Raptor Pharmaceutical Corp, a global biopharmaceutical company announced that a US Procysbi patent, number 9,233,077, is now listed in the FDA publication Approved Drug Products with Therapeutic Equivalence Evaluations, commonly known as the Orange Book. The patent, entitled "Delayed Release Cysteamine Bead Formulation, and Methods of Making and Using Same," covers the enteric coated micro-bead formulation of Procysbi as well as other related formulations. This patent expires in June 2034.
Raptor Pharmaceutical now has five patents for Procysbi listed in the Orange Book, including four that issued in the last three months.
In addition, the US Food and Drug Administration (FDA) granted additional orphan drug designation for Procysbi (cysteamine bitartrate) delayed-release capsules to treat nephropathic cystinosis in children aged 2 to 6 years old. The FDA approved the expanded use of Procysbi in this patient population in August 2015 after initially approving Procysbi for the treatment of nephropathic cystinosis in adults and children 6 years and older in April 2013.
Orphan drug designation for Procysbi has been granted post-approval, conferring the benefit of seven years of orphan drug exclusivity for the treatment of nephropathic cystinosis in pediatric patients 2 to 6 years of age. The seven-year term of orphan exclusivity for this recently approved pediatric indication will expire in August 2022. The FDA's Orphan Drug designation programme grants orphan status to drugs intended for the treatment of rare disorders affecting fewer than 200,000 people in the US Orphan drug designation, if requested and subsequently awarded, is typically granted prior to a New Drug Application (NDA) submission, making potential drugs eligible for benefits including tax credits for clinical research costs and a waiver of Prescription Drug User Fee Act (PDUFA) filing fees.
Procysbi is a cystine depleting agent that is approved in the US for the management of nephropathic cystinosis in adults and children ages 2 years and older. It is contraindicated in patients with a hypersensitivity to penicillamine. The most commonly reported side effects are vomiting, abdominal pain/discomfort, headaches, nausea, diarrhea, anorexia/decreased appetite, breath odor, fatigue, dizziness, skin odor and rash.