Medicines advice

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

For the treatment of community-acquired pneumonia (CAP) in adults when it is considered inappropriate to use other antibacterial agents that are commonly recommended for the initial treatment of these infections. Consideration should be given to official guidance on the appropriate use of antibacterial agents.

In combination with other antiretrovirals for the treatment of adults with multidrug resistant HIV-1 infection for whom it is otherwise not possible to construct a suppressive anti-viral regimen.

For treatment of infections due to aerobic Gram-negative organisms in adults with limited treatment options.

For treatment in adults with primary hypercholesterolaemia (heterozygous familial and non‑familial) or mixed dyslipidaemia, as an adjunct to diet:

• in combination with a statin or statin with other lipid-lowering therapies in patients unable to reach LDL‑C goals with the maximum tolerated dose of a statin
  or
• alone or in combination wtih other lipid-lowering therapies in patients who are statin-intolerant, or for whom a statin is contraindicated.

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

Early breast cancer (EBC)
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

Metastatic breast cancer (MBC)
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 erlotinib is indicated for the first-line treatment of adult patients with metastatic non-small cell lung cancer with activating epidermal growth factor receptor (EGFR) mutations

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

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

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

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

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.

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.

For the 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.

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

Maintenance bronchodilator treatment to relieve symptoms in adult patients with chronic obstructive pulmonary disease.

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 monotherapy for the maintenance treatment of adult patients with platinum-sensitive relapsed high-grade epithelial ovarian, fallopian tube, or primary peritoneal cancer who are in response (complete or partial) to platinum-based chemotherapy.

Indicated for the treatment of hyperkalaemia in adults.

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

For the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) after two or more lines of systemic therapy including a Bruton’s tyrosine kinase (BTK) inhibitor

As monotherapy for the treatment of adult patients with unresectable advanced, recurrent or metastatic oesophageal squamous cell carcinoma after prior fluoropyrimidine and platinum-based combination chemotherapy

As monotherapy for the first-line maintenance treatment of adult patients with locally advanced or metastatic urothelial carcinoma (UC) who are progression-free following platinum-based chemotherapy

For the treatment of adults with primary hypercholesterolaemia (heterozygous familial and non‑familial) or mixed dyslipidaemia, as an adjunct to diet

• in combination with a statin or statin with other lipid‑lowering therapies in patients unable to reach LDL‑C goals with the maximum tolerated dose of a statin, or
• alone or in combination with other lipid‑lowering therapies in patients who are statin‑intolerant, or for whom a statin is contraindicated

For the treatment of active psoriatic arthritis in adult patients who have had an inadequate response or who have been intolerant to a prior disease-modifying antirheumatic drug (DMARD) therapy, alone or in combination with methotrexate (MTX).

Non-tuberculous mycobacterial (NTM) lung infections caused by Mycobacterium avium Complex (MAC) in adults with limited treatment options who do not have cystic fibrosis. Consideration should be given to official guidance on the appropriate use of antibacterial agents.

Adults and adolescents 12 years and older with advanced RET-mutant medullary thyroid cancer (MTC) who require systemic therapy following prior treatment with cabozantinib and/or vandetanib.
Adults with advanced RET fusion-positive thyroid cancer (TC) who require systemic therapy following prior treatment with lenvatinib and/or sorafenib.

As monotherapy for the first‑line treatment of metastatic microsatellite instability‑high (MSI‑H) or mismatch repair deficient (dMMR) colorectal cancer in adults.

Treatment of moderate to severe active rheumatoid arthritis in adult patients who have responded inadequately to, or who are intolerant to one or more disease-modifying anti-rheumatic drugs (DMARDs). Filgotinib may be used as monotherapy or in combination with methotrexate (MTX).

For the treatment of proven nephropathic cystinosis.

As monotherapy for the treatment of adult patients with metastatic castration-resistant prostate cancer and BRCA1/2-mutations (germline and/or somatic) who have progressed following prior therapy that included a new hormonal agent.

As monotherapy for the treatment of adults with advanced rearranged during transfection (RET) fusion-positive non-small cell lung cancer (NSCLC) who require systemic therapy following prior treatment with immunotherapy and/or platinum-based chemotherapy.

In combination with bevacizumab for the maintenance treatment of adult patients with advanced (FIGO stages III and IV) high-grade epithelial ovarian, fallopian tube or primary peritoneal cancer who are in response (complete or partial) following completion of first-line platinum-based chemotherapy in combination with bevacizumab and whose cancer is associated with homologous recombination deficiency (HRD) positive status defined by either a BRCA1/2 mutation and/or genomic instability.

For the treatment of acute bacterial skin and skin structure infections (ABSSSI) in adults.

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