Medicines advice

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 treatment of adult patients with anaplastic lymphoma kinase (ALK) positive advanced non-small cell lung cancer (NSCLC) previously not treated with an ALK inhibitor or whose disease has progressed after prior treatment with an ALK inhibitor.

Treatment of 5q spinal muscular atrophy (Pre-symptomatic infants).

As monotherapy for the treatment of resectable locally advanced head and neck squamous cell carcinoma as neoadjuvant treatment, continued as adjuvant treatment in combination with radiation therapy with or without concomitant cisplatin and then as monotherapy in adults whose tumours express PD-L1 with a CPS ≥ 1.

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 other pulmonary arterial hypertension (PAH) therapies for the treatment of PAH in adult patients with WHO Functional Class (FC) IV.

Toripalimab in combination with cisplatin and paclitaxel for the first-line treatment of adult patients with unresectable advanced, recurrent, or metastatic oesophageal squamous cell carcinoma.

Treatment of adult patients with unresectable or metastatic well-differentiated extra-pancreatic (epNETs) and pancreatic (pNETs) neuroendocrine tumours who have progressed following at least one prior systemic therapy other than somatostatin analogues (SSAs).

Adult patients (18-65 years) diagnosed by clinical history and a positive test of house dust mite sensitisation (skin prick test and/or specific IgE) with at least one of the following conditions:

• persistent moderate to severe house dust mite allergic rhinitis despite use of symptom-relieving medication
• house dust mite allergic asthma not well controlled by inhaled corticosteroids and associated with mild to severe house dust mite allergic rhinitis. Patients' asthma status should be carefully evaluated before the initiation of treatment

In combination with platinum-containing chemotherapy for the treatment of adult patients with primary advanced or recurrent endometrial cancer (EC) and who are candidates for systemic therapy.

Monotherapy for the treatment of adults with progressive or relapsing active chronic inflammatory demyelinating polyneuropathy after prior treatment with corticosteroids or immunoglobulins

treatment of moderate to severe vasomotor symptoms (VMS) associated with menopause.

Treatment of advanced Parkinson's disease with severe motor fluctuations and hyperkinesia or dyskinesia when available oral combinations of Parkinson medicinal products have not given satisfactory results.

Treatment of adult patients with prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC) who have progressed on or after treatment with androgen receptor pathway inhibitor (ARPI) therapy and are considered appropriate to delay taxane-based chemotherapy.

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).

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.

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.

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.

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.

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

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.

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.

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

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.