The US Food and Drug Administration (FDA) has approved the expanded use of cysteamine bitartrate delayed-release capsules (Procysbi, Raptor Pharmaceuticals) for children as young as 2 years with nephropathic cystinosis, according to a company release.
The FDA initially approved Procysbi in 2013 for nephropathic cystinosis in individuals 6 years of age and older.
Cystinosis is a rare, life-threatening metabolic lysosomal storage disorder that leads to the toxic build-up of cystine in all cells, tissues, and organs in the body. Cystine accumulation leads to progressive, irreversible tissue damage and multiorgan failure, including kidney failure, blindness, muscle wasting, and early death.
Nephropathic cystinosis, the most common and severe form of cystinosis, is usually diagnosed in infancy and requires lifelong therapy, according to the National Organization for Rare Diseases.
The approval of Procysbi in children as young as age 2 years is based on efficacy and safety data from an ongoing long-term extension study of children aged 2 to 6 years treated with the drug for 12 months, the company says.
"Strict adherence to cystine depleting therapy from as early an age as possible is critical to maintaining kidney function and leading longer, healthier lives for patients with cystinosis," Craig Langman, MD, chief of kidney diseases at Ann & Robert H. Lurie Children's Hospital of Chicago in Illinois and lead investigator on the extension trial, said in the release.
"The data in the 2- to 6-year-old group confirm the maintenance of stable kidney function over time. Not only will this help these young patients with cystinosis, but the 12 hour dosing schedule for Procysbi could be a significant benefit to the caregivers of these young children," Dr Langman added.
The most common adverse effects with Procysbi are vomiting, nausea, abdominal pain, breath odor, diarrhea, skin odor, fatigue, rash, and headache. Full prescribing information is available at https://www.procysbi.com/.
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Cite this: FDA OKs Expanded Use of Procysbi for Children as Young as 2 - Medscape - Aug 21, 2015.