Medicines advice

For the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older.

As monotherapy for the treatment of adult and paediatric patients 12 years of age or older with a weight of 40 kg and above with paroxysmal nocturnal haemoglobinuria (PNH):

• In patients with haemolysis with clinical symptom(s) indicative of high disease activity.
• In patients who are clinically stable after having been treated with a complement component 5 (C5) inhibitor for at least the past 6 months.

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

Treatment of adult patients with moderately to severely active ulcerative colitis who have had an inadequate response to, lost response to, or were intolerant to conventional therapy or a biologic therapy.

Treatment of moderate-to-severe Dry Eye Disease (keratoconjunctivitis sicca) in adult patients who have not responded adequately to artificial tears.

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

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

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.

Treatment of human immunodeficiency virus-1 (HIV-1) infection in paediatric patients at least 2 years of age and weighing at least 14 kg to less than 25kg without present or past evidence of viral resistance to the integrase inhibitor class, emtricitabine or tenofovir

As an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti-acetylcholine receptor (AChR) or anti-muscle-specific tyrosine kinase (MuSK) antibody positive

For the treatment of adult and paediatric patients aged 6 and above with facial angiofibroma (facial AF) associated with tuberous sclerosis complex (TSC).

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

In combination with platinum-based chemotherapy as neoadjuvant treatment, followed by durvalumab as monotherapy after surgery, is indicated for the treatment of adults with resectable (tumours ≥ 4 cm and/or node-positive) NSCLC and no known EGFR mutations or ALK rearrangements.

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.

Symptomatic treatment of adult patients with overactive bladder (OAB) syndrome.

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

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

In combination with platinum-containing chemotherapy as neoadjuvant treatment, and then continued as monotherapy as adjuvant treatment, for the treatment of resectable non‑small cell lung carcinoma at high risk of recurrence in adults.

Treatment of adult patients with diffuse large B‑cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL) that relapses within 12 months from completion of, or is refractory to, first-line chemoimmunotherapy.

Monotherapy for the treatment of adult patients with germline BRCA1/2-mutations, who have HER2−negative locally advanced or metastatic breast cancer (mBC). Patients should have previously been treated with an anthracycline and a taxane in the (neo)adjuvant or metastatic setting unless patients were not suitable for these treatments. Patients with HR+ breast cancer should also have progressed on or after prior endocrine therapy, or be considered unsuitable for endocrine therapy.

Reduction of elevated intraocular pressure (IOP) in adult patients with primary open-angle glaucoma or ocular hypertension (OHT) for whom monotherapy with a prostaglandin or netarsudil provides insufficient IOP reduction.

As monotherapy for the treatment of adult patients with recurrent or metastatic cervical cancer and disease progression on or after platinum-based chemotherapy.

In combination with safer sex practices for pre-exposure prophylaxis (PrEP) to reduce the risk of sexually acquired human immunodeficiency-1 (HIV-1) infection in high-risk adults and adolescents, weighing at least 35 kg.

Lecanemab is indicated for the treatment of mild cognitive impairment and mild dementia due to Alzheimer’s disease in adult patients that are apolipoprotein E ε4 (ApoE ε4) heterozygotes or non-carriers.

For the treatment of seizures associated with Lennox-Gastaut syndrome as an add-on therapy to other anti-epileptic medicines for patients 2 years of age and older.

For the treatment of flares in adult patients with generalised pustular psoriasis (GPP) as monotherapy.

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

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 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.

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

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

ANTICIPATED: 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).

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 HLA-A*02:01-positive adult patients with advanced (unresectable or metastatic) uveal melanoma.

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.

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

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.

Monotherapy as adjuvant treatment for adult patients with Stage IB (tumours ≥ 4cm) to IIIA (7th edition of the UICC/AJCC-staging system) anaplastic lymphoma kinase (ALK)-positive non-small cell lung cancer (NSCLC) following complete tumour resection.