Medicines advice

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.

For the treatment of opioid-induced constipation (OIC) in adult patients who have previously been treated with a laxative.

For the 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 biologic or have medical contraindications to such therapies.

For the improvement of walking in adult patients with multiple sclerosis (MS) with walking disability (EDSS [expanded disability status scale] 4 to 7).

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.

As monotherapy for the treatment of adults with Philadelphia chromosome negative CD19 positive B-precursor ALL in first or second complete remission with minimal residual disease (MRD) greater than or equal to 0.1%.

For the symptomatic treatment of myotonia in adult patients with non-dystrophic myotonic disorders (NDM).

As monotherapy is indicated for the treatment of adult patients with anaplastic lymphoma kinase (ALK) positive advanced non-small cell lung cancer (NSCLC) whose disease has progressed after:

• alectinib or ceritinib as the first ALK tyrosine kinase inhibitor (TKI) therapy; or
• crizotinib and at least one other ALK TKI.

In preterm infants for the treatment of retinopathy of prematurity (ROP) with zone I (stage 1+, 2+, 3 or 3+), zone II (stage 3+) or AP-ROP (aggressive posterior ROP) disease.

Secondary hyperparathyroidism (HPT)

• Treatment of secondary HPT in adult patients with end-stage renal disease (ESRD) on maintenance dialysis therapy.
• Treatment of secondary HPT in children aged 3 years and older with ESRD on maintenance dialysis therapy in whom secondary HPT is not adequately controlled with standard of care therapy.

Parathyroid carcinoma and primary HPT in adults

• Reduction of hypercalcaemia in adult patients with:
  - parathyroid carcinoma
  - primary HPT for whom parathyroidectomy would be indicated on the basis of serum calcium levels (as defined by   relevant treatment guidelines), but in whom
  parathyroidectomy is not clinically appropriate or is contraindicated

As monotherapy for the treatment of adult patients with metastatic or locally advanced cutaneous squamous cell carcinoma (CSCC) who are not candidates for curative surgery or curative radiation.

Treatment of patients aged 1 year and above with short bowel syndrome (SBS). Patients should be stable following a period of intestinal adaptation after surgery.

Treatment of hyperkalaemia in adult patients

For the treatment of X-linked hypophosphataemia (XLH) with radiographic evidence of bone disease in children 1 year of age and older and adolescents with growing skeletons.

In combination with binimetinib for the treatment of adult patients with unresectable or metastatic melanoma with a BRAF V600 mutation.

In combination with granulocyte-colony stimulating factor (G-CSF) to enhance mobilisation of haematopoietic stem cells to the peripheral blood for collection and subsequent autologous transplantation in children aged 1 year to <18 years with lymphoma or solid malignant tumours, either:
- pre-emptively, when circulating stem cell count on the predicted day of collection after adequate mobilisation with G-CSF (with or without chemotherapy) is expected to be insufficient with regards to desired hematopoietic stem cells yield, or
- who previously failed to collect sufficient haematopoietic stem cells.

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

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

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

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

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

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).

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.

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.

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 neovascular (wet) age-related macular degeneration (wAMD).

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.

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

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).