Chiesi Limited has announced that Procysbi (mercaptamine bitartrate gastro-resistant hard capsules) has been approved for use, in patients with proven nephropathic cystinosis, by the Health Service Executive (HSE) for reimbursement in Ireland.
Nephropathic cystinosis is an ultra-rare, irreversible, progressive, life-long disorder, leading to multiple complications throughout a patient’s lifetime and presenting a heavy burden, causing a reduced quality of life and emotional strain on them and their families.
It also negatively impacts on the patient’s ability to perform everyday functions due to numerous disease-related health and social issues. It can lead to end-stage renal disease and many non-renal complications such as crystal deposition in the conjunctiva. Seldom do patients reach their late 30s without a major, life-altering medical complication caused by the condition. In Ireland, it is estimated that up to 20 patients are affected.
Early and continuous treatment with a cystine-depletion therapy (CDT) represents the mainstay of therapy in patients with nephropathic cystinosis, and whilst a kidney transplant treats end-stage renal failure, CDT must still be taken for life.
Procysbi is a delayed-release formulation of mercaptamine, a CDT, with microgranule technology allowing for drug delivery to the small intestine, extended release and 12-hour dosing, which reduces interruptions for the patient during the day and night (compared to the 6-hourly dosing with immediate-release (IR) mercaptamine formulation).
The HSE’s decision to reimburse Procysbi in Ireland follows a dialogue with Chiesi, and is part of the company’s efforts to expand patient access to an impactful treatment for an ultra-rare condition of high unmet need.
Tom Delahoyde, Managing Director of Chiesi UK and Ireland, said: “This is an encouraging example of how industry, healthcare regulators and patient organisations can collaborate for the benefit of the patient. Chiesi is committed to improving the quality of life for these patients and their families and we will continue to work with the HSE to make this medication available to the patients who need it.”