Patients in England with ultra-rare kidney disease nephropathic cystinosis will soon be able to access a therapy that has been available in other UK nations for some time.
NHS England has given the green light to routine commissioning of Chiesi’s Procysbi (delayed-release mercaptamine bitartrate), more than three years after it was first made available in Wales and months after Scotland and Northern Ireland backed use of the drug.
Until now, the recommended treatment for patients with cystinosis in England has been Recordati’s Cystagon (cysteamine), which has to be taken every six hours, including a dose in the middle of the night.
In contrast, Procysbi only needs to be taken every 12 hours, making it much easier to fit into patients’ daily lives and allowing a full night’s sleep. NHS England’s Clinical Priorities Advisory Group (CPAG) assessment panel has now said that Chiesi’s drug can be used for patients aged one year and above who meet certain clinical criteria, for example if they are struggling to cope with their current treatment.
Cystinosis is an inherited, lifelong genetic disease that causes an amino acid called cystine to build up and form damaging crystals in the body’s cells, organs, and tissues, and both Cystagon and Procysbi are designed to deplete the excess cystine levels in the body.
The disorder most severely affects the kidneys and eyes, and occurs in roughly one in 100,000 to 200,000 live births, with around 200 diagnosed cases in the UK. Around 150 of them are expected to be eligible for Chiesi’s drug under the new commissioning policy.
The decision comes after more than three years of dedicated campaigning by patient organisations Kidney Research UK, Cystinosis Foundation UK, and Metabolic Support UK for access to Procysbi.
“We are delighted to see this decision, which represents an important step forward for the nephropathic cystinosis community in England,” commented Will Newman, chairperson of Cystinosis Foundation UK. “The approval means patients and carers can now explore whether this formulation better suits their individual circumstances, lifestyles, and tolerability needs.”
He also said it had been a “tortuous process” bringing it about, adding: “Some of our patients living with cystinosis have not had a full night’s sleep for over 40 years.”
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