Chiesi Limited stated that the AWMSG has recommended Procysbi (mercaptamine bitartrate gastro-resistant hard capsules) as an option for use within NHS Wales for the treatment of patients with proven nephropathic cystinosis.
Key highlights:
- The All Wales Medicines Strategy Group (AWMSG) is the first health technology assessor in the UK to recommend Procysbi, which will now be available on the NHS in Wales.
- Procysbi is a twice-daily delayed-release formulation of mercaptamine with 12-hour dosing, which reduces cystine accumulation in some cells of patients with nephropathic cystinosis and, when treatment is started early, it delays the development of kidney failure.
Procysbi was considered as an ultra-orphan medicine according to the criteria in the AWMSG appraisal process for a medicine for a rare disease. Procysbi is now reimbursed in Wales, but not in England, Scotland or Northern Ireland.
Nephropathic cystinosis is a rare, lysosomal storage disorder that causes progressive damage throughout the body. It causes multiple organ dysfunction due to the accumulation of cystine in the tissues, leading to complications including kidney failure, diabetes, hypothyroidism, myopathy, and central nervous system deterioration. Sustained treatment with mercaptamine (a cystine-depleting agent) is essential to delay end-stage kidney disease and increase life expectancy. Cystinosis is estimated to affect up to 1 in 100,000 births, equating to fewer than 40 people in Wales.
Before Procysbi was recommended by AWMSG, the only routinely available treatment for patients with nephropathic cystinosis in the UK was immediate-release (IR) mercaptamine (Cystagon). Adherence to IR-mercaptamine can be challenging, especially in adolescents and adults, due to the 6-hourly dosing schedule that requires night-time administration and its association with halitosis (bad breath). Procysbi is a delayed-release formulation of mercaptamine with twice-daily 12-hour dosing, which reduces the administration burden for patient and carers when compared with a 6-hourly IR-mercaptamine regimen.
“We are extremely pleased that patients in Wales with this devastating condition have the opportunity to access Procysbi,” said Sarah O’Connell, head of rare diseases, Chiesi Limited.
“Chiesi is committed to discovering, developing and commercialising innovative therapies to address unmet needs for people living with rare diseases. We look forward to working with NHS England and NHS Scotland in future HTA assessments of Procysbi.”