The Food and Drug Administration (FDA) has approved Procysbi (cysteamine bitartrate; Horizon Therapeutics) delayed-release oral granules in 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.
Procysbi, a cystine-depleting agent, is expected to be available in 75mg and 300mg single-use packets in the first half of 2020 and is currently available in 25mg and 75mg delayed-release capsules. Both the capsules and packets contain the same oral granules.
In cysteamine-naïve patients, starting and maintenance dosages are based on body weight. Dose calculations should be rounded to the nearest incremental dosage that can be administered using the available strengths of delayed-release capsules or packets of oral granules.
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