Medicines advice

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 treatment of partial thickness wounds associated with dystrophic and junctional epidermolysis bullosa (EB) in patients aged 6 months and older.

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

Long-term enzyme replacement therapy (ERT) in adult patients with confirmed diagnosis of Fabry disease (FD) (deficiency of alpha galactosidase).

Controlled ovarian stimulation for the development of multiple follicles in women undergoing assisted reproductive technologies (ART) such as an in vitro fertilisation (IVF) or intracytoplasmic sperm injection (ICSI) cycle.

Film-coated tablets: in combination with other antiretroviral(s) for the treatment of adults with multidrug resistant HIV-1 infection for whom it is otherwise not possible to construct a suppressive anti-viral regimen, for oral loading prior to administration of long-acting lenacapavir injection.

Solution for injection: in combination with other antiretroviral(s) for the treatment of adults with multidrug resistant HIV-1 infection for whom it is otherwise not possible to construct a suppressive anti-viral regimen.

In adults for intravenous induction and maintenance of general anaesthesia.

As monotherapy for the treatment of adult patients with advanced HER2-positive gastric or gastroesophageal junction (GEJ) adenocarcinoma who have received a prior trastuzumab-based regimen.

As monotherapy for the treatment of adult patients with relapsed or refractory diffuse large B‑cell lymphoma (DLBCL), after two or more lines of systemic therapy.

Treatment of haemolytic anaemia due to sickle cell disease (SCD) in adults and paediatric patients 12 years of age and older as monotherapy or in combination with hydroxycarbamide

As monotherapy for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy

For weight management, including weight loss and weight maintenance, as an adjunct to a reduced-calorie diet and increased physical activity in adults with an initial body mass index (BMI) of:

• ≥30 kg/m2 (obesity) or
  
  
• ≥27 kg/m2 to <30 kg/m2 (overweight) in the presence of at least one weight-related comorbid condition (e.g., hypertension, dyslipidaemia, obstructive sleep apnoea [OSA], cardiovascular disease [CVD], prediabetes, or type 2 diabetes mellitus [T2DM])

For the treatment of patients 16 years of age and older with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response, or were intolerant to either conventional therapy, or a biological agent.

For the treatment of disease-related splenomegaly or symptoms in adult patients with moderate to severe anaemia who have primary myelofibrosis, post polycythaemia vera myelofibrosis or post essential thrombocythaemia myelofibrosis and who are Janus Associated Kinase (JAK) inhibitor naïve or have been treated with ruxolitinib.

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 patients with molybdenum cofactor deficiency (MoCD) Type A.

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.

• 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.

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.

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

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

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

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.

Treatment of adult patients with advanced hormone-sensitive prostate cancer.