NHS Backs Chiesi’s Delayed-Release Cystinosis Therapy

NHS England has recommended Chiesi’s delayed-release mercaptamine bitartrate for routine commissioning in the treatment of nephropathic cystinosis, an ultra-rare condition affecting approximately 200 people in the UK. Announced on 12 August 2026, the decision opens access to a twice-daily cystine-depleting therapy for around 150 eligible patients aged one year and above, replacing the four-times-daily schedule required for existing immediate-release therapy and aligning English patients with Scotland, Wales and Northern Ireland.

Nephropathic cystinosis is caused by cystine accumulation within cells, leading to progressive tissue and organ damage, particularly affecting the eyes and kidneys. The condition is lifelong, requires strict adherence to cystine-depleting therapy and, until now in England, has typically been managed with immediate-release mercaptamine on a four-times-daily schedule. The delayed-release formulation offers twice-daily dosing and is expected to reduce daily treatment burden for patients, families and caregivers. 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.

David Game, Consultant Nephrologist and Clinical Lead for the Delayed-Release Mercaptamine Policy Proposal, framed the decision as an important milestone for the cystinosis community, supporting treatment decisions based on individual patient needs. David Garzón, Senior Director for Rare Diseases at Chiesi UK and Ireland, positioned the decision as an important step towards more equitable UK access, following earlier reimbursement decisions in Northern Ireland, Wales and Scotland. The commissioning decision expands Chiesi’s UK rare disease franchise into a genuinely ultra-rare category, positioning it alongside Recordati (Cystadrops for ocular cystinosis) and older immediate-release products in the broader cystinosis treatment landscape.

The commercial signal is a growing UK NHS emphasis on treatment burden reduction alongside pure clinical efficacy in rare disease commissioning decisions. Reformulated therapies with simplified dosing schedules increasingly succeed in NHS reimbursement pathways. Expect other rare disease specialists including Recordati, Alnylam, Ultragenyx and BioMarin to sharpen their positioning around adherence and treatment burden as they engage with NHS England commissioning.

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