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Cuprior (trientine)

£9,958.87
Cuprior (trientine) is a medication indicated for patients aged 5 years and older with Wilson’s disease.
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Libtayo (cemiplimab)

£8,328.00
Libtayo (cemiplimab) is a medication used for the treatment of advanced cutaneous squamous cell carcinoma (CSCC), advanced basal cell carcinoma (BCC) and advanced non-small cell lung cancer (NSCLC).
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Mayzent (Siponimod)

Mayzent (siponimod) is a medication used for the treatment of multiple sclerosis (MS).
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Palynziq (pegvaliase-pqpz)

,
£293.61 – £2,761.67
Palynziq (pegvaliase-pqpz) is indicated for the treatment of phenylketonuria (PKU), to reduce blood phenylalanine concentrations in adult and adolescent patients from 16 years of age who have uncontrolled blood phenylalanine concentrations greater than 600 micromol/L on existing management.
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Piqray (alpelisib)

£6,748.65
Piqray (alpelisib) is a medication used in combination with fulvestrant for the treatment of postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, PIK3CAmutated, advanced or metastatic breast cancer as detected by an FDA-approved test following progression on or after an endocrine-based regimen.
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Rivfloza (nedosiran)

Rivfloza (nedosiran) is a prescription medicine for the treatment of children (9+) and adults with primary hyperoxaluria type 1 (PH1) and relatively preserved kidney function1. Rivfloza (nedosiran) is approved by the FDA in the USA 2.
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Sohonos

Sohonos (palovarotene) is a prescription medicine for the treatment of fibrodysplasia ossificans progressiva (FOP). The medicine is indicated for female patients aged 8 years and older, and male patients aged 10 years and older1. Sohonos (palovarotene) is approved by the FDA in the USA2.
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Tecfidera (dimethyl fumarate)

£1,168.56
Tecfidera (dimethyl fumarate) is a medication used for the treatment of multiple sclerosis (MS).
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Tibsovo (ivosidenib)

£27,631.80
Tibosovo (ivosidenib) is a prescription medicine used to treat adults with an IDH1 mutation who have:
  • Acute myeloid leukemia (AML) that is either newly-diagnosed or relapsed/refractory, or
  • Locally advanced or metastatic bile duct cancer (cholangiocarcinoma)
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Tudcabil (tauroursodeoxycholic acid)

£22.30
Tudcabil (tauroursodeoxycholic acid) is a medication granted orphan designation by the EMA for amyotrophic lateral sclerosis (ALS).
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Veopoz (pozelimab-bbfg)

Veopoz (pozelimab-bbfg) is a medicine prescribed for the treatment of children older than 1 year and adults with CHAPLE disease1. Veopoz (pozelimab-bbfg) is the first FDA-approved treatment for CHAPLE disease2.
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Xphozah (tenapanor)

Xphozah (tenapanor) is a prescription medicine for the treatment of adult patients with chronic kidney disease (CKD) 1. Xphpzah (tenapanor) is approved by the FDA in the USA 2
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