Medicines advice

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.

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.

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 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)

For the treatment of moderate-to-severe atopic dermatitis in adult and adolescents 12 years and older who are candidates for systemic therapy

For routine prevention of recurrent attacks of hereditary angioedema (HAE) in patients aged 12 years and older.

As monotherapy for the maintenance treatment of adult patients with newly diagnosed advanced (FIGO stages III and IV) BRCA1/2‐mutated (germline and/or somatic) high‐grade epithelial ovarian, fallopian tube or primary peritoneal cancer who are in response (complete or partial) following completion of first line platinum‐based chemotherapy.

The treatment of adult patients with polycythaemia vera (PV) who are resistant to or intolerant of hydroxyurea (HU).

For the treatment of severe thrombocytopenia in adult patients with chronic liver disease undergoing invasive procedures.

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.

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%.

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.

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 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).

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.

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

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

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

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

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.

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.