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Why PROCYSBI

How PROCYSBI works PROCYSBI Microbeads Efficacy Safety

How PROCYSBI works

PROCYSBI is the only CDT with 12-hour dosing

Controlling cystine levels is crucial to limiting damage that may be caused by cystinosis. With regular cystine-depleting therapy (CDT) use, some damage to organs may be stabilized or delayed.1-3

Meet Hannah. Like many college students, she loves music, being outdoors, and spending time with her friends.

Hannah also has nephropathic cystinosis, a rare, genetic disorder. She was diagnosed when she was only 15 months old.

Let’s take a closer look into how this condition affects the body.

Cystinosis is a rare, autosomal recessive lysosomal storage disorder that results in accumulation of crystallized cystine within lysosomes throughout the body.

Nephropathic cystinosis is the most common and most severe form of the disease.

In nephropathic cystinosis, a defect in the lysosomal cystine transporter, cystinosin,…

…results in lysosomal accumulation of the amino acid cystine, which forms crystals and affects cells…

…in every organ and tissue of the body.

Progressive damage to organs and tissues throughout the body can cause renal disease, rickets, photophobia, hypothyroidism, diabetes, renal failure, myopathy, and pulmonary dysfunction.

Cystine accumulation begins in utero, causing renal cell damage and symptoms of Fanconi syndrome by 4 to 6 months of age. Without treatment, this may lead to end-stage renal disease within 10 years. Organ damage is irreversible, but it can be delayed with early intervention…

…and treatment with cystine-depleting therapy, such as PROCYSBI (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules.

Cystine-depleting therapy, or CDT, is currently the only treatment available for removing cystine from the body. CDT reduces the accumulation of cystine within the cells when taken at the same times and the same way every day.

PROCYSBI is designed to provide continuous cystine control over 12 hours.

PROCYSBI capsules and packets contain granules, also called microbeads, composed of cysteamine bitartrate surrounded by an acid-resistant enteric coating.

The coated microbeads allow cysteamine bitartrate to pass through the acidic environment of the stomach and dissolve in the more alkaline environment of the small intestine.

Delayed release allows cystine levels to remain within therapeutic range for 12 hours. For PROCYSBI to work properly, the cysteamine bitartrate in the microbeads must release only into the small intestine.

To prevent the microbeads from dissolving in the stomach, the gastric environment must be acidic.

Certain foods and drinks can raise gastric pH, causing the medicine to release too soon in the stomach.

This can cause gastrointestinal distress, such as abdominal pain, nausea, and vomiting. They can also alter the pharmacokinetic properties and effectiveness of PROCYSBI.

This is why PROCYSBI capsules should be administered with high-acidity drinks, such as water or fruit juice (except grapefruit juice), and PROCYSBI granules mixed with applesauce or berry jelly—to ensure that gastric pH levels remain low.

Drugs that increase gastric pH, such as antacids containing bicarbonate or carbonate, may cause PROCYSBI to release prematurely into the stomach.

Patients should take PROCYSBI at least 1 hour before or after medicines containing bicarbonate or carbonate, or other types of antacids. Taking omeprazole with PROCYSBI does not affect the pharmacokinetics of cysteamine when PROCYSBI is taken with orange juice or water.

Advise your patients to avoid high-fat foods right before or after taking PROCYSBI because they may decrease absorption.

Alcohol may increase the rate of cysteamine release and/or adversely alter the pharmacokinetic properties of PROCYSBI, affecting its effectiveness and safety, and therefore should be avoided.

Routine monitoring of cystine levels in the white blood cells is required to determine an effective dose for your patient. Obtain WBC cystine concentration 2 weeks after starting PROCYSBI. Once the therapeutic target is achieved, continue monitoring monthly for treatment-naïve patients and quarterly for switch patients. Patients should then be monitored twice a year at minimum.

Due to the constant toxic accumulation of cystine, consistent adherence to PROCYSBI is essential for continuous cystine control.

Without regular treatment, cystine will continue to accumulate and cause organ and tissue damage throughout the body. Even brief interruptions in dosing can result in a rapid return to toxic levels of cystine.

Hannah: We all have busy lives, and it can be hard to balance your treatments with your personal life, especially if you’re feeling fine!

But I know it’s important, so I create a schedule that works for me and set alarms to remind me when it’s time to take my PROCYSBI.

I always take my PROCYSBI capsules the same way, with water, at the same times every day. My doctor helped me to come up with a meal plan that works best for me, too.

Taking charge of my cystinosis at a young age has definitely helped me to become more responsible and independent. But I know there’s always a group of people around me who support me.

Cystinosis doesn’t define me, I define cystinosis!

Ensuring that your patients understand how to take PROCYSBI correctly will help them to get the maximum benefit from their treatment.

Contact your Horizon representative for more information.

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The Role of PROCYSBI

PROCYSBI is a cystine-depleting therapy (CDT) and is the first and only FDA-approved treatment indicated for cystinosis with 12-hour dosing.4 The formulation of PROCYSBI is driven by cysteamine bitartrate, a crucial element in managing lysosomal storage disorders such as nephropathic cystinosis.5 Cysteamine significantly depletes intracellular cystine by converting it into cysteine and cysteine-cysteamine mixed disulfide, both of which can exit the lysosome.

How it works: Proprietary formula allows release in the small intestine4-6

PROCYSBI granules, also called microbeads, are composed of cysteamine bitartrate surrounded by an acid-resistant enteric coating. The microbeads release cysteamine gradually, which are designed to provide 12 hours of continuous cystine control.4-8

The coating of the microbeads gradually dissolves to help control the release of the cysteamine bitartrate into the alkaline small intestine and bypass absorption by the acidic stomach.4-6

PROCYSBI microbeads release in the small intestine PROCYSBI microbeads release in the small intestine

As the microbeads gradually release cysteamine bitartrate in the small intestine, PROCYSBI is designed to control cystine levels over the dosing interval.4-6

PROCYSBI and the stomach
  • Some medicines, including those that contain bicarbonate or carbonate, may change the acid level in the stomach. PROCYSBI must be taken 1 hour before or after these medicines.8
  • Consumption of alcohol with PROCYSBI may increase the rate of cysteamine release and/or adversely alter the pharmacokinetic properties, effectiveness, and safety of PROCYSBI.8

Certain food and drinks can cause a patient’s stomach acid level to be too low, which can cause the microbeads to release too soon and be absorbed in the stomach.8

Understanding Lysosomal Storage Disorder Cystinosis

Lysosomal storage disorders encompass a group of rare genetic conditions characterized by the accumulation of substances, such as amino acids, within cells, leading to diverse symptoms and organ damage.4 Cystinosis disrupts the body’s ability to process cystine, resulting in its buildup within cells, notably in the kidneys, often leading to renal failure and the need for a kidney transplant.1,9

Cysteamine lowers intra-lysosomal cystine by reacting with cystine to form free cysteine and a mixed disulfide. Consistent use of cysteamine can slow down the progression toward kidney failure in individuals with cystinosis.6

PROCYSBI Microbeads

PROCYSBI microbeads are available in capsules and tear-open packets

Both the capsules and tear-open packets contain the same PROCYSBI microbeads that are designed to provide 12 continuous hours of cystine control to patients.6

  • Capsules are available in 25 mg and 75 mg strengths.
  • Patients who prefer to swallow whole capsules may be good candidates for PROCYSBI in capsules.
  • Packets are available in 75 mg and 300 mg strengths.
  • Patients who may be good candidates for PROCYSBI in packets include those who

   -Take their medicine via a gastrostomy tube (G-tube), 

   -Have difficulty swallowing capsules, 

   -Prefer to open capsules over swallowing them.

PROCYSBI (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules is the only FDA-approved treatment for nephropathic cystinosis with 12-hour dosing.

PROCYSBI also offers a choice, because it’s available in capsules and in tear-open packets.

Both the capsules and the packets contain PROCYSBI granules, also called “microbeads,” composed of cysteamine bitartrate surrounded by an acid-resistant enteric coating.

The coated microbeads allow cysteamine bitartrate to pass through the acidic stomach…

…to the alkaline environment of the small intestine.

Once in the small intestine, the coating begins to dissolve and the microbeads release cysteamine bitartrate gradually, which allows PROCYSBI to control cystine levels continuously over 12 hours.

PROCYSBI microbeads are available in tear-open packets and in capsules.

Patients who may be good candidates for PROCYSBI in packets include those who use a gastrostomy tube, or “G-tube;” have trouble swallowing; or take the time to open capsules at every dose.

Patients who prefer to swallow whole capsules may be good candidates for PROCYSBI in capsules.

PROCYSBI is the only cystinosis treatment that is available in packets and capsules, and it provides patients with 12-hour cystine control.

For more information about PROCYSBI in capsules and packets, visit PROCYSBIHCP.com.

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PROCYSBI® (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules is the first and only 12-hour dosing treatment indicated for cystinosis.1 PROCYSBI uses proprietary technology that releases cysteamine gradually and is designed to provide 12-hour continuous cystine control in adults and children 1 year of age and older.2,5-7

Efficacy

PROCYSBI has been well studied in clinical trials in adults and children as young as 1 year8

PROCYSBI given every 12 hours was found to be noninferior to immediate-release (IR) cysteamine bitartrate given every 6 hours8

Delayed release of cysteamine into the plasma has been shown to allow cystine levels to remain within therapeutic range for a full 12 hours.10

The primary outcome was the WBC cystine levels.

The mean peak WBC cystine level (least-square mean ± SEM) measured in the per-protocol population.

Plasma cysteamine concentrations and leukocyte cystine levels following a single dose of PROCYSBI vs IR cysteamine (N=39)8

Plasma cysteamine concentrations and leukocyte cystine levels chart Plasma cysteamine concentrations and leukocyte cystine levels chart
Study Design

Open-label, randomized, controlled, crossover trial. Using a linear mixed effects statistical analysis model, the least-square mean value of WBC cystine was calculated as 0.52 ± 0.06 nmol ½ cystine/mg protein 12 hours after taking PROCYSBI and 0.44 ± 0.06 nmol ½ cystine/mg protein 6 hours after taking IR cysteamine, a difference of 0.08 ± 0.03 nmol ½ cystine/mg protein (95.8% CI: 0.01 to 0.15) (P < 0.0001).8
In children aged 1 to <6 years, measures of white blood cell (WBC) cystine levels, weight, and height were improved while on PROCYSBI.8

  • Mean WBC cystine levels were controlled below the target level* at 12 and 18 months.8

Mean WBC cystine concentration table Mean WBC cystine concentration table

*Target = <1.0 nmol ½ cystine/mg protein.1
†WBC cystine concentrations were measured 30 minutes after the morning dose.

  • Weight z-scores were8
    -4.0 ± 2.1 at baseline,
    -2.2 ± 1.7 at 12 months,
    -1.3 ± 2.0 at 18 months
  • Children taking PROCYSBI for 18 months reached the 30th mean weight percentile8
  • In the same patients, similar trends were observed for height8
Growth measures improved while on PROCYSBI chart Growth measures improved while on PROCYSBI chart
Study Design

Multicenter, open-label clinical trial in cysteamine-naïve patients aged 1 to <6 years. Of 15 patients aged 1 to <6 years, 14 patients completed 12 months of treatment and 10 completed 18 months of treatment.8
In patients aged ≥2 years, PROCYSBI maintained kidney function at 1 and 2 years7,8

  • 40 out of 41 patients who completed the Phase 3 trial chose to continue with PROCYSBI8
  • 36 of the 40 patients continued treatment for at least 24 months, and 20 of the 40 continued for longer than 60 months8
  • Participants maintained their mean WBC cystine concentrations below 1.0 nmol ½ cystine/mg protein8
Kidney function maintained while on PROCYSBI chart Kidney function maintained while on PROCYSBI chart
Study Design

Multicenter, single-arm, open-label, long-term extension trial including both pediatric (aged ≥2 years) and adult patients. Forty of the 41 patients completing the randomized trial continued treatment with PROCYSBI in an open-label extension trial. Thirteen pediatric patients aged 2 to 6 years were also enrolled in the extension trial and treated with PROCYSBI. Kidney function was assessed monthly for 6 months and then quarterly thereafter; individual patient results are provided for the month they came in for each of their assessments. All patients were continuously measured over this time period. The eGFR of the study population was evaluated and recorded throughout the 24 months of the study. The box plots show the median (–), mean (◆), and the first and third quartiles are denoted by the upper and lower ends of the box, respectively. The lower and upper whiskers denote the minimum and maximum, respectively, and the (o) represents the outliers. The mean eGFR did not decline significantly (P = 0.32) during the course of the 24-month study.8

Consider open-label extension (OLE) study limitations when interpreting results. The OLE is not blinded, not controlled, and includes self-selection bias. During this 24-month period, 1 patient went to transplantation at 17 months, and 1 patient withdrew from the study at 21 months with a decline in eGFR and was placed on maintenance dialysis.

Safety

In clinical trials of PROCYSBI, the most common adverse reactions reported were8
  • Patients aged ≥6 years previously treated with cysteamine (>5%): vomiting, nausea, abdominal pain, breath odor, diarrhea, skin odor, fatigue, rash, and headache
  • Patients aged 1 to <6 years naïve to cysteamine treatment (>10%): vomiting, gastroenteritis/viral gastroenteritis, diarrhea, breath odor, nausea, electrolyte imbalance, and headache
Healthcare provider speaking with a patient Healthcare provider speaking with a patient
If patients experience tolerability issues when starting PROCYSBI, the dose should be temporarily adjusted as follows8:
  • Cysteamine-naïve patients: If a patient experiences initial intolerance, temporarily discontinue PROCYSBI, restart at a lower dosage, and gradually increase dosage
  • Patients switching from IR cysteamine: For patients who may experience temporary intolerance upon starting PROCYSBI, decrease the dosage and then gradually increase to the maintenance dosage
Healthcare provider consulting with an older patient Healthcare provider consulting with an older patient
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References

1. Gahl WA, Balog JZ, Kleta R. Nephropathic cystinosis in adults: natural history and effects of oral cysteamine therapy. Ann Intern Med. 2007;147(4):242-250. 2. Elmonem MA, Veys KR, Soliman NA, van Dyck M, van den Heuvel LP, Levtchenko E. Cystinosis: a review. Orphanet J Rare Dis. 2016;11(47):1-17. 3. Nesterova G, Gahl WA. Cystinosis: the evolution of a treatable disease. Pediatr Nephrol. 2013;28(1):51-59. 4. Veys KR, Besouw MT, Pinxten AM, van Dyck M, Casteels I, Levtchenko EN. Cystinosis: a new perspective. Acta Clin Belg. 2016;71(3):131-137. 5. Dohil R, Fidler M, Gangoiti JA, Kaskel F, Schneider JA, Barshop BA. Twice-daily cysteamine bitartrate therapy for children with cystinosis. J Pediatr. 2010;156(1):71-75. 6. Dohil R, Rioux P. Pharmacokinetic studies of cysteamine bitartrate delayed-release. Clin Pharmacol Drug Dev. 2013;2(2):178-185. 7. Langman CB, Greenbaum LA, Grimm P, et al. Quality of life is improved and kidney function preserved in patients with nephropathic cystinosis treated for 2 years with delayed-release cysteamine bitartrate. J Pediatr. 2014;165(3):528-533. 8. PROCYSBI (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules [prescribing information]. Amgen. 9. Gahl WA, Thoene JG, Schneider JA. Cystinosis. N Engl J Med. 2002;347(2):111-121. 10. Langman CB, Greenbaum LA, Sarwal M, et al. A randomized controlled crossover trial with delayed-release cysteamine bitartrate in nephropathic cystinosis: effectiveness on white blood cell cystine levels and comparison of safety. Clin J Am Soc Nephrol. 2012;7(7):1112-1120.

INDICATION and IMPORTANT SAFETY INFORMATION

INDICATION

PROCYSBI (cysteamine bitartrate) delayed-release capsules and delayed-release oral granules is a cystine-depleting agent indicated for the treatment of nephropathic cystinosis in adults and pediatric patients 1 year of age and older.

IMPORTANT SAFETY INFORMATION

CONTRAINDICATIONS

  • Patients with 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. Monitor patients for development of skin or bone lesions and reduce PROCYSBI dosing if patients develop these lesions.
  • Skin Rash: Severe skin rashes such as erythema multiforme bullosa or toxic epidermal necrolysis have been reported in patients receiving immediate-release cysteamine bitartrate. Discontinue use if severe skin rash occurs.
  • Gastrointestinal (GI) Ulcers and Bleeding: GI ulceration and bleeding have been reported in patients receiving immediate-release cysteamine bitartrate. Monitor for GI symptoms and consider decreasing the dose if severe symptoms occur.
  • Fibrosing Colonopathy: Fibrosing colonopathy has been reported with postmarketing use of PROCYSBI. 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.
  • Central Nervous System (CNS) Symptoms: CNS symptoms such as seizures, lethargy, somnolence, depression, and encephalopathy have been associated with immediate-release cysteamine. Monitor for CNS symptoms; interrupt or reduce the dose for severe symptoms or those that persist or progress.
  • 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; decrease or discontinue the dose until values revert to normal.
  • Benign lntracranial Hypertension: Benign intracranial hypertension (pseudotumor cerebri; PTC) and/or papilledema has been reported in patients receiving immediate-release cysteamine bitartrate treatment. Monitor for signs and symptoms of PTC; interrupt or reduce the dose for signs/symptoms that persist, or discontinue if diagnosis is confirmed.

ADVERSE REACTIONS

The most common adverse reactions reported in PROCYSBI clinical trials (≥ 5%): were:

  • Patients 2 years of age and older previously treated with cysteamine: vomiting, nausea, abdominal pain, headache, conjunctivitis, influenza, gastroenteritis, nasopharyngitis, dehydration, ear infection, upper respiratory tract infection, fatigue, arthralgia, cough, and pain in extremity.
  • Patients 1 year of age and older naïve to cysteamine treatment: vomiting, gastroenteritis/viral gastroenteritis, diarrhea, breath odor, nausea, electrolyte imbalance, headache.

DRUG INTERACTIONS

  • Drugs that increase gastric pH may alter the pharmacokinetics of cysteamine due to the premature release of cysteamine from PROCYSBI and increase WBC cystine concentration. Monitor WBC cystine concentration with concomitant use.
  • 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: Because of the potential risk for serious adverse reactions in breastfed children from cysteamine, breastfeeding is not recommended during treatment with PROCYSBI.

Please see Full Prescribing Information.

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