FDA accepts NDA to make Procysbi available as oral granules in packets to treat nephropathic cystinosis

Horizon Therapeutics plc announced that the FDA has accepted Horizon’s New Drug Application (NDA) for Procysbi (Cysteamine Bitartrate) Delayed-Release Oral Granules in Packets. If approved by the FDA, this new dosage form would provide another option for patients, in addition to the currently available Procysbi delayed-release capsules. The capsules are FDA-approved for children one year of age and older and adults living with nephropathic cystinosis.

The FDA is expected to make a decision on the approval of the proposed new dosage form of granules in packets in 2020. The proposed new dosage form would provide patients and their caregivers with the option of opening a packet containing small, compact granules and sprinkling on certain foods and juice (or administering through a feeding tube). If approved, these packets would provide an alternative to the currently available option of opening individual capsules to remove the granules.

“A major part of our approach at Horizon is to engage with the cystinosis community throughout the year where we listen, learn and find ways to help,” said Gregg Checani, M.D., executive medical director, medical affairs and clinical science, Horizon. “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.”