The Food and Drug Administration (FDA) has accepted for review the New Drug Application (NDA) for Procysbi (cysteamine bitartrate; Horizon Therapeutics) delayed-release oral granules in packets for the treatment of nephropathic cystinosis in patients aged 1 year and older.

Currently, the FDA-approved capsule formulation can be opened and the contents may be mixed with fruit juice (except grapefruit juice), applesauce or berry jelly, if patients have difficulty swallowing the capsules. The new packet formulation would provide an alternative treatment option; patients and caregivers would be able to sprinkle the contents of the packet on certain foods and juice (or administer via a feeding tube). 

“The submission of this NDA is the result of feedback we’ve received from people living with cystinosis, their caregivers and healthcare professionals, and is part of our ongoing reinvestment into the cystinosis community,” said Gregg Checani, MD, executive medical director, medical affairs and clinical science, Horizon. 

The FDA is expected to make a decision on the NDA in 2020.

Related Articles

Procysbi, a cystine-depleting agent, is currently available as 25mg capsules in 60-count bottles and 75mg capsules in 250-count bottles.

For more information visit