Decision expands options for eligible patients
NHS England has recommended delayed‑release mercaptamine bitartrate for routine commissioning for eligible patients aged one year and above with nephropathic cystinosis.
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.
Nephropathic cystinosis affects around 1 in 100,000 to 200,000 live births, with roughly 200 people living with the condition in the UK. Two to three new cases are diagnosed each year.
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.”
He added: “Nephropathic cystinosis is a lifelong condition, and this decision marks an important milestone for the cystinosis community in England.”
Care for people with nephropathic cystinosis is often complex and demanding for patients and families. With treatment options previously limited and requiring multiple daily doses, the twice‑daily regimen may help reduce disruption to daily routines, improve adherence, delay complications and support quality of life.
Around 150 patients are expected to be eligible under NHS England’s new commissioning policy. The condition is caused by the accumulation of cystine within cells, leading to tissue and organ damage, particularly in the eyes and kidneys.
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.”
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.”
Delayed‑release mercaptamine bitartrate is approved across the UK and Europe, including France, Italy, Germany and Ireland. Following NHS England’s decision, the medicine is now routinely available across all UK nations for eligible patients meeting local criteria.
Source: Pharma Times
