS, contact Horizon Pharma USA, Inc. at 1-855-888-4004 or FDA at 1-800-FDA-1088 or



  • Drugs that Increase Gastric pH: Administer PROCYSBI at least 1 hour before or 1 hour after medications containing bicarbonate or carbonate.
  • 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.



  • Breastfeeding is not recommended while taking PROCYSBI.

Part No.: P-PYB-00033                     Derived from Full Prescribing Information December 2017