NHS England has recommended delayed‑release mercaptamine bitartrate for routine commissioning for eligible patients aged one year and above with nephropathic cystinosis. Around 200 people in the UK live with the ultra‑rare condition.
The decision provides access to an additional cystine‑depleting therapy that can be taken twice daily rather than the four‑times‑daily schedule required for the currently commissioned immediate‑release formulation.
Chiesi UK and Ireland said the move expands treatment choice for patients and families.
David Game, Consultant Nephrologist and Clinical Lead for the Delayed‑Release Mercaptamine Policy Proposal, said: “We welcome NHS England’s decision to routinely commission delayed-release mercaptamine bitartrate for eligible people with nephropathic cystinosis.
“Nephropathic cystinosis is a lifelong condition, and this decision marks an important milestone for the cystinosis community in England.”
He added: “By providing eligible patients and their clinicians with access to an additional treatment option, it supports treatment decisions based on individual patient needs and reflects the collaborative efforts of clinicians, patients, families and patient organisations throughout the policy process.”
Nephropathic cystinosis is caused by cystine accumulation within cells, leading to progressive tissue and organ damage, particularly in the eyes and kidneys. Around 150 patients are expected to be eligible under the new commissioning policy.
David Garzón, Senior Director, Rare Diseases at Chiesi UK and Ireland, explained: “This is an important milestone for the nephropathic cystinosis community in England, providing an additional treatment option for these patients with a twice-daily dosing regimen, that can help alleviate some of the treatment burden for patients and their families.”
He 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.”
Delayed‑release mercaptamine bitartrate is approved across the UK and Europe and is now routinely available in all UK nations for eligible patients meeting local criteria.
