Medicines advice

In combination with pemetrexed and platinum chemotherapy for the first‑line treatment of adults with unresectable non-epithelioid malignant pleural mesothelioma.

For the treatment of Friedreich’s ataxia (FA) in adults and adolescents aged 16 years and older.

In combination with pemetrexed and platinum chemotherapy for the first‑line treatment of adults with unresectable non-epithelioid malignant pleural mesothelioma

As monotherapy for the treatment of adult patients with unresectable or metastatic hormone receptor (HR)-positive, HER2-negative breast cancer who have received endocrine-based therapy, and at least two additional systemic therapies in the advanced setting.

Treatment of primary biliary cholangitis (PBC), including pruritus, in adults in combination with ursodeoxycholic acid (UDCA) who have an inadequate response to UDCA alone, or as monotherapy in those unable to tolerate UDCA.

For the treatment of cystic fibrosis (CF) in people aged 6 years and older who have at least one F508del mutation or another responsive mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene.

For the treatment of grade 2 astrocytoma or oligodendroglioma with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation or isocitrate dehydrogenase-2 (IDH2) mutation in adults and paediatric patients 12 years and older, who are not in need of immediate chemotherapy or radiotherapy following surgical intervention.

As an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti‑acetylcholine receptor (AChR) antibody positive.

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.

In combination with other pulmonary arterial hypertension (PAH) therapies for the treatment of PAH in adult patients with WHO Functional Class (FC) II to III, to improve exercise capacity.

For the treatment of moderate or severe postnatal depression (PND) in adults following childbirth.

Treatment of moderate to severe dry eye disease (keratoconjuctivitis sicca) in adult patients, which has not improved despite treatment with tear substitutes.

Topical treatment of acne vulgaris in patients 12 years of age and older

As monotherapy for the treatment of adult patients with smouldering multiple myeloma at high risk of developing multiple myeloma

Treatment of chronic spontaneous urticaria (CSU) in patients aged 12 years and older whose disease is not adequately controlled with H1 antihistamine treatment.

Treatment of sickle cell disease (SCD) in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs) who have βS/βS, βS/β0 or βS/β+, for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA)-matched related HSC donor is not available.

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

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.

As monotherapy for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) who have been previously treated with a Bruton's tyrosine kinase (BTK) inhibitor

In combination with carboplatin and etoposide for the first‑line treatment of adult patients with extensive‑stage small cell lung cancer (ES‑SCLC).

In combination with lazertinib for the first‑line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) Exon 19 deletions or Exon 21 L858R substitution mutations.

In combination with platinum-containing chemotherapy for the treatment of adult patients with primary advanced or recurrent endometrial cancer (EC) and who are candidates for systemic therapy.

In combination with pembrolizumab for the first-line treatment of adult patients with unresectable or metastatic urothelial cancer who are eligible for platinum-containing chemotherapy.

Treatment of adults with moderate-to-severe prurigo nodularis (PN) who are candidates for systemic therapy.

Treatment of moderate-to-severe atopic dermatitis (AD) in combination with topical corticosteroids and/or calcineurin inhibitors in adults and adolescents 12 years and older with a body weight of at least 30 kg, who are candidates for systemic therapy.

In combination with venetoclax with or without obinutuzumab for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL).

For treatment of adult patients with locally advanced, unresectable NSCLC whose tumours have EGFR exon 19 deletions or exon 21 (L858R) substitution mutations and whose disease has not progressed during or following platinum‑based chemoradiation therapy.

In combination with chemoradiotherapy (external beam radiation therapy followed by brachytherapy), is indicated for the treatment of FIGO 2014 Stage III - IVA locally advanced cervical cancer in adults who have not received prior definitive therapy.

Treatment of Duchenne muscular dystrophy resulting from a nonsense mutation in the dystrophin gene, in ambulatory patients aged 2 years and older.

The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.

Treatment of metachromatic leukodystrophy characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity:

• in children with late infantile or early juvenile forms, without clinical manifestations of the disease
• in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline

As monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN) or mast cell leukaemia (MCL).

Add-on treatment for adult patients with relapsing or refractory eosinophilic granulomatosis with polyangiitis.

In adults and children (5 years or older) for the treatment of moderate-to-severe allergic rhinitis and/or conjunctivitis induced by pollen from the birch homologous group. It is indicated in patients with a clinical history of symptoms despite use of symptom-relieving medication and a positive test of sensitisation to a member of the birch homologous group (skin prick test and/or specific IgE).

For treatment of adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.8 g/g).

Treatment of adult patients with unresectable or metastatic, well differentiated extra-pancreatic (epNET) and pancreatic (pNET) neuroendocrine tumours who have progressed following at least one prior systemic therapy
other than somatostatin analogues.

For the treatment of peripheral neuropathic pain in diabetic adults either alone or in combination with other medicinal products for pain.

As monotherapy for the first-line treatment of adult patients with non-small cell lung cancer (NSCLC) expressing PD-L1 (in ≥ 50% tumour cells), with no EGFR, ALK or ROS1 aberrations, who have:

• locally advanced NSCLC who are not candidates for definitive chemoradiation, or
• metastatic NSCLC

In combination with platinum‐based chemotherapy for the first‐line treatment of adult patients with NSCLC expressing PD-L1 (in ≥ 1% of tumour cells), with no EGFR, ALK or ROS1 aberrations, who have:

• locally advanced NSCLC who are not candidates for definitive chemoradiation, or
• metastatic NSCLC

Adult patients (18-65 years) diagnosed by clinical history and a positive test of house dust mite sensitisation (skin prick test and/or specific IgE) with at least one of the following conditions:

• persistent moderate to severe house dust mite allergic rhinitis despite use of symptom-relieving medication
• house dust mite allergic asthma not well controlled by inhaled corticosteroids and associated with mild to severe house dust mite allergic rhinitis. Patients' asthma status should be carefully evaluated before the initiation of treatment

Adolescents (12-17 years) diagnosed by clinical history and a positive test of house dust mite sensitisation (skin prick test and/or specific IgE) with persistent moderate to severe house dust mite allergic rhinitis despite use of symptom-relieving medication.

In combination with bortezomib, lenalidomide and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma