Medicines advice

In combination with pomalidomide and dexamethasone (BPd) for the treatment of adult patients with multiple myeloma who have received at least one prior therapy including lenalidomide.

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

In combination with fulvestrant for the treatment of adult patients with hormone receptor (HR) positive, human epidermal growth factor receptor 2 (HER2) negative (defined as IHC 0 or 1+, or IHC 2+/ISH-) locally advanced or metastatic breast cancer with one or more PIK3CA/AKT1/PTEN-alterations following recurrence or progression on or after an endocrine based regimen.

In combination with carboplatin and paclitaxel for the first-line treatment of adults with primary advanced or recurrent endometrial cancer who are candidates for systemic therapy, followed by maintenance treatment with:

• durvalumab as monotherapy in endometrial cancer that is mismatch repair deficient (dMMR)
• durvalumab in combination with olaparib in endometrial cancer that ismismatch repair proficient (pMMR).

For the treatment of adult patients with metastatic colorectal cancer (mCRC) who have been previously treated with available therapies, including fluoropyrimidine-, oxaliplatin-, and irinotecan-based chemotherapy, with or without an anti-VEGF therapy, and, if RAS wildtype and medically appropriate, an anti-EGFR therapy

In adult women of reproductive age for symptomatic treatment of endometriosis in women with a history of previous medical or surgical treatment for their endometriosis.

In combination with platinum-based chemotherapy as neoadjuvant treatment, and then continued as monotherapy as adjuvant treatment after surgical resection, for the treatment of adults with resectable (tumours ≥4 cm or node positive) non-small cell lung cancer and no known EGFR mutations or ALK rearrangements.

For treatment of 5q spinal muscular atrophy (SMA).

Treatment of adult patients with advanced renal cell carcinoma (RCC) whose disease has progressed on or after treatment with a programmed death receptor-1 (PD-1) / programmed death ligand (PD-L1) inhibitor and a vascular endothelial growth factor tyrosine kinase inhibitor (VEGF-TKI).

Treatment of adult patients with Philadelphia chromosome (Ph)-negative, CD19-positive, B-cell precursor acute lymphoblastic leukaemia (ALL) in the consolidation phase.

In combination with binimetinib for the treatment of adult patients with advanced non-small cell lung cancer with a BRAF V600E mutation.

As monotherapy for the maintenance treatment of adult patients with
advanced (FIGO Stages III and IV) 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.

Treatment of adult patients with previously untreated cell membrane receptor 30-positive (CD30+) Stage III or IV Hodgkin lymphoma (HL) in combination with doxorubicin, vinblastine and dacarbazine (AVD).

In adults as add-on maintenance treatment for uncontrolled chronic obstructive pulmonary disease (COPD) characterised by raised blood eosinophils on a combination of an inhaled corticosteroid (ICS), a long-acting beta2-agonist (LABA), and a long-acting muscarinic antagonist (LAMA), or on a combination of a LABA and a LAMA if ICS is not appropriate.

For prophylaxis of cytomegalovirus (CMV) disease in CMV-seronegative adults who have received a kidney transplant from a CMV-seropositive donor [D+/R-].

For the 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 biologic treatment.

Treatment of adult patients with advanced gastrointestinal stromal tumour (GIST) who have received prior treatment with three or more kinase inhibitors, including imatinib.

For the treatment of adult patients with unresectable or metastatic HER2-positive (IHC3+) solid tumours who have received prior treatment or who have no satisfactory alternative treatment options.

As monotherapy for the treatment of adult patients with mantle cell lymphoma (MCL) who have received at least one prior therapy

Treatment of osteoporosis in postmenopausal women at increased risk of fracture.

In combination with carboplatin and pemetrexed for the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with activating epidermal growth factor receptor (EGFR) Exon 20 insertion mutations

Treatment of moderate to severe chronic hand eczema (CHE) in adults for whom topical corticosteroids (TCS) are inadequate or inappropriate.

In combination with tremelimumab for the first line treatment of adults with advanced or unresectable hepatocellular carcinoma

Treatment of postmenopausal women, and men, with estrogen receptor (ER)-positive, HER2-negative, locally advanced or metastatic breast cancer with an activating ESR1 mutation who have disease progression following at least one line of endocrine therapy including a CDK 4/6 inhibitor.

Treatment of sickle cell disease (SCD) in patients 12 years of age and older with recurrent vaso-occlusive crises (VOCs) who have βS/βS, βS/β0 or βS/β+, for whom haematopoietic stem cell (HSC) transplantation is appropriate and a human leukocyte antigen (HLA)-matched related HSC donor is not available.

For the treatment of Duchenne muscular dystrophy (DMD) in patients 6 years of age and older

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

For the treatment of proven nephropathic cystinosis.

In combination with an aromatase inhibitor is indicated for the adjuvant treatment of patients with hormone receptor (HR)-positive, human epidermal growth factor receptor 2 (HER2)-negative early breast cancer at high risk of recurrence.s

In pre- or perimenopausal women, or in men, the aromatase inhibitor should be combined with a luteinising hormone-releasing hormone (LHRH) agonist.

Treatment of primary biliary cholangitis (PBC), including pruritus, in adults in combination with ursodeoxycholic acid (UDCA) who have an inadequate response to UDCA alone, or as monotherapy in those unable to tolerate UDCA.

In combination with carboplatin and etoposide for the first‑line treatment of adult patients with extensive‑stage small cell lung cancer (ES‑SCLC).

Treatment of adult patients with extensive-stage small cell lung cancer (ES-SCLC) with disease progression on or after at least two prior lines of therapy including platinum-based chemotherapy.

In combination with fluoropyrimidine- and platinum-containing chemotherapy, for the first-line treatment of adult patients with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-negative gastric or gastro-oesophageal junction (GEJ) adenocarcinoma whose tumours are Claudin (CLDN) 18.2 positive.

Short-term treatment of mild to moderate acute ulcerative colitis limited to the rectum (ulcerative proctitis) in adult patients.

For the treatment of adult patients with moderately to severely active ulcerative colitis (UC) who have had an inadequate response, lost response, or were intolerant to either conventional therapy, a biologic treatment, or Janus kinase (JAK) inhibitor.

For the treatment of adult patients with moderately to severely active Crohn's disease (CD) who have had an inadequate response, lost response, or were intolerant to either conventional therapy or a biologic treatment.

In combination with lazertinib for the first‑line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) Exon 19 deletions or Exon 21 L858R substitution mutations.

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

The presence of a nonsense mutation in the dystrophin gene should be determined by genetic testing.

Treatment of metachromatic leukodystrophy characterized by biallelic mutations in the ARSA gene leading to a reduction of the ARSA enzymatic activity:

• in children with late infantile or early juvenile forms, without clinical manifestations of the disease
• in children with the early juvenile form, with early clinical manifestations of the disease, who still have the ability to walk independently and before the onset of cognitive decline

As monotherapy for the treatment of adult patients with aggressive systemic mastocytosis (ASM), systemic mastocytosis with an associated haematological neoplasm (SM-AHN) or mast cell leukaemia (MCL).