Scottish Medicines Consortium Approves Procysbi for NHSScotland Use, Offering New Hope and Choice for Cystinosis Patients

The Scottish Medicines Consortium (SMC) has today accepted mercaptamine (Procysbi) for use within NHSScotland, following a third resubmission assessed under the orphan equivalent medicine process. This landmark decision marks a pivotal moment for individuals living with nephropathic cystinosis and their families, as it introduces long-awaited choice in primary treatment in Scotland for the first time.

Procysbi, a 12-hourly delayed-release formulation of mercaptamine, offers an alternative to immediate-release cysteamine – known as Cystagon – which has been the sole treatment option for decades. The approval means patients and carers can now explore whether this formulation better suits their individual circumstances, lifestyles, and tolerability needs.

“This is a transformative moment for our community,” said Alex Hutchison, a Scotland-based Trustee from Cystinosis Foundation UK. “For years, Scottish patients have had no choice in how their condition is managed. The acceptance of Procysbi not only validates the lived experience of those who advocated for its inclusion, but also empowers families to make treatment decisions that reflect their realities. We can only hope that this helps neighbouring countries in gaining acceptance for use in their area, given that the Republic of Ireland, Wales and now Scotland’s governance bodies have all approved usage.”

Laura Smith van Carroll, Head of Insight & Advocacy at Metabolic Support UK, commented: “As an organisation founded by a family whose child was diagnosed with cystinosis, we understand firsthand the challenges that come with managing this rare and complex condition. The approval of Procysbi in Scotland marks a significant milestone for the cystinosis community, offering treatment choice, as well as the potential for improved quality of life of both those living with and caring for. We would like to thank everyone who participated in and helped disseminate the cystinosis survey, which was instrumental in providing SMC with clear, reliable evidence of the lived experience.”

“This is great news for people living with this rare disease and their families.” explained Alison Railton, Director of Policy and Public Affairs at Kidney Research UK. “Having access to this treatment could make a meaningful difference, particularly for children and their parents. The twice-daily dosing means families no longer need to wake during the night, supporting a more consistent night’s sleep. We believe everyone living with cystinosis should have access to the most effective treatments available to prevent them from developing kidney disease and eventually needing dialysis or a kidney transplant.”

The decision follows a long SMC process covering a total of 4 submissions over a period of 8 years.

For people living with cystinosis, this means the possibility of improved adherence, reduced side effects, and a treatment schedule that may better align with daily life – particularly for children, young people, and working adults whose routines are disrupted by frequent dosing.

The acceptance of Procysbi is a testament to the persistence of patient advocates, clinicians, and researchers who have worked tirelessly to ensure that choice, dignity, and quality of life are central to cystinosis care in Scotland.

Cystinosis Foundation UK were extremely fortunate to partner with Metabolic Support UK and Kidney Research UK to collaboratively write a Patient Group submission, and to prepare for and contribute to the Patient and Clinician Engagement (PACE) meeting. We extend our heartfelt thanks to the two patient and carer voices who attended the PACE meeting — their stories made a profound difference to this outcome.