Medicines advice

For the treatment of severe thrombocytopenia in adult patients with chronic liver disease who are scheduled to undergo an invasive procedure.

Symptomatic treatment of myotonia in adult patients with non-dystrophic myotonic disorders.

In combination with obinutuzumab (VenG) is indicated for the treatment of adult patients with previously untreated chronic lymphocytic leukaemia (CLL).

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 with other lipid-lowering therapies in patients who are statin-intolerant, or for whom a statin is contra-indicated

For the treatment of hyperkalaemia in adults.

As a single agent, for the adjuvant treatment of adult patients with HER2-positive early breast cancer who have residual invasive disease, in the breast and / or lymph nodes, after neoadjuvant taxane-based and HER2-targeted therapy.

In combination with nab-paclitaxel for the treatment of adult patients with unresectable locally advanced or metastatic triple-negative breast cancer (TNBC) whose tumours have PD-L1 expression ≥  1% and who have not received prior chemotherapy for metastatic disease.

For the treatment of adult men with non-metastatic castration resistant prostate cancer who are at high risk of developing metastatic disease.

In combination with carboplatin and etoposide, is indicated for the first-line treatment of adult patients with extensive-stage small cell lung cancer.

As an adjunct to diet in adult patients with genetically confirmed familial chylomicronaemia syndrome (FCS) and at high risk for pancreatitis, in whom response to diet and triglyceride lowering therapy has been inadequate.

Treatment of adult patients with advanced soft tissue sarcoma, after failure of anthracyclines and ifosfamide, or who are unsuited to receive these agents.  Efficacy data are based mainly on liposarcoma and leiomyosarcoma patients.

Treatment of severe osteoporosis in postmenopausal women at high risk of fracture.

In combination with axitinib is indicated for the first-line treatment of adult patients with advanced renal cell carcinoma (RCC).

For the treatment of adult patients with secondary progressive multiple sclerosis (SPMS) with active disease evidenced by relapses or imaging features of inflammatory activity.

In combination with rituximab (anti-CD20 antibody) for the treatment of adult patients with previously treated Follicular Lymphoma (Grade 1 - 3a).

In combination with lenalidomide and dexamethasone for the treatment of adult patients with multiple myeloma who have received at least one prior therapy

For the treatment of the following infections in adults:

• Complicated urinary tract infection (cUTI), including pyelonephritis
• Complicated intra-abdominal infection (cIAI)
• Hospital-acquired pneumonia (HAP), including ventilator associated pneumonia (VAP)

Treatment of patients with bacteraemia that occurs in association with, or is suspected to be associated with, any of the infections listed above.

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

Consideration should be given to official guidance on the appropriate use of antibacterial agents.

As monotherapy is indicated in multiple myeloma (MM) for the maintenance treatment of adult patients with newly diagnosed MM who have undergone autologous stem cell transplantation (ASCT).

In combination with rituximab for the treatment of adult patients with Waldenström's macroglobulinaemia.

For the treatment of neuronal ceroid lipofuscinosis type 2 (CLN2) disease, also known as tripeptidyl peptidase 1 (TPP1) deficiency.

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 cyclophosphamide, doxorubicin and prednisone (CHP) is indicated for adult patients with previously untreated systemic anaplastic large cell lymphoma (sALCL).

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.

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

Treatment of chronic immune thrombocytopenia in adult patients who are refractory to other treatments.

For the treatment of patients with ROS1-positive, advanced non-small-cell lung cancer (NSCLC) not previously treated with ROS-1 inhibitors.

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.

For adult patients with relapsing remitting multiple sclerosis.

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.

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

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.

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

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

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)

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 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