Medicines advice

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

Adolescents (12-17 years) diagnosed by clinical history and a positive test of house dust mite sensitisation (skin prick test and/or specific IgE) with persistent moderate to severe house dust mite allergic rhinitis despite use of symptom-relieving medication.

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.

Treatment of hyperkalaemia in adult patients.

Treatment of giant cell arteritis in adult patients.

As an add-on to standard therapy for the treatment of generalised myasthenia gravis (gMG) in adult patients who are anti‑acetylcholine receptor (AChR) antibody positive.

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).

Add-on treatment for adult patients with relapsing or refractory eosinophilic granulomatosis with polyangiitis.

IMFINZI in combination with gemcitabine and cisplatin as neoadjuvant treatment, followed by IMFINZI as monotherapy adjuvant treatment after radical cystectomy, is indicated for the treatment of adults with resectable muscle invasive bladder cancer (MIBC).

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

In combination with gemcitabine and oxaliplatin is indicated for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma not otherwise specified (DLBCL NOS) who are ineligible for autologous stem cell transplant (ASCT).

For treatment of HLA-A*02:01-positive adult patients with advanced (unresectable or metastatic) uveal melanoma.

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.

For the treatment of peripheral neuropathic pain in diabetic adults either alone or in combination with other medicinal products for pain.

Treatment of adult men with metastatic hormone-sensitive prostate cancer (mHSPC) in combination with androgen deprivation therapy.

As an add-on therapy with intranasal corticosteroids (INCS) for the treatment of adults with severe chronic rhinosinusitis with nasal polyposis (CRSwNP) for whom therapy with systemic corticosteroids (SCS) and/or surgery do not provide adequate disease control.

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.

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 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 (Pre-symptomatic infants).

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

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.

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)

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

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

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.