Key Takeaways
- Joenja has secured reimbursement in England for the treatment of people aged 12 years and over with the ultra-rare immune disorder, activated phosphoinositide 3-kinase delta syndrome.
- NICE, England’s health technology institute, said that Pharming had addressed its uncertainties about the product that had previously led to it rejecting the drug.
- It was able to apply quality-adjusted life year (QALY) weighting that increased the threshold at which the treatment could be considered a cost-effective use of National Health Service resources, from £100,000 per QALY to around £180,000.
- Joenja, which is approved for marketing in the UK, the US and Israel, is experiencing a protracted regulatory review in the EU.
- Marketing applications are under review in other countries.
England has become the first European country to agree to reimburse Joenja (leniolisib), Pharming’s ultra-rare immune disorder drug, which received UK marketing approval last September but is undergoing a protracted...
Joenja, for treating people aged 12 years and over with activated phosphoinositide 3-kinase delta syndrome (APDS), was recommended for use via England’s National Health Service...
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