This site is intended for healthcare professionals

FDA approves new dosage form for Procysbi to treat nephropathic cystinosis.- Horizon Therapeutics

Read time: 1 mins
Published:27th Feb 2020
Horizon Therapeutics plc announced that the FDA has approved Procysbi (cysteamine bitartrate) delayed-release oral granules in packets for adults and children one year of age and older living with nephropathic cystinosis. This new dosage form provides another option for people living with cystinosis, in addition to the currently available Procysbi capsules. Both the capsules and the packets contain the same Procysbi granules, also called microbeads, that provide 12 hours of cystine control Providing oral granules in packets offers a tear-open option for those living with cystinosis who may have difficulty swallowing, need to sprinkle the granules on certain foods or liquids, or administer medication through a gastrostomy tube (g-tube). Families living with cystinosis are often challenged with managing multiple, different medications on daily basis and the oral granules in tear-open packets will provide an alternative option for accessing Procysbi day-to-day. Procysbi oral granules in packets in 75 mg and 300 mg strengths are expected to be available in the first half of the year. Procysbi capsules will continue to be available in 25 mg and 75 mg strengths.
Condition: Nephropathic Cystinosis
Type: drug

Learning Zones

The Learning Zones are an educational resource for healthcare professionals that provide medical information on the epidemiology, pathophysiology and burden of disease, as well as diagnostic techniques and treatment regimens.