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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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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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

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

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.