Medicines advice

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 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.

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

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%.

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.

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.

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.

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

In combination with lenalidomide and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma who are ineligible for autologous stem cell transplant.

In adult men for the treatment of non-metastatic castration-resistant prostate cancer (NM-CRPC) who are at high risk of developing metastatic disease.

Treatment of proliferative diabetic retinopathy in adults.

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.

For the treatment of adult and paediatric patients with vision loss due to inherited retinal dystrophy caused by confirmed biallelic RPE65 mutations and who have sufficient viable retinal cells.

For the treatment of adult patients with CD30-positive cutaneous T-cell lymphoma (CTCL) after at least one prior systemic therapy.

For the prophylaxis of migraine in adults who have at least four migraine days per month.

For the treatment of adult patients with early primary progressive multiple sclerosis (PPMS) in terms of disease duration and level of disability, and with imaging features characteristic of inflammatory activity.

with prednisolone for the treatment of adult men with newly diagnosed high-risk metastatic hormone-sensitive prostate cancer (mHSPC) in combination with androgen deprivation therapy (ADT)

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).

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

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

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

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 hyperkalaemia (HK) in adult patients.

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

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 the treatment of hyperkalaemia in adults.

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.