Medicines advice

For the treatment of adults with insufficiently controlled type 2 diabetes (T2D) to improve glycaemic control as an adjunct to diet and exercise:

• As monotherapy when metformin is considered inappropriate due to intolerance or contraindications
• In combination with other medicinal products for the treatment of diabetes

For adult patients treated with a direct factor Xa (FXa) inhibitor (apixaban or rivaroxaban) when reversal of anticoagulation is needed due to life-threatening or uncontrolled bleeding.

For the treatment of neovascular (wet) age-related macular degeneration (wAMD).

As adjunctive therapy of seizures associated with  Dravet syndrome (DS) in conjunction with clobazam, for patients 2 years of age and older.

As adjunctive therapy of seizures associated with Lennox‑Gastaut syndrome (LGS) in conjunction with clobazam, for patients 2 years of age and older.

For the treatment of adults experiencing an episode of acquired thrombotic thrombocytopenic purpura (aTTP), in conjunction with plasma exchange and immunosuppression.

For the treatment of adult patients with moderate to severe limbal stem cell deficiency (defined by the presence of superficial corneal neovascularisation in at least two corneal quadrants, with central corneal involvement, and severely impaired visual acuity) due to physical or chemical ocular burns. A minimum of 1-2 mm2 of undamaged limbus is required for biopsy.

In combination with a selective serotonin reuptake inhibitor (SSRI) or serotonin–norepinephrine reuptake inhibitor (SNRI), for adults with treatment-resistant major depressive disorder, who have not responded to at least two different treatments with antidepressants in the current moderate to severe depressive episode.

For prevention of relapse in recurrent non-infectious uveitis affecting the posterior segment of the eye (NIU-PS).

Monotherapy for the treatment of adult patients who have relapsed or refractory acute myeloid leukaemia (AML) with a FLT3 mutation.

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

As monotherapy or in combination with platinum and 5-fluorouracil (5-FU) chemotherapy, is indicated for the first-line treatment of metastatic or unresectable recurrent head and neck squamous cell carcinoma (HNSCC) in adults whose tumours express PD-L1 with a CPS ≥ 1.

In combination with trastuzumab and chemotherapy for the adjuvant treatment of adult patients with human epidermal growth factor receptor 2 (HER2)-positive early breast cancer (eBC) at high risk of recurrence.

In combination with bendamustine and rituximab for the treatment of adult patients with relapsed/refractory diffuse large B-cell lymphoma (DLBCL) who are not candidates for haematopoietic stem cell transplant.

For the treatment of hyperkalaemia (HK) in adult patients.

Treatment of adult patients with moderately to severely active ulcerative colitis who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a tumour necrosis factor-alpha (TNFα) antagonist.

Extended adjuvant treatment of adult patients with early-Etage hormone receptor positive HER2-overexpressed/amplified breast cancer and who completed adjuvant trastuzumab-based therapy less than one year ago.

Treatment of adult patients with moderately to severely active Crohn’s disease who have had an inadequate response with, lost response to, or were intolerant to either conventional therapy or a tumour necrosis factor-alpha (TNFα) antagonist.

For the prevention of vaso-occlusive complications of Sickle Cell Disease in patients over 2 years of age.

In combination with metformin for the treatment of adults with type II diabetes mellitus (T2DM), to improve glycaemic control when this has not been provided by metformin alone or metformin combined with another oral glucose-lowering medicinal product or with basal insulin.

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.

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.

For the treatment of eosinophilic esophagitis (EoE) in adults (older than 18 years of age).

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

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

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

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

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

For the treatment of hyperkalaemia in adults.

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.

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 statis is contraindicated.

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

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

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

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