Medicines advice

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.

As adjuvant treatment in combination with tamoxifen or an aromatase inhibitor, of endocrine responsive early stage breast cancer in pre- and perimenopausal women at higher risk of disease recurrence (young age, high grade tumour, lymph node involvement). In women who have received chemotherapy, premenopausal status must be confirmed after completion of chemotherapy.

For adult patients with relapsing remitting multiple sclerosis.

For the treatment of adult patients 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 eculizumab for at least the past 6 months.

For 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). Upadacitinib may be used as monotherapy or in combination with methotrexate.

Indication under review: For the treatment of:

• hospital-acquired pneumonia (HAP), including ventilator associated pneumonia (VAP), in adults
• bacteraemia that occurs in association with, or is suspected to be associated with HAP or VAP, in adults.

for prevention of photoxicity in adult patients with erythropoietic protoporphyria (EPP). Scenesse is to be administered under a Post-Authorisation Safety Study (PASS) protocol in line with the approved European Medicines Agency Risk Management Plan.

Maintenance treatment in adult patients with moderate to severe chronic obstructive pulmonary disease (COPD) who are not adequately treated by a combination of an inhaled corticosteroid and a long‑acting beta2‑agonist or combination of a long-acting beta2‑agonist and a long‑acting muscarinic antagonist.

In adult men for the treatment of metastatic hormone-sensitive prostate cancer (mHSPC) in combination with androgen deprivation therapy (ADT).

As monotherapy for the treatment of adult patients with germline BRCA1/2 mutations, who have HER2-negative locally advanced or metastatic breast cancer.

As an add-on therapy with intranasal corticosteroids for the treatment of adults with severe chronic rhinosinusitis with nasal polyposis for whom therapy with systemic corticosteroids and/or surgery do not provide adequate disease control.

For the treatment of active non-radiographic axial spondyloarthritis with objective signs of inflammation as indicated by elevated C-reactive protein (CRP) and/or magnetic resonance imaging (MRI) evidence in adults who have responded inadequately to nonsteroidal anti-inflammatory drugs (NSAIDs).

In combination with bortezomib, thalidomide and dexamethasone is indicated for the treatment of adult patients with newly diagnosed multiple myeloma who are eligible for autologous stem cell transplant.

For the treatment of insomnia in children and adolescents aged 2-18 with autism spectrum disorder (ASD) and / or Smith-Magenis syndrome, where sleep hygiene measures have been insufficient.

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

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.

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.

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 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

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.

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 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 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)

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 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.

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.

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