Medicines advice

As monotherapy or in combination with obinutuzumab for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL)

As monotherapy for the treatment of adult patients with chronic lymphocytic leukaemia (CLL) who have received at least one prior therapy

In combination with pomalidomide and dexamethasone for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least two prior therapies, including lenalidomide and a proteasome inhibitor, and have demonstrated disease progression on the last therapy.

In adults for the treatment of symptomatic chronic heart failure with reduced ejection fraction.

Prophylaxis of migraine in adults who have at least 4 migraine days per month

In adults and adolescents 12 years and older as add-on maintenance treatment for severe asthma with type 2 inflammation characterised by raised blood eosinophils and/or raised fractional exhaled nitric oxide (FeNO), who are inadequately controlled with high dose inhaled corticosteroid (ICS) plus another medicinal product for maintenance treatment.

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

For the treatment of:

• patients with 5q spinal muscular atrophy (SMA) with a bi-allelic mutation in the survival motor neuron 1 (SMN1) gene and a clinical diagnosis of SMA type 1, or
• patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to three copies of the SMN2 gene

Maintenance treatment of asthma, in adults not adequately controlled with a maintenance combination of a long-acting beta2-agonist and medium dose of inhaled corticosteroid, and who experienced one or more asthma exacerbations in the previous year.

In combination with other antiretroviral medicinal products, for the treatment of adults infected with human immunodeficiency virus 1 (HIV-1) without past or present evidence of resistance to the non-nucleoside reverse transcriptase inhibitor (NNRTI) class.

For the treatment of adults infected with human immunodeficiency virus 1 (HIV-1) without past or present evidence of resistance to the non-nucleoside reverse transcriptase inhibitor (NNRTI) class, lamivudine, or tenofovir.

For treatment in combination with dabrafenib (Tafinlar) for the treatment of adult patients with unresectable or metastatic melanoma with a BRAF V600 mutation. This is an abbreviated submission to remove the SMC restriction to first-line treatment.

For the treatment of adult and paediatric patients 12 years of age and older, with solid tumours that have a neurotrophic tyrosine receptor kinase (NTRK) gene fusion:

• who have a disease that is locally advanced, metastatic or where surgical resection is likely to result in severe morbidity, and
• who have not received a prior NTRK inhibitor
• who have no satisfactory treatment options

Treatment of adult patients with oral ulcers associated with Behçet’s disease who are candidates for systemic therapy.

In combination with low-dose cytarabine, for the treatment of newly diagnosed de novo or secondary acute myeloid leukaemia (AML) in adult patients who are not candidates for standard induction chemotherapy.

In combination with fulvestrant for the treatment of postmenopausal women, and men, with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative, locally advanced or metastatic breast cancer with a PIK3CA mutation after disease progression following endocrine therapy as monotherapy.

As add-on therapy with intranasal corticosteroids (INC) for the treatment of adults (18 years and above) with severe chronic rhinosinusitis with nasal polyps for whom therapy with INC does not provide adequate disease control.

Treatment of corneal cystine crystal deposits in adults and children from 2 years of age with cystinosis.

Substitution treatment for opioid drug dependence, within a framework of medical, social and psychological treatment. The intention of the naloxone component is to deter intravenous misuse. Suboxone is indicated in adults and adolescents over 15 years of age who have agreed to be treated for addiction.

As treatment in pre- and perimenopausal women with advanced breast cancer suitable for hormonal manipulation.

For the treatment of active psoriatic arthritis in adult patients who have responded inadequately to, or who are intolerant to one or more DMARDs. Upadacitinib may be used as monotherapy or in combination with methotrexate

As maintenance treatment of asthma in adults and adolescents 12 years of age and older not adequately controlled with inhaled corticosteroids and inhaled short acting beta2-agonists

As maintenance treatment of asthma in adult patients not adequately controlled with a maintenance combination of a long acting beta2 agonist and a high dose of an inhaled corticosteroid who experienced one or more asthma exacerbations in the previous year.

For the treatment of active psoriatic arthritis in adult patients who have responded inadequately to, or who are intolerant to one or more DMARDs. Upadacitinib may be used as monotherapy or in combination with methotrexate

In combination with cetuximab, for the treatment of adult patients with metastatic colorectal cancer (CRC) with a BRAF V600E mutation, who have received prior systemic therapy.

Treatment of moderate to severe atopic dermatitis in adult patients who are candidates for systemic therapy.

As monotherapy or in combination with obinutuzumab for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL)

In infants and children from 1 month to less than 7 years of age for:

• treatment in monotherapy of infantile spasms (West's syndrome)
  
  
• treatment in combination with other antiepileptic medicinal products for patients with resistant partial epilepsy (focal onset seizures) with or without secondary generalisation, that is where all other appropriate medicinal product combinations have proved inadequate or have not been tolerated.

Single use treatment of mild actinic keratoses lesions with a maximum diameter of 1.8 cm on the face and scalp (hairless areas).

As monotherapy for the treatment of adult patients with metastatic gastric cancer including adenocarcinoma of the gastroesophageal junction, who have been previously treated with at least two prior systemic treatment regimens for advanced disease.

Treatment in adults of other chronic fibrosing interstitial lung diseases with a progressive phenotype.

For the treatment of adult patients with mycosis fungoides (MF) or Sézary syndrome (SS) who have received at least one prior systemic therapy.

In combination with chemotherapy in:

• the neoadjuvant treatment of adult patients with HER2-positive, locally advanced, inflammatory, or early stage breast cancer at high risk of recurrence
• the adjuvant treatment of adult patients with HER2-positive early breast cancer at high risk of recurrence

In combination with docetaxel in adult patients with HER2-positive metastatic or locally recurrent unresectable breast cancer, who have not received previous anti-HER2 therapy or chemotherapy for their metastatic disease.

In combination with bevacizumab for the treatment of adult patients with advanced or unresectable hepatocellular carcinoma (HCC) who have not received prior systemic therapy.

For the treatment of wild‑type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM)

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

For the treatment of primary chronic immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments (e.g. corticosteroids, immunoglobulins).

Topical treatment of mycosis fungoides-type cutaneous T-cell lymphoma (MF-type CTCL) in adult patients.

Treatment of patients with a body weight of 10kg or above with atypical haemolytic uremic syndrome (aHUS) who are complement inhibitor treatment-naïve or have received eculizumab for at least 3 months and have evidence of response to eculizumab.

As monotherapy for the maintenance treatment of adult patients with advanced epithelial (FIGO Stages III and IV) high-grade ovarian, fallopian tube or primary peritoneal cancer who are in response (complete or partial) following completion of first-line platinum-based chemotherapy.