procysbi
Procysbi (Cysteamine Bitartrate) is a cystine depleting agent used to treat Cystinosis.
Cysteamine Bitartrate · by Horizon
Key facts
- Active ingredient
- Cysteamine Bitartrate
- Drug class
- Cystine Depleting Agent
- Form
- Capsule, Oral pellets, Powder, delayed release
- Strength
- Cysteamine Bitartrate EQ 25MG Base · Cysteamine Bitartrate EQ 75MG Base
- Type
- Prescription (Rx)
- Brand or generic
- Brand-name
- May treat
- cystinosis
- Manufacturer
- Horizon
- FDA application
- NDA203389
What is Procysbi?
From the FDA label:PROCYSBI, for oral administration, is a cystine-depleting agent that lowers the cystine content of cells in patients with nephropathic cystinosis, an inherited defect of lysosomal transport. PROCYSBI contains the bitartrate salt of cysteamine. The chemical name for cysteamine bitartrate is ethanethiol, 2-amino, (2 R ,3 R )-2,3-dihydroxybutanedioate (1:1) (salt). Cysteamine bitartrate is a highly water soluble white powder with a molecular weight of 227.24 and the molecular formula C 2 H 7 NS ∙ C 4 H 6 O 6 . It has the following chemical structure: Each PROCYSBI delayed-release capsule contains either 25 mg cysteamine (equivalent to 74 mg cysteamine bitartrate) or 75 mg cysteamine (equivalent to 221 mg cysteamine bitartrate). Each packet of PROCYSBI delayed-release oral granules contains either 75 mg cysteamine (equivalent to 221 mg cysteamine bitartrate) or 300 mg cysteamine (equivalent to 884 mg cysteamine bitartrate). PROCYSBI delayed release granules contain the following inactive ingredients: Eudragit ® L 30 D-55 (methacrylic acid-ethyl acrylate copolymer), hypromellose, microcrystalline cellulose, purified water, sodium lauryl sulfate, talc, and triethyl citrate. Additionally the capsule shell contains the following inactive ingredients: gelatin, ink (blue and white), and titanium dioxide. Chemical Structure
How to use
Procysbi is sold in more than one form (Capsule, Oral pellets and Powder, delayed release), and each is dosed differently. The instructions below come from the FDA label for application NDA203389 — follow the label that came with the product you were actually prescribed.
Recommended Dosage in Cysteamine-Naïve Patients See full prescribing information for weight-based dosing tables for the starting and maintenance dosage. ( 2.2 ) For initial intolerance, temporarily discontinue and then re-start PROCYSBI at a lower dosage and gradually increase to the maintenance dosage. ( 2.2 ) Switching from Immediate-release Cysteamine to PROCYSBI Start with a total daily dose of PROCYSBI equal to the previous total daily dose of immediate-release cysteamine bitartrate. ( 2.3 ) Dose Titration Adjust dose to achieve a therapeutic target white blood cell (WBC) cystine concentration. ( 2.4 , 2.5 ) If a dose adjustment is required, increase the dosage by 10%. The maximum dosage is 1.95 grams/m 2 per day. ( 2.4 ) If adverse reactions occur, decrease the dosage. Some patients may be unable to achieve their therapeutic target. ( 2.4 ) Preparation and Administration ( 2.6 ) Capsules : Swallow whole; do not crush or chew capsules or capsule contents. Take the capsules with fruit juice (except grapefruit juice) or water. Oral Granules: Do not crush or chew the granules. Sprinkle and mix the granules in applesauce, berry jelly or fruit juice (except grapefruit juice). For patients who cannot swallow the capsules or with gastrostomy tubes, see the full prescribing information on how to prepare and administer the capsules and oral granules. Administer PROCYSBI at least 1…
Side effects
The following adverse reactions are also discussed in other sections of the labeling: Ehlers-Danlos-like Syndrome [see Warnings and Precautions (5.1) ] Skin Rash [see Warnings and Precautions (5.2) ] Gastrointestinal (GI) Ulcers and Bleeding [see Warnings and Precautions (5.3) ] Fibrosing Colonopathy [see Warnings and Precautions (5.4) ] Central Nervous System Symptoms [see Warnings and Precautions (5.5) ] Leukopenia and/or Elevated Phosphatase Levels [see Warnings and Precautions (5.6) ] Benign Intracranial Hypertension [see Warnings and Precautions (5.7) ] Most common adverse reactions in: Patients 6 years of age and older previously treated with cysteamine (≥5%) are: vomiting, nausea, abdominal pain, breath odor, diarrhea, skin odor, fatigue, rash, and headache. ( 6.1 ) Patients 1 year to less than 6 years naïve to cysteamine treatment (>10%) are: vomiting, gastroenteritis/viral gastroenteritis, diarrhea, breath odor, nausea, electrolyte imbalance and headache. ( 6.1 ) To report SUSPECTED ADVERSE REACTIONS, contact Amgen Inc. at 1 800 77 AMGEN (1 800 772 6436) or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch . 6.1 Clinical Trials Experience Because clinical trials are conducted under widely varying conditions, adverse reaction rates observed in the clinical trials of a drug cannot be directly compared with rates in the clinical trials of another drug and may not reflect the…
Warnings
Important safety information
Ehlers-Danlos-like Syndrome: Reduce dosage if skin and bone lesions occur. ( 5.1 ) Skin Rash: Discontinue if severe skin rash such as erythema multiforme bullosa or toxic epidermal necrolysis occurs. ( 5.2 ) Gastrointestinal (GI) Ulcers and Bleeding: Monitor for GI symptoms and consider decreasing the dose if severe symptoms occur. ( 5.3 ) Fibrosing Colonopathy: Evaluate patients with severe, persistent, and/or worsening abdominal symptoms for fibrosing colonopathy. If the diagnosis is confirmed, permanently discontinue PROCYSBI and switch to immediate-release cysteamine bitartrate capsules ( 5.4 ) Central Nervous System (CNS) Symptoms: Monitor for CNS symptoms; interrupt or reduce the dose for severe symptoms or those that persist or progress ( 5.5 ). Leukopenia and/or Elevated Alkaline Phosphatase Levels: Monitor white blood cell count and alkaline phosphatase levels; decrease or discontinue the dose until values revert to normal. ( 5.6 ) Benign Intracranial Hypertension: Monitor for signs and symptoms; interrupt or reduce the dose for signs/symptoms that persist, or discontinue if diagnosis is confirmed. ( 5.7 ) 5.1 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 [see Dosage and Administration (2.1 , 2.4) ] . 5.2 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 [see Contraindications (4) ] . 5.3 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 [see Dosage and Administration (2.1 , 2.4) ] . 5.4 Fibrosing Colonopathy Fibrosing colonopathy, including colonic stricture formation, has been reported with postmarketing use of PROCYSBI in pediatric and young adult patients with nephropathic cystinosis. Some of these patients had been treated with PROCYSBI for prolonged periods of time. Reported symptoms include: abdominal pain, vomiting, bloody or persistent diarrhea, and fecal incontinence. Evaluate patients with severe, persistent, and/or worsening abdominal symptoms for fibrosing colonopathy. If the diagnosis is confirmed, permanently discontinue PROCYSBI and switch to immediate-release cysteamine bitartrate capsules. An association between methacrylic acid-ethyl acrylate copolymer (an inactive ingredient in PROCYSBI) and fibrosing colonopathy cannot be ruled out. 5.5 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. 5.6 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. 5.7 Benign Intracranial Hypertension Benign intracranial hypertension (pseudotumor cerebri; PTC) and/or papilledema have 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.
Who should not take Procysbi
The use of PROCYSBI is contraindicated in patients with a serious hypersensitivity reaction, including anaphylaxis, to penicillamine or cysteamine. Hypersensitivity to penicillamine or cysteamine ( 4 )
Overdose — what happens if you take too much
One case of overdosing with PROCYSBI has been reported. A 16-year-old male patient suffered nausea and vomiting after he mistakenly took a second dose of PROCYSBI 30 minutes after his usual dose. Two cases of overdosing with immediate-release cysteamine bitartrate have been reported in two patients. In the first case, the patient immediately vomited after ingesting an unknown dose and did not develop any symptoms. The second case involved an accidental ingestion of a 200 to 250 mg/kg dose by a healthy 13-month-old child. Vomiting and dehydration were experienced. The child was hospitalized and fluids were administered. The patient fully recovered from the overdosing. Should overdosing occur, the respiratory and cardiovascular systems should be supported appropriately. No specific antidote is known. Hemodialysis may be considered since cysteamine is poorly bound to plasma proteins.
U.S. Poison Control: call or text 1-800-222-1222 (free, 24/7). In an emergency call 911. From the FDA label; not medical advice.
Interactions
7.1 Drugs that Increase Gastric pH Drugs that increase the gastric pH (e.g., medications containing bicarbonate or carbonate) may alter the pharmacokinetics of cysteamine due to the premature release of cysteamine from PROCYSBI and increase WBC cystine concentration. Concomitant administration of 20 mg omeprazole did not affect the pharmacokinetics of cysteamine when PROCYSBI was administered with 240 mL of orange juice or with 240 mL of water [see Clinical Pharmacology (12.3) ]. Monitor WBC cystine concentration when drugs that increase the gastric pH are concomitantly used [see Dosage and Administration (2.5) ]. 7.2 Use with Alcohol 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. Therefore, do not consume alcoholic beverages during treatment with PROCYSBI [see Dosage and Administration (2.6) ]. 7.3 Other Medications Used for the Management of Fanconi Syndrome PROCYSBI can be administered with other electrolyte and mineral replacements necessary for management of Fanconi syndrome, as well as vitamin D and thyroid hormone.
Drug class
May treat (source: NIH RxClass)
See how Procysbi ranks — best-rated cystine depleting agent for:
Dosage forms
Capsule, Oral pellets and Powder, delayed release
The forms currently marketed in the U.S., per the FDA National Drug Code Directory.
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Frequently asked questions
- What does Procysbi treat?
- Procysbi (Cysteamine Bitartrate) may be used to treat cystinosis, based on NIH RxClass drug-classification data (conditions a drug may treat — not a verbatim copy of the FDA-approved label). This is general reference, not medical advice.
- Is there a coupon or discount for Procysbi?
- pharmaranks is an independent ratings site, not a pharmacy — we don't issue coupons or sell Procysbi. To pay less, ask your prescriber or pharmacist whether a generic equivalent exists, check the manufacturer's copay/savings card and patient-assistance program, and compare a pharmacy discount card against your insurance copay. Prices vary by pharmacy, insurance, and location, so always confirm at the counter. This is general reference, not medical or financial advice.
- Who makes Procysbi?
- Procysbi is marketed by Horizon. You can see Horizon's full profile, rating, and other products on pharmaranks.
- Is Procysbi a brand-name or generic drug?
- Procysbi is a brand-name product with the active ingredient Cysteamine Bitartrate.
- Is Procysbi available over the counter?
- No. Procysbi is a prescription (Rx) medication in the US — you need a prescription from a licensed clinician (in person or via telehealth); it isn't sold over the counter. For some conditions there are OTC alternatives — ask your pharmacist.
- What forms does Procysbi come in?
- Procysbi is currently marketed as capsule, oral pellets and powder, delayed release, per the FDA's National Drug Code Directory.
- What class of drug is Procysbi?
- Procysbi is classified as cystine depleting agent, per the FDA's Established Pharmacologic Class.
- Is Procysbi FDA-registered?
- Procysbi is on record with the U.S. FDA under application number NDA203389. You can verify this on its official FDA label.
- Is Procysbi safe?
- There's no single safe-or-not verdict, and we don't yet have a composite recall-safety score for Procysbi. Review its FDA-label side effects and warnings below, and always consult a licensed professional.
- What are the side effects of Procysbi?
- Procysbi's side effects are taken directly from its FDA label. From the label: The following adverse reactions are also discussed in other sections of the labeling: Ehlers-Danlos-like Syndrome [see Warnings and Precautions (5.1) ] Skin Rash [see Warnings and Precautions (5.2) ] Gastrointestinal (GI) Ulcers and Bleeding [see Warnings and Precautions (5.3) ] Fibrosing Colonopath… Both common and serious reactions are documented in full on this page. This is general reference from the FDA, not medical advice — always consult a professional.
Clinical content sourced from the FDA label via openFDA (U.S. FDA). View the FDA label on DailyMed → Provided for general reference only — not medical advice. Always consult a licensed professional and the current prescribing information.
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