Medicines advice

Treatment of arginase 1 deficiency (ARG1-D), also known as hyperargininemia, in adults, adolescents and children aged 2 years and older.

In combination with oxaliplatin, 5-fluorouracil (5-FU) and leucovorin (LV) for the first-line treatment of adult patients with metastatic adenocarcinoma of the pancreas

The acute treatment of the headache phase of migraine attacks with or without aura in adults where treatment with a mono-entity product has been insufficient.

As monotherapy for the treatment of adults and adolescents 12 years and older with advanced rearranged during transfection (RET)-mutant medullary thyroid cancer (MTC).

Treatment of patients aged 12 years and older with acute graft versus host disease who have inadequate response to corticosteroids.

In adults with established or at high risk for atherosclerotic cardiovascular disease to reduce cardiovascular risk by lowering LDL-C levels, as an adjunct to correction of other risk factors:

in adults with established or at high risk for atherosclerotic cardiovascular disease to reduce cardiovascular risk by lowering LDL-C levels, as an adjunct to correction of other risk factors:
• in patients on a maximum tolerated dose of a statin and not adequately controlled with additional ezetimibe treatment or,
• in patients who are either statin-intolerant, or for whom a statin is contraindicated, and not adequately controlled with ezetimibe treatment or,
• in patients already being treated with the combination of bempedoic acid and ezetimibe as separate tablets.

Treatment of adult patients with relapsing forms of multiple sclerosis (RMS) with active disease as defined by clinical or imaging features.

In adults for treatment of neovascular (wet) age-related macular degeneration (nAMD)

In combination with tremelimumab for the first line treatment of adults with advanced or unresectable hepatocellular carcinoma.

Add-on treatment for severe refractory eosinophilic asthma in adults, adolescents and children aged 6 years and older.

As monotherapy for the treatment of adult patients with metastatic colorectal cancer (mCRC) who have been previously treated with available therapies, including fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, with or without an anti-vascular endothelial growth factor (VEGF) therapy and, if rat sarcomas virus (RAS) wildtype and medically appropriate, an anti-epidermal growth factor receptor (EGFR) therapy.

As monotherapy for the treatment of adult patients with unresectable or metastatic urothelial carcinoma (UC), harbouring susceptible fibroblast growth factor receptor 3 (FGFR3) genetic alterations who have previously received at least one line of therapy containing a programmed death receptor-1 (PD-1) or programmed death-ligand 1 (PD-(L)1) inhibitor in the unresectable or metastatic treatment setting.

For the prevention of ototoxicity caused by cisplatin chemotherapy in patients 1 month to < 18 years of age with localised, non-metastatic, solid tumours.

Treatment of polymyalgia rheumatica (PMR) in adult patients who have had an inadequate response to corticosteroids or who experience a relapse during corticosteroid taper.

Treatment of human immunodeficiency virus-1 (HIV-1) infection without any known mutations associated with resistance to the integrase inhibitor class, emtricitabine or tenofovir in paediatric patients aged from 2 years and with body weight at least 14 kg to less than 25 kg.

for the treatment of mild cognitive impairment (MCI) and mild dementia due to Alzheimer’s disease (AD) in adult patients that are apolipoprotein E ε4 (APOE4) heterozygotes on non-carriers.

For the treatment of transfusion-dependent β-thalassaemia in patients 12 years of age and older for whom haematopoietic stem cell transplantation is appropriate and a human leukocyte antigen (HLA)-matched related haematopoietic stem cell (HSC) donor is not available.

Treatment of COVID-19 in adults who do not require supplemental oxygen and who are at increased risk for progression to severe COVID-19.

Treatment of mild to moderate coronavirus disease 2019 (COVID-19) in adults with a positive SARS-COV-2 diagnostic test and who have at least one risk factor for developing severe illness.

For treatment of mild cognitive impairment and mild dementia due to Alzheimer’s disease in adult patients that are apolipoprotein E ɛ 4 (ApoEɛ4) heterozygotes or non-carriers.

As monotherapy for the treatment of adults and adolescents 12 years and older with advanced RET fusion-positive thyroid cancer (TC) who are radioactive iodine-refractory (if radioactive iodine is appropriate).

In combination with pemetrexed and platinum-based chemotherapy for the first-line treatment of adult patients with advanced NSCLC whose tumours have EGFR exon 19
deletions or exon 21 (L858R) substitution mutations.

Treatment of adult patients with previously untreated cell membrane receptor 30-positive (CD30+) Stage III or IV Hodgkin lymphoma (HL) in combination with doxorubicin, vinblastine and dacarbazine (AVD).

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

In combination with carboplatin and pemetrexed for the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with activating epidermal growth factor receptor (EGFR) Exon20 insertion mutations.

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

Treatment of osteoporosis in postmenopausal women at increased risk of fracture.

In combination with carboplatin and paclitaxel, is indicated for the first-line treatment of primary advanced or recurrent endometrial carcinoma in adults.

In combination with an aromatase inhibitor is indicated for the adjuvant treatment of patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative early breast cancer at high risk of recurrence.s

In pre- or perimenopausal women, or in men, the aromatase inhibitor should be combined with a luteinising hormone-releasing hormone (LHRH) agonist.

In combination with bortezomib, lenalidomide, and dexamethasone, for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant (ASCT).

Treatment of postmenopausal women, and men, with estrogen receptor (ER)-positive, HER2-negative, locally advanced or metastatic breast cancer with an activating ESR1 mutation who have disease progression following at least one line of endocrine therapy including a CDK 4/6 inhibitor.

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

Treatment of adult patients with Philadelphia chromosome (Ph)-negative, CD19-positive, B-cell precursor acute lymphoblastic leukaemia (ALL) in the consolidation phase.

In combination with carboplatin and paclitaxel for the first-line treatment of adults with primary advanced or recurrent endometrial cancer who are candidates for systemic therapy, followed by maintenance treatment with:

• durvalumab as monotherapy in endometrial cancer that is mismatch repair deficient (dMMR)
• durvalumab in combination with olaparib in endometrial cancer that ismismatch repair proficient (pMMR).

In adults as add-on maintenance treatment for uncontrolled chronic obstructive pulmonary disease (COPD) characterised by raised blood eosinophils on a combination of an inhaled corticosteroid (ICS), a long-acting beta2-agonist (LABA), and a long-acting muscarinic antagonist (LAMA), or on a combination of a LABA and a LAMA if ICS is not appropriate.

Treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC) with disease progression on or after at least two prior lines of therapy including platinum-based chemotherapy.

Treatment of moderate to severe chronic hand eczema (CHE) in adults for whom topical corticosteroids (TCS) are inadequate or inappropriate.

In combination with ipilimumab (YERVOY®) (NIVO + IPI) is indicated for the treatment of adult patients with mismatch repair deficient (dMMR) or microsatellite instability high (MSI-H) colorectal cancer (CRC) in the following settings: first-line treatment of unresectable or metastatic CRC.

For routine prophylaxis of bleeding episodes in patients 12 years of age and older, weighing at least 35 kg, with:

• severe haemophilia A (congenital factor VIII deficiency, FVIII < 1%)
  without factor VIII inhibitors, or
• severe haemophilia B (congenital factor IX deficiency, FIX < 1%) without
  factor IX inhibitors