Medicines advice

As an adjunct to diet in adult patients with genetically confirmed familial chylomicronaemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate.

As monotherapy in the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have haemolytic anaemia.

As an adjunct to diet in adult patients with genetically confirmed familial chylomicronaemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate.

As monotherapy for the treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with an IDH1 R 132 mutation who were previously treated by at least one prior line of systemic therapy.

As monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least three prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.

• In combination with trametinib for the treatment of paediatric patients aged 1 year and older with low-grade glioma (LGG) with a BRAF V600 mutation who require systemic therapy.
  
  
• In combination with trametinib for the treatment of paediatric patients aged 1 year and older with high-grade glioma (HGG) with a BRAF V600 mutation who have received at least one prior radiation and/or chemotherapy treatment.

As monotherapy is indicated for the treatment of adult patients with relapsed and refractory multiple myeloma (RRMM), who have received at least three prior therapies, including an immunomodulatory agent (IMiD), a proteasome inhibitor (PI), and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.

For treatment of severe and moderately severe haemophilia B (congenital Factor IX deficiency) in adult patients without a history of Factor IX inhibitors.

In combination with bevacizumab for the treatment of adult patients with metastatic colorectal cancer (CRC) who have received two prior anticancer treatment regimens including fluoropyrimidine-, oxaliplatin- and irinotecan-based chemotherapies, anti-VEGF agents, and/or anti-EGFR agents.

Treatment of moderate to severe vasomotor symptoms (VMS) associated with menopause.

Adjuvant treatment of adults and adolescents 12 years of age and older with Stage IIB or IIC melanoma.

As monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least 3 prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.

As monotherapy is indicated for the treatment of adult patients with advanced NSCLC whose tumours have an activating HER2 (ERBB2) mutation and who require systemic therapy following platinum-based chemotherapy with or without immunotherapy.

in a combination regimen with ivacaftor for the treatment of cystic fibrosis (CF) in patients aged 2 years to less than 6 years (granules in sachet) and 6 years and older (film-coated tablets) who have at least one F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

treatment of cystic fibrosis (CF) in patients aged 1 year and older (granules in sachet) or 6 years and older (film-coated tablets) who are homozygous for the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

in a combination regimen with ivacaftor tablets for the treatment of patients with cystic fibrosis (CF) aged 6 years and older who are homozygous for the F508del mutation or who are heterozygous for the F508del mutation and have one of the following mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene: P67L, R117C, L206W, R352Q, A455E, D579G, 711+3A→G, S945L, S977F, R1070W, D1152H, 2789+5G→A, 3272-26A→G, and 3849+10kbC→T.

For the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.

In adults for the treatment of chronic kidney disease.

In combination with trastuzumab, fluoropyrimidine and platinum-containing chemotherapy for the first-line treatment of locally advanced unresectable or metastatic HER2-positive gastric or gastro-oesophageal junction adenocarcinoma in adults whose tumours express PD-L1 with a CPS ≥ 1.

For treatment of advanced (unresectable or metastatic) melanoma in adults and adolescents 12 years of age and over.

For the treatment of invasive candidiasis in adults. Consideration should be given to official guidance on the appropriate use of antifungal agents.

In combination with dexamethasone for the treatment of multiple myeloma in adult patients who have received at least four prior therapies and whose disease is refractory to at least two proteasome inhibitors, two immunomodulatory agents and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy.

In combination with bortezomib and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy

Relugolix is indicated:
- for the treatment of adult patients with advanced hormone-sensitive prostate cancer.
- for the treatment of high-risk localised and locally advanced hormone dependent prostate cancer in combination with radiotherapy.
- as neo-adjuvant treatment prior to radiotherapy in patients with high-risk localised or locally advanced hormone dependent prostate cancer.

As monotherapy for the adjuvant treatment of adults with non-small cell lung carcinoma who are at high risk of recurrence following complete resection and platinum‑based chemotherapy.

For the treatment of adult patients with visual impairment due to macular oedema secondary to branched and central retinal vein occlusion (BRVO and CRVO).

For treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age

Treatment of active moderate to severe hidradenitis suppurativa (HS) in adults with an inadequate response to conventional systemic HS therapy.

Treatment of moderate-to-severe atopic dermatitis in adults and adolescents 12 years and older with a body weight of at least 40 kg who are candidates for systemic therapy.

In combination with standard cytarabine and anthracycline induction and standard cytarabine consolidation chemotherapy, followed by quizartinib single-agent maintenance therapy for adult patients with newly diagnosed acute myeloid leukaemia (AML) that is FMS-like tyrosine kinase 3 internal tandem duplication (FLT3-ITD) positive.

As monotherapy is indicated for the treatment of adult patients with marginal zone lymphoma (MZL) who have received at least one prior anti-CD20-based therapy.

For treatment of advanced Parkinson's disease with severe motor fluctuations and hyperkinesia or dyskinesia when available oral combinations of Parkinson medicinal products have not given satisfactory results

as monotherapy for the treatment of human leukocyte antigen (HLA)-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma

Treatment of patients with molybdenum cofactor deficiency (MoCD) Type A.

In adult women of reproductive age for symptomatic treatment of endometriosis in women with a history of previous medical or surgical treatment for their endometriosis.

For treatment of adult patients with locally advanced or metastatic cholangiocarcinoma with a fibroblast growth factor receptor 2 (FGFR2) fusion or rearrangement that have progressed after at least one prior line of systemic therapy.

For treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 2 months of age and older.

As an add-on to ravulizumab or eculizumab for the treatment of adult patients with paroxysmal nocturnal haemoglobinuria (PNH) who have residual haemolytic anaemia.

Paroxysmal nocturnal hemoglobinuria (PNH), who have haemolytic anaemia.