Home › NDC Lookup › Ingredients › Cysteamine Bitartrate › 75987-0140-14
PROCYSBI Cysteamine bitartrate 75 mg Granule, Delayed Release — NDC 75987-0140-14 package photo

PROCYSBI Cysteamine bitartrate 75 mg Granule, Delayed Release

by Horizon Therapeutics USA, Inc. · 120 PACKET in 1 CARTON (75987-140-14) / 1 GRANULE, DELAYED RELEASE in 1 PACKET
NDC 75987-0140-14
🏷️ FDA NDC (as labeled) 75987-140-14 billing pads the product segment with a zero
This package
Contains1 granule, delayed release in 1 packet Pack sizes2 compare ↓
Also priced by: Part D plans $118.14/unit — full pricing hub ↓
Also comes in: 1 granule 75987-0140-13
Rx only Brand On market Non-controlled
🗂️ Data synced Jul 24, 2026 · sources: openFDA · FDA label (DailyMed) · FDA Orange & Purple Book · First Databank · CMS NADAC, ASP, Medicare & Medicaid · RxNorm
📋 All sources & update times →

This page is curated, coded, and maintained by

Compiled from public FDA, CMS, DailyMed, NADAC, and related drug data sources. Editorial policy

🆔 Identity & classification

FDA NDC (as labeled) 75987-140-14
Product NDC 75987-140
11-digit billing NDC 75987014014
NCPDP billing unit EA — each (per item)
RxCUI 1421467, 1421473, 1421475, 1421477, 2284510, 2284514, 2284515, 2284516
UNII QO84GZ3TST
Application # NDA213491
SPL Set ID d3a3ec28-f746-463a-bb92-3bc8826db09e
Established class (EPC) Cystine Depleting Agent
Mechanism of action Cystine Disulfide Reduction
DEA schedule Non-controlled
Marketing category NDA
Marketing status On market
FDA listing status Listed (active directory)
Marketing start 2020-02-14
Route ORAL
Dosage form GRANULE, DELAYED RELEASE
Substance CYSTEAMINE BITARTRATE
GPI-14 56400030103020
GCN Seq No 080755
GCN 47723
HICL code 009274
Ingredient (HICL) Cysteamine Bitartrate
HIC1 code R
Therapeutic class — broad (HIC1) Kidney/Urinary Tract
HIC2 code R1
Therapeutic class — intermediate (HIC2) Affect Primarily Kidneys/Urinary Tract
HIC3 code R1W
Therapeutic class — specific (HIC3) Cystine-Depleting Agents, Nephropathic Cystinosis
AHFS code 92:92.00.00
AHFS class Other Miscellaneous Therapeutic Agents
FDB label name PROCYSBI DR 75 MG GRANULE PKT
FDB brand name Procysbi
Legend status F — Federal legend — prescription drug or device
Why two NDCs? The FDA registers this code as 75987-140-14 — a 5-3-2 layout, and that's what's printed on the package and shown on DailyMed. For insurance claims, every NDC is standardized to a uniform 11-digit 5-4-2 format by adding a zero to the product segment → 75987-0140-14. Same drug, same package — only the format differs.
Where does this data come from?
Identifiers from the FDA openFDA NDC Directory and Structured Product Labeling; RxCUI from RxNorm (NLM); GPI from Medi-Span; GCN / HIC / AHFS / legend from First Databank.

🏷️ RxNorm drug class

This medicine belongs to the Cystine Depleting Agent class.

Pharmacologic class Cystine Depleting Agent
Drug family (ATC) Amino acids and derivatives, Other ophthalmologicals
How it works Cystine Disulfide Reduction
Where does this data come from?
Therapeutic classes from RxNorm RxClass (U.S. National Library of Medicine) — Established Pharmacologic Class (FDA), ATC drug family (WHO) and mechanism of action, matched by this product’s RxCUI.

🏭 Manufacturer & labeler

LabelerHorizon Therapeutics USA, Inc.
Application holderHORIZON THERAPEUTICS USA INC
FDA applicationNDA213491 (NDA)
Labeler code75987
First marketedFeb 2020
Product typeHuman Prescription Drug
Portfolio12 products on file
The labeler markets the product; the application holder owns the FDA approval. They’re often the same company but can differ (e.g. a repackager or an authorized generic). A mailing address / phone appears here when the manufacturer includes it in the product’s FDA label (not all do).
Where does this data come from?
Labeler, application holder and registered establishment from the FDA openFDA NDC Directory and Drugs@FDA; address/contact from the product’s FDA label.

🩺 Clinical

Label name PROCYSBI DR 75 MG GRANULE PKT Ingredient Cysteamine Bitartrate
📗 Our plain-language guide HelloPharmacist
  • Cystinosis is a rare inherited condition where your body can't properly move a substance called cystine out of the recycling compartments inside your cells. Over time, cystine buil...
  • What exactly is cystinosis, and why do I need this medication for life?
  • Your doctor will measure cystine levels in your white blood cells regularly — usually about 5 to 6 hours after a dose. These levels tell your doctor whether the dose is in the righ...
  • How do I know if the medication is working?
📖 Read our full Cysteamine guide →
Where does this data come from?
Plain-language summary from MedlinePlus (U.S. National Library of Medicine); supplement & herbal interactions and nutrient depletion data from the Natural Medicines database; our full guide is HelloPharmacist editorial content.

💊 What it looks like

Color Blue / blue / white
ShapeCapsule
ImprintPRO;75;mg
Size22 mm
ScoringNot scored
One label can cover several strengths, so colors may be combined — always confirm a loose pill against the dispensed prescription label or a pharmacist.
Where does this data come from?
Physical description (imprint, shape, color, scoring, coating) from this product’s FDA Structured Product Labeling (SPL), mirrored from DailyMed / openFDA.

🧪 Inactive Ingredients / Excipients

Inactive ingredients, also called excipients, are components of the drug product other than the active ingredient. They may include fillers, dyes, coatings, preservatives, flavors, or other formulation ingredients.

A current SPL was checked, but it does not contain a structured or narrative inactive-ingredient list for this product. This does not mean the product has no inactive ingredients.
Where does this data come from?
Source: official FDA Structured Product Labeling (SPL) via DailyMed and the openFDA label index. Structured IACT rows and label-wide narrative are kept separate; availability and product-level specificity depend on the submitted label.

Inactive ingredient FAQ

Are inactive ingredients the same for every manufacturer?
No. Inactive ingredients can differ by manufacturer, dosage form, strength, and package / product version.
Why might an inactive ingredient be missing?
Some SPLs do not provide a complete structured inactive-ingredient list, and older or unusual labels may only include the information in narrative text.
Can inactive ingredients matter?
Yes. They can matter for allergies, intolerances, dyes, gluten / lactose concerns, preservatives, and formulation differences — but confirm with a pharmacist or the manufacturer when it’s clinically important.

💲 Pricing

A drug doesn't have one price. Each row is a different public payment system, and none is what you'd pay at the counter — that depends on your insurance. The ⓘ on each row explains what it measures.

Price systemPer gPer package
Retail pharmacies payNADAC · weekly Not in the retail survey — common for institutional, discontinued, or low-volume packs.
Medicaid paysCMS SDUD · 12 mo No recent Medicaid claims on file for this NDC — rare and low-volume NDCs are suppressed in the public data.
Medicare drug plans payPart D · Q2 2026 $118.14 —
Where does this data come from?
NADAC (National Average Drug Acquisition Cost) is the CMS weekly pharmacy-acquisition-cost survey — what pharmacies pay. ASP (Average Sales Price) is the CMS Medicare Part B drug-payment file, published quarterly. Medicaid pays is computed by us from CMS State Drug Utilization Data (total reimbursed ÷ units, trailing 12 months) — gross of rebates and inclusive of dispensing fees, so it reflects what Medicaid paid, not an acquisition cost. Medicare drug plans pay is the median negotiated point-of-sale unit cost across plans listing this NDC in the CMS quarterly Prescription Drug Plan pricing files, before rebates. The VA pays is the federal contract price (FSS, and the statutory Big 4 ceiling where listed) from the VA National Acquisition Center pharmaceutical price file. All are free public government data; each measures a different payer, so the figures are not directly comparable.

🔁 Therapeutic equivalents

ProductLabelerPackNADAC/unitTEStatusPrice vs. this
Procysbi 75 mgthis 75987-0140-14 Horizon 1 granule — — FDA listed —
About this product: this is the brand-name version. We did not find an FDA-approved generic match for this exact strength, form and route.
Where does this data come from?
Equivalents are other NDCs of the same ingredient, form and route from the openFDA NDC Directory, ranked least-expensive-first by NADAC. Therapeutic-equivalence (AB) ratings come from the FDA Orange Book; biologics use the FDA Purple Book for biosimilar & interchangeable status.

⏳ Availability & generic status

🏛️
2020
First FDA approval
Feb 2020
📍
2026
Currently FDA-listed
6 years listed
🛡️
2037
Latest patent/protection listed
not a guaranteed launch date
🔒No FDA-approved generic found

We did not find an FDA-approved generic match for this exact strength, form and route. Patent/protection dates below may affect future generic timing.

🛡️ Latest patent/protection date listed: FDA patent/protection data lists protections through Feb 2037. This may affect when a full generic version becomes widely available, but it is not a guaranteed launch date.
📅 FDA approved Feb 14, 2020 RLD RS ⏳ ~10.4 yr to latest listed protection

Why the date isn’t exact: Generic timing can change because patents may be challenged, settled, licensed, added, removed, or worked around with a narrower label — and FDA approval does not always mean a pharmacy can get the generic today.

Patents & exclusivity — FDA Orange Book
US 8026284 — method of use (U-1399)
US 9198882 — method of use (U-1399)
US 9192590 — method of use (U-1399)
US 9192590 — method of use (U-1399)
US 8026284 — method of use (U-1399)
US 9198882 — method of use (U-1399)
US 9925156 — method of use (U-1399)
US 10143665 — method of use (U-1399)
US 10328037 — method of use (U-1399)
US 9925157 — method of use (U-1399)
US 9925158 — method of use (U-1399)
US 10548859 — method of use (U-1399)
US 9925157 — method of use (U-1399)
US 9925158 — method of use (U-1399)
US 10548859 — method of use (U-1399)
US 9925156 — method of use (U-1399)
US 10143665 — method of use (U-1399)
US 10328037 — method of use (U-1399)
US 10905662 — method of use (U-1399)
US 10905662 — method of use (U-1399)
US 9173851 — drug product
US 9233077 — drug product
US 9173851 — drug product
US 9233077 — drug product
US 10905662*PED — drug product
US 10905662*PED — drug product
US 8026284*PED — drug product
US 9198882*PED — drug product
US 9192590*PED — drug product
US 9173851*PED — drug product
US 9192590*PED — drug product
US 9173851*PED — drug product
US 8026284*PED — drug product
US 9198882*PED — drug product
US 9925156*PED — drug product
US 10143665*PED — drug product
US 10328037*PED — drug product
US 9233077*PED — drug product
US 9925157*PED — drug product
US 9925158*PED — drug product
US 10548859*PED — drug product
US 9925157*PED — drug product
US 9925158*PED — drug product
US 10548859*PED — drug product
US 9925156*PED — drug product
US 10143665*PED — drug product
US 10328037*PED — drug product
US 9233077*PED — drug product
2020 2022 2024 2026 2028 2030 2032 2034 2036
Today
LOE
Substance patent Formulation patent Method-of-use patent Exclusivity Pediatric +6mo
🏛️FDA exclusivity
FDA-granted marketing protection. It’s separate from patents and may be shorter than patent protection.
🧪Product / substance patents
Patents covering the active ingredient, product, formulation, or related drug features.
🎯Method-of-use patents
Patents covering specific approved uses. These can sometimes be carved out with a “skinny label,” but not always.
🛈 What do these terms mean?
Patent
Legal protection listed in the Orange Book that may delay generic approval or launch. Issued by the U.S. Patent & Trademark Office.
Substance patent
Covers the active drug molecule itself — the hardest to design around. A generic generally can’t launch until it expires.
Formulation (product) patent
Covers a specific formulation or dosage form. A generic can sometimes work around it with a different formulation.
Method-of-use patent
A patent covering one specific approved use of the drug — not necessarily the whole molecule. A generic can sometimes launch with a “skinny label” that carves out the protected use and keeps the others.
Skinny label
A generic label that omits a still-patented use when the FDA allows it — letting a generic reach the market for the unprotected uses.
Exclusivity
FDA-granted marketing protection, separate from patents — e.g. 5-yr new chemical entity, 7-yr orphan drug, or a +6-month pediatric extension.
Paragraph IV
A generic applicant’s formal challenge to a listed patent. It can potentially lead to earlier generic entry, but often involves litigation or a settlement.
RLD / RS
Reference Listed Drug — the brand product the FDA uses as the reference for generic applications. Reference Standard — the product the FDA expects generics to compare against in bioequivalence testing.
TE / AB rating
FDA therapeutic-equivalence rating. An AB rating generally means the FDA considers a generic therapeutically equivalent to — and substitutable for — the brand.
LOE (loss of exclusivity)
The latest patent or exclusivity currently listed — the loss-of-exclusivity / latest-listed-protection date shown on this page. Paragraph-IV challenges and settlements can move the real date earlier; FDA approval and a manufacturer’s decision to market can move it later.

Built from the FDA Orange Book. The bars above are scaled to each protection’s expiry; the red LOE marker is the last one to lapse.

Listed patents (48)
PatentTypeUse codeExpires
US 8026284 ↗ Method of use U-1399 Sep 22, 2027
US 9198882 ↗ Method of use U-1399 Jan 26, 2027
US 9192590 ↗ Method of use U-1399 Jan 26, 2027
US 9192590 ↗ Method of use U-1399 Jan 26, 2027
US 8026284 ↗ Method of use U-1399 Sep 22, 2027
US 9198882 ↗ Method of use U-1399 Jan 26, 2027
US 9925156 ↗ Method of use U-1399 Jan 26, 2027
US 10143665 ↗ Method of use U-1399 Aug 16, 2036
US 10328037 ↗ Method of use U-1399 Aug 16, 2036
US 9925157 ↗ Method of use U-1399 Jan 26, 2027
US 9925158 ↗ Method of use U-1399 Jan 26, 2027
US 10548859 ↗ Method of use U-1399 Aug 16, 2036
US 9925157 ↗ Method of use U-1399 Jan 26, 2027
US 9925158 ↗ Method of use U-1399 Jan 26, 2027
US 10548859 ↗ Method of use U-1399 Aug 16, 2036
US 9925156 ↗ Method of use U-1399 Jan 26, 2027
US 10143665 ↗ Method of use U-1399 Aug 16, 2036
US 10328037 ↗ Method of use U-1399 Aug 16, 2036
US 10905662 ↗ Method of use U-1399 Aug 16, 2036
US 10905662 ↗ Method of use U-1399 Aug 16, 2036
US 9173851 ↗ Drug product — Jun 17, 2034
US 9233077 ↗ Drug product — Jun 17, 2034
US 9173851 ↗ Drug product — Jun 17, 2034
US 9233077 ↗ Drug product — Jun 17, 2034
US 10905662*PED ↗ Drug product — Feb 16, 2037
US 10905662*PED ↗ Drug product — Feb 16, 2037
US 8026284*PED ↗ Drug product — Mar 22, 2028
US 9198882*PED ↗ Drug product — Jul 26, 2027
US 9192590*PED ↗ Drug product — Jul 26, 2027
US 9173851*PED ↗ Drug product — Dec 17, 2034
US 9192590*PED ↗ Drug product — Jul 26, 2027
US 9173851*PED ↗ Drug product — Dec 17, 2034
US 8026284*PED ↗ Drug product — Mar 22, 2028
US 9198882*PED ↗ Drug product — Jul 26, 2027
US 9925156*PED ↗ Drug product — Jul 26, 2027
US 10143665*PED ↗ Drug product — Feb 16, 2037
US 10328037*PED ↗ Drug product — Feb 16, 2037
US 9233077*PED ↗ Drug product — Dec 17, 2034
US 9925157*PED ↗ Drug product — Jul 26, 2027
US 9925158*PED ↗ Drug product — Jul 26, 2027
US 10548859*PED ↗ Drug product — Feb 16, 2037
US 9925157*PED ↗ Drug product — Jul 26, 2027
US 9925158*PED ↗ Drug product — Jul 26, 2027
US 10548859*PED ↗ Drug product — Feb 16, 2037
US 9925156*PED ↗ Drug product — Jul 26, 2027
US 10143665*PED ↗ Drug product — Feb 16, 2037
US 10328037*PED ↗ Drug product — Feb 16, 2037
US 9233077*PED ↗ Drug product — Dec 17, 2034
Common questions
Is there a generic version of PROCYSBI DR 75 MG GRANULE PKT?
No FDA-approved generic equivalent is currently listed in the FDA Orange Book for PROCYSBI DR 75 MG GRANULE PKT. Based on the patents and exclusivity currently listed, the Orange Book estimate is that full-label generic entry may be delayed until Feb 2037 — an estimate, not a guaranteed launch date.
The FDA approved a generic — why can’t I get it at my pharmacy yet?
FDA approval and pharmacy availability are two different things. The FDA can approve a generic years before it actually reaches pharmacies, because the brand company may still hold patents or have a settlement that delays the launch. A manufacturer also has to choose to make and sell it, and have supply ready. So a drug can be “FDA-approved generic exists” and still be brand-only at the counter today.
Why do different websites show different generic release dates?
Generic availability is not based on one single date. Some sources use the first exclusivity expiration, some use the last product patent, and others use the latest method-of-use patent. Patent challenges, settlements, licenses, and label carve-outs can also change the real-world launch date. This page shows the underlying Orange Book dates so you can see why estimates may differ.
What does “FDA listed” mean?
It means the product appears in the FDA’s official NDC directory. That’s a good sign a product exists and is intended for the U.S. market, but on its own it does not confirm a pharmacy can fill it today. Where we have recent retail pricing data (NADAC) for a product, we label it “Availability likely” instead.
What does a patent or protection date mean here?
It’s the latest date currently listed in the FDA Orange Book for a patent or exclusivity on the brand product. It can affect when a full generic version becomes widely available — but it is not a guaranteed generic launch date. Generics sometimes arrive earlier (through a settlement or patent challenge) or later (a manufacturer still has to make and sell one).
What does “current Orange Book estimate” mean?
It means we are using the latest patent and exclusivity dates currently listed in the FDA Orange Book. It is not a guaranteed launch date.
Can a generic come out before the last patent expires?
Sometimes. A generic company may challenge a patent, settle with the brand manufacturer, receive a license, or obtain approval with a narrower label that avoids a patented use. In other cases, the last listed protection may delay full-label generic competition.
Can a generic come out after the listed dates?
Yes. Even after patents or exclusivity expire, a generic still needs FDA approval and a manufacturer must choose to market it. Supply, litigation, business decisions, or regulatory issues can delay actual availability.
What is the difference between patents and exclusivity?
Patents are legal protections usually issued by the U.S. Patent and Trademark Office. FDA exclusivity is marketing protection granted by the FDA. They are separate, and either one can affect generic timing.
Why are there multiple patent dates?
One drug can have several patents covering different things: the active ingredient, a formulation, a manufacturing process, or a specific approved use. That is why a page may show several expiration dates instead of one simple generic date.
Built from FDA Orange Book patent and exclusivity data. Dates are refreshed from public FDA data when available; the marker is max(latest patent expiry, latest exclusivity expiry). Paragraph-IV settlements and first-filer 180-day exclusivity can shift the real date; a method-of-use patent may allow an earlier skinny-label generic for non-protected indications. Generic launch timing is an estimate, not a guarantee.
Where does this data come from?
Patents and exclusivity from the FDA Orange Book (small-molecule drugs), refreshed from public FDA data. Generic launch timing is an estimate, not a guarantee.

📊 Medicare Part D spend CMS · PART D · 2026 (Q1)

Medicare Part D (outpatient prescription) spending for Procysbi — the program that covers self-administered drugs. 1 manufacturer.
⚠️ Drug-level data: CMS publishes Part D spending by drug, not by NDC — these figures combine every manufacturer, strength and package size sold under the name Procysbi. That’s a different level of aggregation than the Medicaid card above, which is specific to this exact 11-digit NDC (pack size included), so the two aren’t directly comparable.
Period
Total Part D spend
$8.98M
Claims incl. refills
120
Beneficiaries
43
Spend / beneficiary
$208,844.15
Spend / claim
$74,835.82
Trend by period
💵 About the dollar figures: spending is what Part D plans paid before confidential manufacturer rebates, so the program’s real net cost is lower. A blank patient count means fewer than 11 people — CMS hides counts that small to protect privacy. Source: CMS Medicare Quarterly Part D Spending by Drug (data.cms.gov), updated quarterly.

📦 Packaging — all sizes for this product

Package NDCDescription Marketing startStatus
75987-0140-13 60 PACKET in 1 CARTON (75987-140-13) / 1 GRANULE, DELAYED RELEASE in 1 PACKET 2020-02-14 Active
75987-0140-14 You're viewing this 120 PACKET in 1 CARTON (75987-140-14) / 1 GRANULE, DELAYED RELEASE in 1 PACKET 2020-02-14 Active

Pack size FAQ

What quantity is in NDC 75987-0140-14?
NDC 75987-0140-14 is listed by the FDA — 120 packet in 1 carton / 1 granule, delayed release in 1 packet.
What NDC number is used to bill for this package of PROCYSBI Cysteamine bitartrate 75 mg Granule, Delayed Release?
Bill NDC 75987-0140-14 — the 11-digit billing format is 75987014014. Pharmacy and medical claims use the 11-digit form; the FDA label may print a shorter form of the same code.

🧭 About this NDC listing & data coverage

Finished prescription product
What data is (and isn’t) available for this NDC — tap to expand
NDC identity (package / product / labeler codes) ✓ Available
Labeler ✓ Available
Product & package description ✓ Available
Marketing category & status ✓ Available
Active ingredient / dosage form / route ✓ Available
FDA label (SPL via DailyMed) ✓ Available
Package photos ✓ Available
Inactive ingredients (structured) — Not published for this NDC The labeler did not submit a structured excipient list, or no SPL is available.
NADAC pharmacy acquisition price (CMS) — Not published for this NDC CMS publishes NADAC only for NDCs reported in its retail-pharmacy survey.
Orange Book / therapeutic-equivalence data ✓ Available
HCPCS J-code billing crosswalk — Not published for this NDC Most self-administered / retail products have no J-code — that is normal.
Medicaid utilization (CMS SDUD) — Not published for this NDC CMS reports utilization only for NDCs with Medicaid claims above its privacy threshold.
“Not published” reflects what the public FDA / CMS / NLM sources provide for this exact package code — it is a property of the data feeds, not a judgment about the product.

Questions about this listing

Why is there no price listed?
The pricing shown on our NDC pages comes from CMS NADAC, a voluntary survey of retail community pharmacy invoices. CMS does not publish a NADAC for every NDC — packages outside the retail survey (institutional and hospital products, bulk packages, discontinued items, and many OTC items) may never receive one. A missing price reflects the survey's scope, not this product's actual cost, and does not mean the product is free or unavailable.
Is the NDC printed on the package the same as the 11-digit billing NDC?
Yes, they identify this exact package in different formats. The FDA registers it as 75987-140-14, which is what is printed on the packaging and shown on DailyMed. Insurance claims use a fixed 11-digit 5-4-2 format, so the short segment is padded with a leading zero: 75987-0140-14, written without dashes as 75987014014. The Identity section at the top of this page lists every form of this code.
What do the three segments of this NDC mean?
In 75987-0140-14, the first segment (75987) is the labeler code FDA assigned to Horizon Therapeutics USA, Inc.; the middle segment (0140) identifies this specific product — its ingredient, strength, and dosage form; and the last segment (14) identifies this exact package size and type. Together they name one specific package of one specific product.
Is this package still being marketed?
Yes, per the latest FDA NDC Directory data on this page: this package is listed as actively marketed, with no marketing end date reported by Horizon Therapeutics USA, Inc.. Listing status can change — the directory data on this page refreshes weekly.
Does this product come in other package sizes?
Yes — the FDA directory lists 1 other package presentation of this same product, including 1 granule (75987-0140-13). Each has its own NDC and its own page — see the package list near the top of this page.
Who lists this product with the FDA?
Horizon Therapeutics USA, Inc. is the labeler of record for this NDC — the company under whose FDA-assigned code the package is listed. The labeler may be the manufacturer itself or a distributor marketing the product under its own code.
Do I need a prescription for this product?
This NDC is listed with FDA as a prescription product, so it is dispensed under a prescriber's order. Your pharmacist can tell you whether any over-the-counter forms of the same medication exist.
This page identifies an FDA-listed package (the NDC) and reports public regulatory and pricing data about the listing. It is reference information, not a medical recommendation — talk to your pharmacist or prescriber about your own medication.
Where does this data come from?
Listing facts (marketing category, packager status, marketing dates) from the FDA openFDA NDC Directory; label availability from DailyMed; pricing coverage from CMS NADAC; equivalence scope from the FDA Orange Book.

📄 Full prescribing information FDA SPL

The complete FDA label for this product — the official prescribing information, verbatim, section by section. Jump with a chip, search within the label, or expand everything.
🎯 Indications and Usage ⏱️ Dosage and Administration 💊 Dosage Forms and Strengths ⛔ Contraindications ⚠️ Warnings and Cautions 🤒 Adverse Reactions 🔄 Drug Interactions 👥 Use in Specific Populations 🤰 Pregnancy 🧒 Pediatric Use 🧓 Geriatric Use 🆘 Overdosage 🧬 Clinical Pharmacology 🧬 Mechanism of Action 📦 How Supplied / Storage and Handling 📦 Storage and Handling 📋 Description 💬 Information for Patients
🎯 Indications and Usage 50 words ▾

1 INDICATIONS AND USAGE PROCYSBI is indicated for the treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older. PROCYSBI is a cystine-depleting agent indicated for the treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older. ( 1 )

⏱️ Dosage and Administration ~3 min read ▾

2 DOSAGE AND ADMINISTRATION 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 hour before or 1 hour after medications containing bicarbonate or carbonate. Do not eat for at least 2 hours before and for at least 30 minutes after taking PROCYSBI.

If unable to take PROCYSBI without eating, take with food but limit the amount of food to approximately 4 ounces (½ cup) 1 hour before through 1 hour after administration. Avoid high fat food close to dosing. Avoid drinking alcohol while taking PROCYSBI.

2.1Important Dosing Instructions Initiate cysteamine treatment immediately after diagnosis of nephropathic cystinosis. Cysteamine-naïve Patients: Start PROCYSBI at a fraction of the maintenance dosage. Patients 1 year to less than 6 years : Gradually increase the dosage, allowing a minimum of 2 weeks between adjustments [see Dosage and Administration (2.2) ] .

Patients 6 years of age and older : Gradually increase the dosage over 4 to 6 weeks until the maintenance dosage is achieved. Patients switching from immediate-release cysteamine: Start the total daily dosage of PROCYSBI at a dosage equal to the previous total daily dosage of immediate-release cysteamine [see Dosage and Administration (2.3) ] . If adverse reactions occur, decrease the PROCYSBI dosage.

After the maintenance dosage of PROCYSBI is achieved: The dosage may need to be further increased to achieve a therapeutic target WBC cystine concentration. The maximum dosage of PROCYSBI is 1.95 grams/m 2 of body surface area per day [see Dosage and Administration (2.4 , 2.5) ] . Round dose calculations to the nearest incremental dosage that can be administered using the available strengths of PROCYSBI delayed-release capsules or packets of oral granules.

Only use whole capsules or entire contents of the packets.

2.2Starting and Maintenance Dosing in Cysteamine-Naïve Patients Start treatment with a dosage equal to ⅙ to ¼ of the maintenance dosage. The maintenance dosage after initial dose escalation is 1.3 g/m 2 of body surface area per day divided into two doses given every 12 hours. Table 1 shows the recommended starting and maintenance dosages of PROCYSBI, converted from body-surface area to body weight.

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 [see Dosage and Administration (2.4 , 2.5) ] . 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 mainte…

💊 Dosage Forms and Strengths 118 words ▾

3 DOSAGE FORMS AND STRENGTHS PROCYSBI delayed-release capsules: 25 mg cysteamine: the capsules have a light blue opaque cap imprinted with "PRO" in white ink and a light blue opaque body imprinted with "25 mg" in white ink. 75 mg cysteamine: the capsules have a dark blue opaque cap imprinted with "PRO" in white ink and a light blue opaque body imprinted with "75 mg" in white ink. PROCYSBI delayed-release oral granules: 75 mg cysteamine: white to off-white granules in single-use packets 300 mg cysteamine: white to off-white granules in single-use packets Delayed-release capsules: 25 mg and 75 mg cysteamine ( 3 ) Delayed-release oral granules: 75 mg and 300 mg cysteamine in single-use packets ( 3 )

⛔ Contraindications 29 words ▾

4 CONTRAINDICATIONS 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 )

⚠️ Warnings and Cautions ~2 min read ▾

5 WARNINGS AND PRECAUTIONS 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 )

5.1Ehlers-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) ] .

5.2Skin 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) ] .

5.3Gastrointestinal 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) ] .

5.4Fibrosing 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.

5.5Central 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 p…

🤒 Adverse Reactions ~3 min read ▾

6 ADVERSE REACTIONS 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 .

6.1Clinical 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 rates observed in clinical practice. The data described below reflect exposure to cysteamine in 345 patients with nephropathic cystinosis (246 patients receiving immediate-release cysteamine as cysteamine hydrochloride or phosphocysteamine, and 80 patients receiving PROCYSBI) in open-label clinical trials.

Clinical Trials Experience with PROCYSBI in Patients Switched from Immediate-Release Cysteamine Bitartrate Sixty-two patients with nephropathic cystinosis (38 males and 24 females) received PROCYSBI in two clinical trials at doses ranging from 0.29 grams/m 2 per day to 2.19 grams/m 2 per day [see Clinical Studies (14.2) ]. All patients were switched from immediate-release cysteamine to PROCYSBI. Forty-three patients, ages 6 to 26 years old, received PROCYSBI in an 8-week, open-label, randomized, cross-over trial comparing PROCYSBI to immediate-release cysteamine bitartrate.

Forty of 43 patients continued PROCYSBI treatment in an open-label extension trial (36 patients were treated with PROCYSBI for longer than 2 years, and 20 patients were treated for longer than 5 years). An additional 19 patients (6 renal transplanted patients and 13 patients aged 2 to 6 years) were enrolled directly into this trial (12 patients were treated with PROCYSBI for longer than 2 years, and 9 patients were treated for longer than 5 years). In the open-label, randomized, cross-over trial, a higher incidence of adverse reactions was reported in patients during the PROCYSBI treatment period compared with the immediate-release cysteamine bitartrate treatment period (see Table 2 ).

Other significant adverse reactions reported during clinical trials included hypersensitivity reactions, including anaphylaxis. Table 2: Adverse Reactions in ≥5% of Patients with Nephropathic Cystinosis in a Randomized, Cross-Over Trial Adverse Reaction Immediate-Release Cysteamine PROCYSBI (n = 41) % (n = 43) % Vomiting/emesis 12 19 Nausea 7 16 Abdominal pain/discomfort 0 14 Headache 0 9 Dizziness 0 5 Anorexia/loss of appetite 5 2 In the open-label extension trial (N=59), the most commonly reported adverse reactions (>15%) were vomiting, headache, diarrhea, nausea, conjunctivitis, influenza, gastroenteritis, nasopharyngitis, abdominal pain, dehydration, ear infection, upper respiratory tract infection, fatigue, arthralgia, cough, and pain in extremity.

Clinical Trials Experience with PROCYSBI in Cysteamine-Naïve Patients Seventeen cysteamine-naïve patients (fifteen patients between the ages of 1 and 5 years, one 9-year old and one 22-year old) received PROCYSBI in an open-label cl…

🔄 Drug Interactions 176 words ▾

7 DRUG INTERACTIONS

7.1Drugs 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) ].

7.2Use 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) ].

7.3Other 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.

👥 Use in Specific Populations ~2 min read ▾

8 USE IN SPECIFIC POPULATIONS Lactation: Breastfeeding is not recommended. ( 8.2 )

8.1Pregnancy Risk Summary There are no available data on PROCYSBI use in pregnant women to inform any drug-associated risks for birth defects or miscarriage [see Data ] . Cysteamine (administered as cysteamine bitartrate) was teratogenic and fetotoxic in rats at doses less than the recommended human maintenance dose. The background risk of major birth defects and miscarriage for the indicated population is unknown.

In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively. Advise pregnant women of the potential risk to a fetus. Data Animal Data Embryo-fetal development studies were conducted in rats using oral administration of cysteamine bitartrate, with a dose range of 37.5 to 150 mg/kg per day of cysteamine equivalent (about 0.2 to 0.7 times the recommended human maintenance dose based on body surface area).

Cysteamine bitartrate was fetotoxic and produced adverse developmental effects. Observed teratogenic findings were cleft palate, kyphosis, heart ventricular septal defects, microcephaly and exencephaly.

8.2Lactation Risk Summary There is no information on the presence of cysteamine in human milk, the effects on the breast-fed infant, or the effects on milk production. Cysteamine is present in the milk of lactating rats [see Data ]. Because of the potential for serious adverse reactions in breastfed infants from cysteamine, breastfeeding is not recommended.

Data A decrease in survival occurred in neonatal rats nursed by mothers receiving cysteamine [see Nonclinical Toxicology (13) ] .

8.4Pediatric Use The safety and effectiveness of PROCYSBI have been established in pediatric patients 1 year of age and older for the treatment of nephropathic cystinosis. Use of PROCYSBI is supported by evidence from patients switched to PROCYSBI from immediate-release cysteamine bitartrate in two trials: an open-label, randomized, cross-over trial in adults and pediatric patients aged 6 years and older (n=43) and an open-label extension trial in pediatric patients aged 2 years and older (n=59). Another open-label trial was conducted in cysteamine naïve pediatric patients 1 year to less than 6 years of age (n=15) [see Clinical Trials (14.2) ] .

The safety profile in pediatric patients was similar to adults. In patients less than 6 years of age, vomiting occurred in 12/15 cysteamine treatment naïve patients compared to 11/13 patients switched from immediate-release cysteamine to PROCYSBI. The safety and effectiveness of PROCYSBI have not been established in patients less than 1 year of age.

8.5Geriatric Use No studies with PROCYSBI have been conducted in geriatric patients.

🤰 Pregnancy 170 words ▾

8.1Pregnancy Risk Summary There are no available data on PROCYSBI use in pregnant women to inform any drug-associated risks for birth defects or miscarriage [see Data ] . Cysteamine (administered as cysteamine bitartrate) was teratogenic and fetotoxic in rats at doses less than the recommended human maintenance dose. The background risk of major birth defects and miscarriage for the indicated population is unknown.

In the U.S. general population, the estimated background risk of major birth defects and miscarriage in clinically recognized pregnancies is 2 to 4% and 15 to 20%, respectively. Advise pregnant women of the potential risk to a fetus. Data Animal Data Embryo-fetal development studies were conducted in rats using oral administration of cysteamine bitartrate, with a dose range of 37.5 to 150 mg/kg per day of cysteamine equivalent (about 0.2 to 0.7 times the recommended human maintenance dose based on body surface area).

Cysteamine bitartrate was fetotoxic and produced adverse developmental effects. Observed teratogenic findings were cleft palate, kyphosis, heart ventricular septal defects, microcephaly and exencephaly.

🧒 Pediatric Use 156 words ▾

8.4Pediatric Use The safety and effectiveness of PROCYSBI have been established in pediatric patients 1 year of age and older for the treatment of nephropathic cystinosis. Use of PROCYSBI is supported by evidence from patients switched to PROCYSBI from immediate-release cysteamine bitartrate in two trials: an open-label, randomized, cross-over trial in adults and pediatric patients aged 6 years and older (n=43) and an open-label extension trial in pediatric patients aged 2 years and older (n=59). Another open-label trial was conducted in cysteamine naïve pediatric patients 1 year to less than 6 years of age (n=15) [see Clinical Trials (14.2) ] .

The safety profile in pediatric patients was similar to adults. In patients less than 6 years of age, vomiting occurred in 12/15 cysteamine treatment naïve patients compared to 11/13 patients switched from immediate-release cysteamine to PROCYSBI. The safety and effectiveness of PROCYSBI have not been established in patients less than 1 year of age.

🧓 Geriatric Use 13 words ▾

8.5Geriatric Use No studies with PROCYSBI have been conducted in geriatric patients.

🆘 Overdosage 135 words ▾

10 OVERDOSAGE 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.

🧬 Clinical Pharmacology ~3 min read ▾

12 CLINICAL PHARMACOLOGY

12.1Mechanism of Action Cysteamine is an aminothiol that participates within lysosomes in a thiol-disulfide interchange reaction converting cystine into cysteine and cysteine-cysteamine mixed disulfide, both of which can exit the lysosome in patients with cystinosis.

12.2Pharmacodynamics Normal individuals and persons heterozygous for cystinosis have WBC cystine concentrations of less than 0.2 and usually below 1 nmol ½ cystine/mg protein, respectively, when measured using the mixed leukocyte assay. Untreated patients with nephropathic cystinosis have elevations of WBC cystine concentration above 2 nmol ½ cystine/mg protein. After the administration of a single dose of PROCYSBI, peak concentrations of cysteamine were observed at 3 hours post-dose.

The nadir of WBC cystine closely followed the peak concentrations at 3.5 hours post-dose, and returned to baseline WBC concentrations at 12 hours-post dose. The cystine concentration in WBC lysate was measured with LC/MS/MS and total protein content in human WBC lysate was measured using the bicinchoninic acid (BCA) assay. A correction factor was applied to the total protein content for the difference in results from the Lowry method.

The cystine concentration in nmol ½ cystine/mg protein was calculated by multiplying nmol cystine/mg protein by 2 [see Dosage and Administration (2.5) ].

12.3Pharmacokinetics The pharmacokinetics of cysteamine with administration of PROCYSBI were evaluated in 43 patients age ranged from 6 to 26 years (mean age 12 years) with cystinosis and with an estimated glomerular filtration rate of greater than 30 mL/minutes/1.73 m2. Table 4 shows the mean pharmacokinetic parameters for PROCYSBI and immediate-release cysteamine bitartrate at steady state. The mean (± SD) dose for PROCYSBI was 656 ± 144 mg/m2 (given every 12 hours) and for immediate-release cysteamine bitartrate was 404 ± 88 mg/m2 (given every 6 hours).

Table 4: Pharmacokinetic parameters for cysteamine at steady state* after administration of PROCYSBI or immediate-release cysteamine bitartrate Immediate-release cysteamine bitartrate given every 6 hours PROCYSBI given every 12 hours C max (mg/L) 2.7 ± 1.4 3.6 ±

1.8AUC 0-6h (min*mg/L) 351 ± 153 NA AUC 0-12h (min*mg/L) NA 726 ± 339 AUC inf (min*mg/L) 380 ± 157 785 ± 358 T max (min) 73 ± 31 188 ± 88 t½ (min) 90 ± 24 253 ± 403 CL/F (L/min) 1.4 ± 0.8 1.2 ±

0.8Vd/F (L) 198 ± 159 382 ± 404 Absorption The pharmacokinetics of cysteamine with administration of PROCYSBI are consistent with a delayed-release formulation; the mean T max for cysteamine was 188 minutes with PROCYSBI compared with 73 minutes for immediate-release cysteamine bitartrate. The mean plasma cysteamine peak and AUC were similar when a single PROCYSBI dose of 600 mg was administered with 240 mL orange juice or with 240 mL water. The systemic exposure to cysteamine was similar when PROCYSBI was administered with orange juice as a whole capsule and sprinkled in applesauce in the fasted state.

In a food effect study conducted in healthy subjects (n=20), administration of a meal 30 minutes following PROCYSBI administration (intact capsules) decreased C max by 34% and AUC 0-t by 32% compared to administration of a meal 2 hours post-dose [see Dosage and Administration (2.6) ] . Distribution Cysteamine was moderately bound to human plasma proteins, predominantly to albumin, with mean protein binding of about 52%. Plasma protein binding was independent of concentration over the concentration range achieved clinically with the recommended doses.

The volume of distribution (Vd/F) was 382 L for PROCYSBI compared with 198 L for immediate-release cysteamine bitartrate. Elimination After each dose of PROCYSBI the cysteamine concentration in the blood continues to decline for approximately 30 minutes and the WBC cystine concentration increases accordingly . The apparent clearance (Cl/F) of cysteamine was similar between PROCYSBI (1.2 ±

0.8 L/min) and immediate-release cystea…

🧬 Mechanism of Action 36 words ▾

12.1Mechanism of Action Cysteamine is an aminothiol that participates within lysosomes in a thiol-disulfide interchange reaction converting cystine into cysteine and cysteine-cysteamine mixed disulfide, both of which can exit the lysosome in patients with cystinosis.

📦 How Supplied / Storage and Handling ~1 min read ▾

16 HOW SUPPLIED/STORAGE AND HANDLING How Supplied PROCYSBI (cysteamine bitartrate) delayed-release capsules 25 mg cysteamine : A hard gelatin capsule with light blue opaque cap imprinted with "PRO" in white ink and light blue opaque body imprinted with "25 mg" in white ink, supplied as bottle of 60 capsules (NDC 75987-100-04). Each bottle contains one desiccant canister and one oxygen absorber canister. 75 mg cysteamine : A hard gelatin capsule with dark blue opaque cap imprinted with "PRO" in white ink and light blue opaque body imprinted with "75 mg" in white ink, supplied as bottle of 250 capsules (NDC 75987-101-08).

Each bottle contains one desiccant canister and two oxygen absorber canisters. PROCYSBI (cysteamine bitartrate) delayed-release oral granules 75 mg cysteamine: Single-use packets containing white to off-white granules, supplied as 60 packets in a carton (NDC 75987-140-13). 75 mg cysteamine: Single-use packets containing white to off-white granules, supplied as 120 packets in a carton (NDC 75987-140-14).

300 mg cysteamine: Single-use packets containing white to off-white granules, supplied as 60 packets in a carton (NDC 75987-145-13). 300 mg cysteamine: Single-use packets containing white to off-white granules, supplied as 120 packets in a carton (NDC 75987-145-14). Storage and Handling Prior to dispensing, store PROCYSBI delayed-release capsules and PROCYSBI delayed-release oral granules in a refrigerator, 2°C to 8°C (36°F to 46°F).

Dispense PROCYSBI delayed-release capules and PROCYSBI delayed-release oral granules with a 4 month discard date. Dispense in original packaging. Do not subdivide or repackage.

Do not remove desiccant or oxygen absorber(s) from the bottle of PROCYSBI delayed-release capsules. Keep bottles tightly closed in a dry place. Protect from light and moisture.

Instructions for the Patient Store PROCYSBI delayed-release capsules and PROCYSBI delayed-relase oral granules at room temperature, 20°C to 25°C (68°F to 77°F) in the original packaging. Do not subdivide or repackage. Protect from light and moisture.

Do not store PROCYSBI delayed-release oral granules in opened packets.

📦 Storage and Handling 75 words ▾

Storage and Handling Prior to dispensing, store PROCYSBI delayed-release capsules and PROCYSBI delayed-release oral granules in a refrigerator, 2°C to 8°C (36°F to 46°F). Dispense PROCYSBI delayed-release capules and PROCYSBI delayed-release oral granules with a 4 month discard date. Dispense in original packaging.

Do not subdivide or repackage. Do not remove desiccant or oxygen absorber(s) from the bottle of PROCYSBI delayed-release capsules. Keep bottles tightly closed in a dry place.

Protect from light and moisture.

📋 Description 191 words ▾

11 DESCRIPTION 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

💬 Information for Patients ~3 min read ▾

17 PATIENT COUNSELING INFORMATION Advise the patient to read the FDA-approved patient labeling (Patient Information and Instructions for Use) Ehlers-Danlos-like Syndrome Advise patients and caregivers that PROCYSBI may cause abnormalities of the skin, bones, and joints. Advise patients to report any skin changes or problems with their bones or joints to their physician [see Warnings and Precautions (5.1) ] . Skin Rash Advise patients and caregivers to contact their physician immediately if they experience a skin rash [see Warnings and Precautions (5.2) ] .

Gastrointestinal Ulcers and Bleeding Advise patients and caregivers that PROCYSBI may cause ulcers and bleeding. Advise patients to contact their physician immediately if they experience stomach pain, nausea, vomiting, loss of appetite, or are vomiting blood [see Warnings and Precautions (5.3) ] . Fibrosing Colonopathy Advise patients and caregivers that fibrosing colonopathy has been reported with PROCYSBI.

Advise patients to contact their physician immediately if they experience severe, persistent and/or worsening stomach pain, vomiting, bloody or persistent diarrhea, or inability to control bowel movements [see Warnings and Precautions (5.4) ] . Central Nervous System Symptoms Advise patients and caregivers that PROCYSBI may impair their ability to perform tasks such as driving or operating machinery. Advise patients to contact their physician immediately if they experience seizures, lethargy, somnolence, depression, and encephalopathy [see Warnings and Precautions (5.5) ] .

Benign Intracranial Hypertension Advise patients and caregivers that PROCYSBI may cause benign intracranial hypertension. Advise patients to contact their physician immediately if they experience headache, tinnitus, dizziness, nausea, double vision, blurry vision, loss of vision, or eye pain [see Warnings and Precautions (5.7) ] . Use by Pregnant Women Advise patients and to contact their physician immediately if they suspect they may be pregnant.

Discuss with the patient the individual risks and benefits of continuing PROCYSBI during pregnancy [see Use in Specific Populations (8.1) ] . Breastfeeding Advise patients that breastfeeding is not recommended while taking PROCYSBI [see Use in Specific Populations (8.2) ] . Laboratory Monitoring Discuss with the patient and caregivers the importance of required laboratory testing to determine the correct dose of PROCYSBI [see Dosage and Administration (2.5) ] .

Administration Advise patients and caregivers to follow the instruction below for taking PROCYSBI. Capsules: Swallow PROCYSBI capsules whole. Do not crush or chew capsules or capsule contents.

Take PROCYSBI capsules with fruit juice (except grapefruit juice) or water. For patients who cannot swallow the capsules, the capsules can be opened and the capsule contents sprinkled on and mixed in applesauce, berry jelly or fruit juice (except grapefruit juice) and administered orally. For patients with a gastrostomy tube, follow the instructions in the Instructions for Use.

Oral Granules: Do not crush or chew the granules. Sprinkle and mix intact granules from on applesauce, berry jelly or fruit juice (except grapefruit juice) and administer orally. For patients with a gastrostomy tube, follow the instruction in the Instructions for Use Administer PROCYSBI at least 1 hour before or 1 hour after medications containing bicarbonate or carbonate.

Do not eat for at least 2 hours before and for at least 30 minutes after taking PROCYSBI. If unable to take PROCYSBI without eating, take with food but limit the amount of food to approximately 4 ounces (½ cup) 1 hour before through 1 hour after administration. Avoid high fat food close to dosing of PROCYSBI .

Avoid drinking alcohol while taking PROCYSBI. Take PROCYSBI consistently and to not miss doses. If a dose is missed, take the dose as soon as possible up to 8 hours after the scheduled time.

However, if a dose is missed and the next scheduled dose is due in less t…

Source: FDA Structured Product Labeling, mirrored from DailyMed / openFDA. Prefer the government’s original formatting? View this label on DailyMed ↗
← 75987-0130-15 · Horizon 75987-0145-13 · Horizon →
For educational and professional reference only — not medical advice. Pricing reflects published NADAC and CMS ASP (free public data) and may differ from your acquisition cost; always verify before billing or dispensing.