Medicines advice

As monotherapy for the treatment of adult patients with relapsed or refractory mantle cell lymphoma (MCL) not previously treated with a BTK inhibitor.

Treatment of wild-type or variant transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).

Acute treatment of migraine with or without aura in adults.

In adults and children (5 years or older) for the treatment of moderate-to-severe allergic rhinitis and/or conjunctivitis induced by pollen from the birch homologous group. It is indicated in patients with a clinical history of symptoms despite use of symptom-relieving medication and a positive test of sensitisation to a member of the birch homologous group (skin prick test and/or specific IgE).

Treatment of adult patients with severe alopecia areata.

In combination with palbociclib and fulvestrant for the treatment of adult patients with PIK3CA‑mutated, oestrogen receptor (ER)‑positive, HER2‑negative, locally advanced or metastatic breast cancer, following recurrence on or within 12 months of completing adjuvant endocrine treatment.

As monotherapy for the treatment of adult patients with folate receptor-alpha (FRα) positive, platinum-resistant high grade serous epithelial ovarian, fallopian tube, or primary peritoneal cancer who have received one to three prior systemic treatment regimens.

Treatment of 5q Spinal Muscular Atrophy (SMA) – higher dosage.

In combination with mycophenolate mofetil (MMF) for the treatment of adult patients with active Class III or IV, with or without concomitant Class V, lupus nephritis (LN).

Parathyroid hormone (PTH) replacement therapy indicated for the treatment of adults with chronic hypoparathyroidism.

As monotherapy for the treatment of symptomatic, inoperable plexiform neurofibromas (PN) in adult patients with neurofibromatosis type 1 (NF1).

As an adjunct to a reduced-calorie diet and increased physical activity for weight management, including weight loss and weight maintenance, in adults with an initial Body Mass Index (BMI) of
• ≥30 kg/m2 (obesity), or
• ≥27 kg/m2 to <30 kg/m2 (overweight) in the presence of at least one weight-related comorbidity.

Treatment of wild-type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM).

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

In combination with fulvestrant for the treatment of adult patients with hormone receptor positive (HR+), human epidermal growth factor receptor 2 negative (HER2-; 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.

Add-on therapy with intranasal corticosteroids for the treatment of adult patients with severe chronic rhinosinusitis with nasal polyps (CRSwNP) for whom therapy with systemic corticosteroids and/or surgery do not provide adequate control

For the treatment of postmenopausal women, and men, with oestrogen receptor-positive (ER+), human epidermal factor receptor 2-negative (HER2-), locally advanced or metastatic (m) breast cancer (BC) with an activating oestrogen receptor 1 (ESR1) mutation (mut) who have disease progression following at least one line of endocrine therapy (ET) including a cyclin-dependent kinase 4/6 inhibitor (CDK4/6i).

Tepkinly as monotherapy is indicated for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) after two or more lines of systemic therapy.

In adolescents aged 12 years and over and adults for the treatment of adrenal insufficiency (AI)

Treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL), primary mediastinal large B-cell lymphoma (PMBCL) and follicular lymphoma grade 3B (FL3B), after two or more lines of systemic therapy.

As monotherapy for the first-line treatment of adult patients with non-small cell lung cancer (NSCLC) expressing PD-L1 (in ≥ 50% tumour cells), with no EGFR, ALK or ROS1 aberrations, who have:

• locally advanced NSCLC who are not candidates for definitive chemoradiation, or
• metastatic NSCLC

In combination with platinum‐based chemotherapy for the first‐line treatment of adult patients with NSCLC expressing PD-L1 (in ≥ 1% of tumour cells), with no EGFR, ALK or ROS1 aberrations, who have:

• locally advanced NSCLC who are not candidates for definitive chemoradiation, or
• metastatic NSCLC

In combination with bortezomib, lenalidomide and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma

Routine prevention of recurrent attacks of hereditary angioedema (HAE) in adult and adolescent patients aged 12 years and older.

For treatment of adult patients with diffuse large B-cell lymphoma (DLBCL), high-grade B-cell lymphoma (HGBCL), primary mediastinal large B-cell lymphoma (PMBCL) and follicular lymphoma grade 3B (FL3B), who have relapsed within 12 months from completion of, or are refractory to, first line chemoimmunotherapy.

For treatment of adult patients (aged 18 years and over) with relapsed or refractory (R/R) B-cell precursor acute lymphoblastic leukaemia (ALL).

Treatment of adult patients with relapsed or refractory (R/R) chronic lymphocytic leukaemia (CLL) who have been previously treated with a Bruton’s tyrosine kinase inhibitor (BTKi).

For treatment of symptomatic anaemia associated with chronic kidney disease (CKD) in adults on chronic maintenance dialysis.

Acalabrutinib in combination with bendamustine and rituximab (BR) is indicated for the treatment of adult patients with previously untreated mantle cell lymphoma (MCL) who are not eligible for autologous stem cell transplant (ASCT).

Prevention of phototoxicity in adult patients with erythropoietic protoporphyria (EPP)

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.

Treatment of X-linked hypophosphataemia with radiographic evidence of bone disease in adults

In combination with bortezomib, lenalidomide and dexamethasone for the treatment of adult patients with newly diagnosed multiple myeloma.

Anticipated MA: For the routine prevention of recurrent attacks of hereditary angioedema (HAE) in adults and adolescents aged 12 years and older.

As monotherapy for the treatment of adult patients with mismatch repair deficient (dMMR)/ microsatellite instability‑high (MSI‑H) recurrent or advanced endometrial cancer (EC) that has progressed on or following prior treatment with a platinum‑containing regimen.

In adults as add-on maintenance treatment for uncontrolled chronic obstructive pulmonary disease (COPD) characterised by raised blood eosinophils (EOS) 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.

In combination with 5-fluorouracil/leucovorin/oxaliplatin/docetaxel (FLOT) chemotherapy as neoadjuvant and adjuvant treatment, followed by adjuvant durvalumab monotherapy, for the treatment of adults with resectable gastric or gastro‑oesophageal junction adenocarcinoma.

in combination with lenalidomide and rituximab is indicated for the treatment of adult patients with relapsed or refractory (R/R) follicular lymphoma (FL) who have received at least one line of systemic therapy.

Treatment of symptomatic chronic heart failure with left ventricular ejection fraction (LVEF) ≥40% in adults.

In combination with rituximab, cyclophosphamide, doxorubicin, vincristine and prednisolone (R-CHOP) alternating with rituximab, dexamethasone, high-dose cytarabine and cisplatin (R-DHAP) or rituximab, dexamethasone, high-dose cytarabine and oxaliplatin [R-DHAOx]) without ibrutinib, followed by ibrutinib monotherapy is indicated for the treatment of adult patients with previously untreated mantle cell lymphoma (MCL) who would be eligible for autologous stem cell transplantation (ASCT).