Horizon Therapeutics announced the availability of Procysbi (cysteamine bitartrate) delayed-release oral granules in tear-open packets for the treatment of nephropathic cystinosis in patients aged 1 year and older. 

The new packets provide an alternative treatment option for patients and caregivers. The contents of the packet can be sprinkled on certain foods (applesauce, berry jelly) and juice (except grapefruit juice) and administered orally or mixed with applesauce and administered via a gastrostomy tube. 

“The day-to-day challenges of managing a rare disease like cystinosis can be overwhelming, so new developments like this one can make an important difference in the lives of families living with cystinosis.” said Paul Grimm, MD, pediatric nephrologist and professor of pepediatrics at The Lucile Salter Packard Children’s Hospital. “Physicians now have the option of prescribing tear-open packets for any of their patients who have had to open individual capsules to remove the granules.”

Procysbi, a cystine-depleting agent, is now available in 75mg and 300mg single-use packets and in 25mg and 75mg delayed-release capsules. Both the capsules and packets contain the same oral granules that have a coating designed to break down in low acid environments. Doses should be rounded to the nearest incremental dosage that can be administered using the available strengths of either the capsules or packets; the entire capsule or contents of a packet should be used.


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Nephropathic cystinosis is a rare metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues and organs in the body.

For more information visit horizontherapeutics.com.

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This article originally appeared on MPR