Medicines advice

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 fluoropyrimidine- and platinum-containing chemotherapy, for the first-line treatment of adult patients with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-negative gastric or gastro-oesophageal junction (GEJ) adenocarcinoma whose tumours are Claudin (CLDN) 18.2 positive.

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) Exon 20 insertion mutations

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

For the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older

Treatment of adult patients with complement 3 glomerulopathy (C3G) in combination with a renin-angiotensin system (RAS) inhibitor, or in patients who are RAS-inhibitor intolerant, or for whom a RAS inhibitor is contraindicated

Treatment of activated phosphoinositide 3-kinase delta (P13Kδ) syndrome (APDS) in adult and paediatric patients 12 years of age and older.

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

Prevention of miscarriage in women presenting with bleeding in the first trimester of pregnancy and have a history of recurrent miscarriage.

Monotherapy for the treatment of adult patients with unresectable or metastatic hormone receptor (HR)-positive, HER2-low or HER2-ultralow breast cancer who have received at least one endocrine therapy in the metastatic setting and who are not considered suitable for endocrine therapy as the next line of treatment

Short-term treatment of mild to moderate acute ulcerative colitis limited to the rectum (ulcerative proctitis) in adult patients.

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

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

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

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 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.

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

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.

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.

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.