Posted from: Tuesday, February 06, 2018 - 12:51 PM - Present

Procysbi Receives Expanded Indication

December 27, 2017 – The U.S. FDA has expanded the indication for Procysbi® (cysteamine bitartrate), manufactured by Horizon Pharmaceuticals, to treat children as young as one year old who have been diagnosed with nephropathic cystinosis.

A rare hereditary disease, nephropathic cystinosis affects an estimated 500 individuals in America. The disorder interferes with the body’s ability to remove cystine, an amino acid, from lysosomes. Lysosomes make up part of the human cell, and are responsible for breaking down certain molecules. The excess cystine crystalizes inside of the cell, leading to tissue and organ damage. In both the infantile form and late-onset form of the disorder, renal failure will occur if the disease is left untreated.

Recommended dosing with Procysbi is based on the patient’s treatment history, and dosage is titrated until the target white blood cell cystine concentration is reached. Patients should take the capsules whole if possible, but the capsules can be broken open and mixed with certain foods or liquids if necessary. Procysbi is available in both 25mg and 75mg dosage forms.


Adverse reactions or quality problems experienced with the use of a recalled product may be reported to the FDA’s MedWatch Adverse Event Reporting Program either online, by U.S. mail or by fax.

Actual drug patent expiration dates and availability of new medications are subject to change due to patent litigation, settlement agreements, additional patents, exclusivities, and final FDA approval.  Distribution and availability of new medications at pharmacies may not occur immediately following FDA approval.  Patients are advised to speak with their healthcare professional or pharmacist regarding appropriateness as well as actual availability. 

*This is provided for information only.  The reference to any medication above does not mean the medication is covered by your plan.

Last Updated Wednesday, November 13, 2019 - 02:52 AM.