NHS England approves delayed-release treatment for rare kidney disease
pharmafile | August 10, 2026 | News story | | NHS England, kidney disease, ultra-rare inherited disorder
NHS England has approved the routine commissioning of delayed-release mercaptamine bitartrate for eligible patients with nephropathic cystinosis, providing an additional treatment option for people living with the ultra-rare inherited disorder.
The decision means eligible patients aged one year and older who meet the commissioning criteria can now access the twice-daily treatment through NHS England specialised services. The medicine is already routinely available in Scotland, Wales and Northern Ireland.
Nephropathic cystinosis affects around 200 people in the UK and is caused by the build-up of cystine within cells, leading to progressive damage to organs including the kidneys and eyes. Approximately 150 patients in England are expected to be eligible for the treatment.
Current therapies often require multiple daily doses, creating a significant treatment burden for patients and carers. The delayed-release formulation is administered every 12 hours and may help improve treatment adherence while reducing disruption to daily life.
David Game, Consultant Nephrologist and Clinical Lead for the commissioning proposal, said: “Nephropathic cystinosis is a lifelong condition, and this decision marks an important milestone for the cystinosis community in England. By providing eligible patients and their clinicians with access to an additional treatment option, it supports treatment decisions based on individual patient needs.”
The decision means delayed-release mercaptamine bitartrate is now routinely available across all four UK nations, subject to local access criteria.
David Garzón, Senior Director, Rare Diseases at Chiesi UK and Ireland, added: “Following earlier reimbursement decisions in Northern Ireland, Wales and Scotland, this decision marks an important step towards more equitable access across the UK, reflecting the collaborative efforts of clinicians, patient organisations, patients and families.”
Patient organisations also welcomed the announcement. Will Newman, Chairperson of Cystinosis Foundation UK, said the approval gives patients and carers greater choice in selecting the treatment that best suits their individual circumstances and lifestyles.
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