Type of information: Granting of a Market Authorisation in the US
Product name: Procysbi®
Therapeutic area: Rare diseases
- Procysbi® is a cystine depleting agent. It was engineered to bypass absorption in the stomach with an extended terminal half-life so that patients experience steady drug levels in their bodies for the full 12-hour dosing period.
Company: Raptor Pharmaceuticals Europe, now Horizon Pharma (Ireland)
Disease: nephropathic cystinosis
- • On December 27, 2017, Horizon Pharma announced the FDA has approved an expansion to the indication for Procysbi® (cysteamine bitartrate) delayed-release capsules to include children one year of age and older living with nephropathic cystinosis. With this update to the indication, the Procysbi® prescribing information now includes revised guidance for physicians administering the medicine to pediatric patients, including new clinical evidence and dosing information for very young children. Procysbi® was previously FDA-approved for adults and children as young as two years of age.
- The Procysbi® labeling was updated based on a long-term, prospective, open-label study that enrolled 17 people living with nephropathic cystinosis, including 15 children between the ages of 1 and 5 years old, who had not previously been treated with cysteamine therapy. Children enrolled in the study experienced lowering of white blood cell (WBC) cystine levels from poor controlled to well controlled at 12 and 18 month measurements. WBC levels are the biomarker for disease control. Additionally, they experienced measured improvements in growth milestones including weight and height. The most common adverse reactions (>10%) in patients treated in clinical trials reflected in the FDA approved product labeling were vomiting, gastroenteritis/viral gastroenteritis, diarrhea, breath odor, nausea, electrolyte imbalance and headache. This study was required by the FDA as a post-marketing commitment after Procysbi® was approved.
- • On September 12, 2013, Raptor Pharmaceutical has announced that the European Commission has approved Procysbi® gastro-resistant hard capsules of cysteamine (as mercaptamine bitartrate) as an orphan medicinal product for the treatment of proven nephropathic cystinosis for marketing in the European Union (EU). The positive recommendation by the European Medicines Agency's (EMA) Committee for Medicinal Products for Human Use (CHMP) has been adopted in June 2013.
- The EC approval of Procysbi® was based on data from six clinical trials, including a multi-center randomized, active-controlled Phase 3 trial of 43 patients with nephropathic cystinosis and extension data from that trial. The CHMP concluded that the submitted study data support clinical efficacy of Procysbi® similar to that of immediate-release cysteamine with a dosing schedule that may promote a better compliance.
- It is contraindicated in patients with a hypersensitivity to any form of cysteamine (mercaptamine) or any of Procysbi®'s excipients, penicillamine and for breast-feeding women.
- • On 27 June 2013, the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending the granting of a marketing authorisation for the medicinal product Procysbi®,25 mg, 75 mg, gastro-resistant capsules, hard intended for the treatment of nephropathic cystinosis. Procysbi® was designated as an orphan medicinal product on 20 September 2010.
- The benefits with Procysbi® are its ability to reduce cystine accumulation in some cells (e.g. leukocytes, muscle and liver cells) of nephropathic cystinosis patients and, when treatment is started early, it delays the development of renal failure. The most common side effects are gastrointestinal disorders such as vomiting, nausea, diarrhoea. Other very common side effects are: anorexia, lethargy, pyrexia. A pharmacovigilance plan for Procysbi® will be implemented as part of the marketing authorisation.
Submission of marketing authorization application USA :
Submission of marketing authorization application UE:
Withdrawal of marketing authorization application USA:
Withdrawal of marketing authorization application UE:
US authorization: 2013-04-30/2017-12-27
UE authorization: 2013-09-12
Favourable opinion UE: 2013-06-27
Favourable opinion USA:
Orphan status USA:
Orphan status UE: 2010-09-20
Pediatric exclusivit _USA:
Pediatric exclusivity UE: