Prescribing PROCYSBI

How to start your patients on PROCYSBI

Patients can enroll in the TranscendRare™ program before or after they have been prescribed PROCYSBI® (cysteamine bitartrate) delayed-release capsules. TranscendRare, a HorizonCares™ program, will provide the customized support patients need to navigate the insurance process and make the most of their treatment.

Follow these easy steps to start a patient on PROCYSBI.

CallUs
Fill out the Prescription Enrollment Form (PEF)

Have your patient complete the sections that require his or her information. Fill out the “Clinical Information” section and sign the PEF.

Download the PEF ›

CallUs
Meet with your patient

Talk to your patient and determine whether PROCYSBI is right for him or her. Complete the Prescription Enrollment Form (PEF), sign the document, and submit it to TranscendRare to be processed.

Prevent delays on your PROCYSBI prescriptions

A TranscendRare representative or an Accredo Specialty Pharmacy representative may call your practice about your patient’s Prescription Enrollment Form. Please make sure you or a staff member:

  • Return the call as soon as possible.
  • Provide any additional information needed to help process your patient’s prescription.

TranscendRare also offers reimbursement assistance for eligible patients.

Learn more ›

Dosing Information

Patients naïve to cysteamine1

  • Start treatment with a dosage equal to 1/6 to 1/4 of the maintenance dosage.
  • The maintenance dosage after initial dose escalation is 1.3 g/m2 of body surface area per day divided into 2 doses given every 12 hours (see table below)
    • Patients 1 year to less than 6 years: Increase the dosage in 10% increments to the maintenance dosage, while monitoring WBC cystine concentrations. Allow a minimum of 2 weeks between dosage adjustments. If a patient achieves the therapeutic target WBC cystine concentration at a dosage below the recommended weight-based maintenance dosage, then stop dosage escalation and use the dosage as the patient’s maintenance dosage.
    • Patients 6 years of age and older: Gradually increase the dosage over 4 to 6 weeks until the maintenance dosage is achieved.
  • If a patient experiences initial intolerance, temporarily discontinue PROCYSBI, and then restart at a lower dosage, and gradually increase dosage.
Starting and Maintenance Dosage of PROCYSBI by Body Weight in Cysteamine-Naïve Patients 1 Year of Age and Older (Dosage Rounded Using Available Capsule Strengths)
Weight in kilograms (kg) Starting PROCYSBI Dosage in mg every 12 hours, as a Fraction of the Maintenance Dosage Maintenance PROCYSBI Dosage in mg every 12 hours*
1/6 of dosage 1/4 of dosage
5 or less 25 50 200
6 to 10 50 75 300
11 to 15 75 100 400
16 to 20 100 125 500
21 to 25 100 150 600
26 to 30 125 175 700
31 to 40 125 200 800
41 to 50 150 225 900
51 and greater 175 250 1000

*Higher dosage may be required to achieve target therapeutic WBC cystine concentration.

Patients converting to PROCYSBI from immediate-release (IR) cysteamine1

  • The starting total daily dose of PROCYSBI is equal to the previous total daily dose of IR cysteamine bitartrate.
  • Measure white blood cell (WBC) cystine concentration 2 weeks after initiation of PROCYSBI.
  • Titrate as needed to achieve target WBC cystine concentration. Do not exceed 1.95 grams/m2 per day.

Adverse reactions and dose titration1

  • If adverse reactions occur, decrease the PROCYSBI dose.
  • For patients who have initial intolerance, temporarily discontinue PROCYSBI and then re-start at a lower dose and gradually increase to the target dose.
  • Some patients may be unable to achieve their therapeutic target due to poor tolerability of PROCYSBI.

Monitoring treatment response: White blood cell cystine concentration testing and dose adjustment

  • Regular white blood cell (WBC) cystine testing is an important element of the effective treatment of cystinosis.2
  • PROCYSBI maintenance dose may need to be adjusted to achieve target WBC cystine concentration.1
  • The therapeutic goal is to maintain cystine concentration below 1 nmol ½ cystine/mg protein.1
  • If the WBC cystine concentration is greater than the target, consider the following before dose adjustment:1
    • Adherence to medication and dosing interval
    • The timing between the last dose and the blood draw for the laboratory measurement
    • The timing of PROCYSBI administration in relation to food or other administration instructions
  • If a dose adjustment is required, increase the dose by 10%. Do not exceed 1.95 grams/m2 per day due to an increased risk of adverse reactions.1

WBC monitoring frequency1

Patients one year to less than 6 years naïve to cysteamine
2 weeks after PROCYSBI initiation
Monitor during dose titration
Once target WBC cystine concentration is reached, monitor monthly for 3 months
Then quarterly for 1 year
Then twice yearly (at minimum)
Patients 6 years and older naïve to cysteamine
Monthly for 3 months after reaching maintenance PROCYSBI dose
Then quarterly for 1 year
Then twice yearly (at minimum)
Obtain measurement after reaching the maintenance PROCYSBI dose
Patients converting to PROCYSBI from immediate-release (IR) cysteamine
2 weeks after PROCYSBI initiation
Continue monitoring if further titration is needed
Once target WBC cystine concentration is reached, monitor quarterly for 6 months
Then twice yearly (at minimum)
Monitoring timing
Obtain blood samples for WBC cystine concentration measurement 12 hours after dosing with PROCYSBI. In addition, it is important to accurately record the time of the last dose, the actual dose, and the time the blood sample was taken.12

Twice-daily dosing with 12-hour PROCYSBI1

  • PROCYSBI provides continuous 12-hour cystine control.
  • Patients and caregivers can choose the 12-hour dosing interval that works best for them (e.g., 6 a.m. and 6 p.m. or 9 a.m. and 9 p.m.).

Administration Information1

PROCYSBI should be taken in a consistent manner (i.e., in a fasted state or with a similar type and amount of food) from day to day

Taking PROCYSBI with food or drink

  • Because food may affect the absorption of cysteamine, patients are advised not to eat for 2 hours before and 30 minutes after taking PROCYSBI.
  • If necessary, patients may eat approximately 4 ounces (½ cup) of food in the hour before or after taking PROCYSBI.
    • Foods high in fat should be avoided.
    • Recommended foods (studied in clinical trials) include applesauce, berry jelly, and fruit juice (but not grapefruit juice).
    • PROCYSBI can be administered with electrolyte (except bicarbonate) and mineral replacements necessary for management of Fanconi syndrome, as well as with vitamin D and thyroid hormone.
    • Use in other foods and liquids has not been studied and therefore is not recommended.
    • Do not drink alcohol while taking PROCYSBI.

PROCYSBI should be swallowed whole1

Swallow PROCYSBI capsules whole. Do not crush or chew PROCYSBI or the capsule contents.

  • Take with water or fruit juice (except grapefruit juice).

Opening PROCYSBI capsules1

HAND_CAPSULE_2_RT-thumb

  • To open a PROCYSBI capsule, a patient or caregiver holds each capsule end between thumb and index finger and gently twists the ends in opposite directions.
  • Capsules are not to be pinched in the center or crushed.
  • More than one capsule may need to be opened depending on the dose prescribed.

AS_CAPSULE_BREAK_2_RT-thumb copy

PROCYSBI can be mixed with certain foods or juices1

Do not crush or chew PROCYSBI or the capsule contents.

If your patient has difficulty swallowing, PROCYSBI may be mixed in a clean container with one of the options listed below:

Mix PROCYSBI with applesauce or berry jelly1

  • Open and sprinkle capsule contents onto ½ cup (4 ounces) of applesauce or berry jelly and gently mix.*
  • Swallow the applesauce or berry jelly mixture within 30 minutes. Do not save the mixture for later use.

Mix PROCYSBI with fruit juice (except grapefruit juice)1

  • Open and sprinkle capsule contents into ½ cup (4 ounces) of fruit juice and stir gently until mixed.
  • Drink all the fruit juice mixture within 30 minutes. Do not save the fruit juice mixture for later use.

Taking PROCYSBI with foods or juices not listed above is not recommended.

Give PROCYSBI through a G-tube1

It is best to use a straight (bolus) feeding tube. For people who have a gastrostomy tube (G-tube) that is size 14 French or larger, PROCYSBI may be given as follows:

  • Flush the G-tube button with 5 mL of water to clear the button.
  • Place about ½ cup (4 ounces) of applesauce into a clean container (use only strained applesauce with no chunks); open capsules, and sprinkle contents onto applesauce.
    • Use at least ⅛ cup (1 ounce) of applesauce for children ≤25 kg starting PROCYSBI at a dose of 1 or 2 capsules.
  • Gently mix and place the tip of a catheter-tip syringe at the bottom of the container.
    • Adult: Draw up about 40 mL of the mixture and repeat until full dose is administered. If the entire half a cup (118 mL) of applesauce is used to mix with the dose, then it may take 3 syringes to administer all of the mixture
    • Child: Draw up at least 10 mL of the mixture for doses of 1 or 2 capsules.
  • Place the tip into the feeding tube and fill the feeding tube with the mixture.
  • Hold the feeding tube in a horizontal (straight across) position and give the mixture through the G-tube at a quick and steady rate of 10 mL over 10 seconds.
  • Give all the mixture through the G-tube within 30 minutes of mixing. Do not save the applesauce mixture for later use.
  • Draw up at least 10 mL of fruit juice (except grapefruit juice) or water into another catheter-tip syringe, swirl gently. Flush the G-tube using enough fruit juice or water so that there is no mixture left.1

*Volume of applesauce needed may depend upon patient size and medication amount. Please consult your doctor.

Learn more in the Instructions For Use.


Download the IFU

References

1. PROCYSBI [package insert]. Lake Forest, IL: Horizon Pharma USA, Inc; 2017.

2. Dalton, Neil. The importance of accurate cystine level testing. Cystinosis Research Network. Available at https://cystinosis.org/images/research/article-library/cystagon/2009_01_Cystine_Level_Testing.pdf. Accessed December 4, 2015.

P-PYB-00223

IMPORTANT SAFETY INFORMATION

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.

CONTRAINDICATIONS:

  • 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.
    • 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.

ADVERSE REACTIONS:

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 www.fda.gov/medwatch.

 

DRUG INTERACTIONS:

  • 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.

USE IN SPECIFIC POPULATIONS

Lactation:

  • Breastfeeding is not recommended while taking PROCYSBI.

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