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# Horizon NDA Accepted for New Procysbi Dosage Form
- URL: https://www.fdaweb.com/horizon-nda-accepted-for-new-procysbi-dosage-form/
- Published: 2019-07-18T12:00:00.000Z
- Updated: 2026-09-15T01:38:40.000Z
- Author: David McFarland
- Tags: Drugs, #legacy-id-D5144568

FDA has accepted for review a Horizon Therapeutics NDA for Procysbi (cysteamine bitartrate) delayed-release oral granules in packets, a new dosage form intended to provide another option for patients. Currently, Procysbi is approved in a delayed-release capsule form for treating patients with nephropathic cystinosis. If approved, the oral granules would provide the option of sprinkling them on certain foods.

Nephropathic cystinosis is a rare, life-threatening metabolic lysosomal storage disorder that causes toxic accumulation of cystine in all cells, tissues, and organs in the body, according to the company. If untreated, elevated cystine leads to progressive, irreversible tissue damage and multi-organ failure, including kidney failure, blindness, muscle wasting and premature death.