"The U.S. Food and Drug Administration today approved Procysbi (cysteamine bitartrate) for the management of nephropathic cystinosis in children and adults. Procysbi was granted orphan product designation because it is intended to treat a rare dis"...
PROCYSBI (cysteamine bitartrate) is indicated for the management of nephropathic cystinosis in adults and children ages 6 years and older.
DOSAGE AND ADMINISTRATION
PROCYSBI is available as a capsule in 25 mg and 75 mg strengths. Directions for use specific to the route and available methods of administration are presented below. PROCYSBI should be taken at least 2 hours after and at least 30 min before eating.
PROCYSBI therapy should be initiated promptly once the diagnosis is confirmed (i.e., increased white blood cell (WBC) cystine concentration). Cysteamine-na´ve patients should be started on 1/6 to 1/4 of the maintenance dose of PROCYSBI. The dose should be raised gradually over 4 to 6 weeks to help reduce the risk of side-effects. WBC cystine level and/or cysteamine concentration measurements, taken ½ hour after dose administration, are recommended for new patients after the maintenance dose is achieved.
The recommended PROCYSBI maintenance dose is 1.3 gram/m²/day, in two divided doses given every 12 hours. (2.3) The dose can be increased up to1.95 grams/m²/day if the white blood cell cystine level remains higher than the target WBC cystine level and/or the target cysteamine concentration has not been achieved.
TABLE 1: Approximation of 1.3 gram/m²/day
dose of PROCYSBI
|Weight in Pounds||mg of PROCYSBI Every 12 hours|
PROCYSBI (cysteamine bitartrate) delayed-release capsules – Oral Administration
- PROCYSBI should be swallowed whole. Patients should not crush or chew capsules or capsule contents.
- Alternatively, for patients who have difficulty
swallowing capsules, PROCYSBI (cysteamine bitartrate) delayed-release capsules
can be opened and administered as follows:
- Open capsule.
- Sprinkle intact granules on approximately 4 ounces (½ cup) of applesauce or berry jelly.
- Eat mixture within 30 minutes of preparation.
- PROCYSBI may also be emptied into a small volume of
either orange juice or apple juice:
- Mix or Sprinkle intact granules into a small volume of either orange juice or apple juice (approximately 4 ounces (½ cup)).
- Shake gently for 5 minutes then administer by spoon or cup within 30 minutes.
PROCYSBI (cysteamine bitartrate) delayed-release capsules - Feeding Tube Administration
- For patients who have a 12 French or larger gastrostomy
(G)-tube in place, PROCYSBI (cysteamine bitartrate) delayed-release capsules
can be administered as follows:
- Open capsule.
- Mix intact granules into approximately 4 ounces (½ cup) of applesauce.
- Administer mixture via feeding tube within 30 minutes.
- Flush with approximately 8 ounces (1 cup) of orange juice or apple juice to clear the tube.
USE IN OTHER FOODS AND LIQUIDS HAS NOT BEEN STUDIED CLINICALLY AND IS THEREFORE NOT RECOMMENDED.
Administration with Food
Patients should not eat for at least 2 hours before taking PROCYSBI and at least 30 minutes after taking PROCYSBI. Patients who are unable to take PROCYSBI without eating should eat only a small amount (approximately 4 ounces (½ cup)) of food between 1 hour before taking PROCYSBI and 1 hour after taking PROCYSBI. Patients should take PROCYSBI in a consistent manner in regard to food.
Patients should be instructed that if a dose is missed, it should be taken as soon as possible. However, if a patient has missed a dose and the next scheduled dose is due in less than 4 hours, the patient should be instructed to not take the missed dose, and to take the next dose on time. Patients should be instructed not to take 2 doses at one time to make up for a missed dose.
Titration Based on WBC Cystine: Adjust the PROCYSBI dosage to produce the target WBC cystine level. Measured concentrations of cystine in WBCs vary according to analytical techniques for WBC protein levels. Therefore, target concentrations of cystine in WBC should be determined by individual analytical laboratories using local methodology and calibration of protein assay.
Titration Based on Plasma Cysteamine: If WBC cystine measurements are not available, plasma cysteamine measurement may be used to help guide PROCYSBI dose titration. Titrate the PROCYSBI dosage to maintain a cysteamine concentration > 0.1 mg/L, 30 min after dosing.
WBC cystine levels (or plasma cysteamine concentration if adequate WBC cystine testing is not available) should be measured as follows:
- Monthly for 3 months, then quarterly for one year, then twice yearly at a minimum for patients never treated with immediate release cysteamine before.
- Two weeks, then quarterly for 6 months then twice yearly at a minimum for patients switching from immediate-release to PROCYSBI.
Measurement Timing: WBC cystine and/or plasma cysteamine measurements must be obtained 12.5 hours after the evening dose the day before, and therefore 30 minutes after the following morning dose is given.
- In well-controlled and adherent patients with nephropathic cystinosis, the plasma cysteamine is > 0.1 mg/L, and the WBC cystine is < 1.0 nmol ½ cystine/mg protein.
- If the plasma cysteamine is > 0.1 mg/L, but the WBC cystine is > 1.0 nmol ½ cystine/mg protein, the physician is advised to investigate the following parameters: adherence to dosing interval, adherence to medication, or the relationship between administration of PROCYSBI and fasted/fed state.
PROCYSBI dosing may need to be increased to achieve the target WBC cystine concentration (or plasma cysteamine concentration) or decreased if there is evidence of intolerance to PROCYSBI [see WARNINGS AND PRECAUTIONS and ADVERSE REACTIONS]). If a dose adjustment is required, it is recommended to adjust the dose by 10%. The dose of PROCYSBI can be increased to a maximum of 1.95 grams/m²/day to achieve the target WBC cystine concentration (or plasma cysteamine concentration). The dose of 1.95 grams/m²/day of immediate release cysteamine bitartrate has been associated with an increased rate of withdrawal from treatment due to intolerance and an increased incidence of adverse reactions.
Patients who initially tolerate PROCYSBI poorly should temporarily stop taking PROCYSBI, then re-start PROCYSBI at a lower dose and gradually increase the dose to the proper dose. [see WARNINGS AND PRECAUTIONS]. Some patients may be unable to achieve their therapeutic target due to poorer tolerability of PROCYSBI.
Transferring Patients from Immediate-release Cysteamine Bitartrate Capsules
Patients with cystinosis taking immediate-release cysteamine bitartrate may be transferred to a total daily dose of PROCYSBI equal to their previous total daily dose of immediate-release cysteamine bitartrate. Patients being transferred from immediate-release cysteamine bitartrate to PROCYSBI should have their WBC cystine levels and/or plasma cysteamine concentration measured in 2 weeks, and quarterly for 6 months then twice yearly at a minimum .
Dosage Forms And Strengths
- Each 25 mg delayed-release capsule contains 74 mg cysteamine bitartrate, equivalent to 25 mg cysteamine. The capsules are light blue opaque cap imprinted with 'Raptor' logo in white ink and light blue opaque body imprinted with '25 mg' in white ink.
- Each 75 mg delayed-release capsule contains 221 mg cysteamine bitartrate, equivalent to 75 mg cysteamine. The capsules are dark blue opaque cap imprinted with 'Raptor' logo in white ink and light blue opaque body imprinted with '75 mg' in white ink.
Storage And Handling
25 mg Delayed-release Capsule: A hard gelatin capsule with light blue opaque cap imprinted with Raptor Logo in white ink and light blue opaque body imprinted with “25 mg” in white ink, supplied as bottle of 60 capsules (NDC 4966-3001-06).
75 mg Delayed-release Capsule: A hard gelatin capsule with dark blue opaque cap imprinted with Raptor Logo in white ink and light blue opaque body imprinted with “75 mg” in white ink, supplied as bottle of 250 capsules (NDC 4966-3002-25).
Store at 20° to 25°C (68° to 77°F); excursions permitted between 15° and 30°C (59° and 86°F) [See USP Controlled Room Temperature]. Protect from light and moisture.
Dispense only in original packaging. Do not subdivide or repackage. Do not remove desiccant or oxygen absorber from the container. Keep bottles tightly closed in a dry place.
Manufactured for: Raptor Pharmaceuticals Inc. 9 Commercial Blvd, Suite 200 Novato, CA 94949. Update: April 2013 Part No. XXX
Last reviewed on RxList: 5/13/2013
This monograph has been modified to include the generic and brand name in many instances.
Additional Procysbi Information
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