Medicines advice

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.

Indicated in children and adolescents from 6 to 17 years old for the treatment of clinically significant, progressive fibrosing interstitial lung diseases (ILDs).
Indicated in adults, adolescents and children aged 6 years and older for the treatment of systemic sclerosis associated interstitial lung disease (SSc-ILD).

With prednisone or prednisolone for the treatment of adult patients with metastatic castration resistant prostate cancer (mCRPC) and BRCA1/2 gene mutations (germline and/or somatic) in whom chemotherapy is not clinically indicated.

Treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older.

As an adjunct to a reduced-calorie diet and increased physical activity to reduce the risk of major adverse cardiovascular events (cardiovascular death, non-fatal myocardial infarction, or non-fatal stroke) in adults with established cardiovascular disease and either obesity or overweight (body mass index [BMI] ≥27 kg/m2).

For the treatment of adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion (UPE) ≥1.0 g/day (or UP/C ≥0.75 g/g).

Tafasitamab is indicated in combination with lenalidomide and rituximab for the treatment of adult patients with relapsed or refractory follicular lymphoma (FL) (Grade 1-3a) after at least one line of systemic therapy.

As monotherapy for the treatment of adult patients with relapsed and refractory multiple myeloma, who have received at least 3 prior therapies, including an immunomodulatory agent, a proteasome inhibitor, and an anti-CD38 antibody and have demonstrated disease progression on the last therapy.

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.

Adult patients with previously untreated CD30+ Stage IIB with risk factors, Stage III or Stage IV HL in combination with etoposide, cyclophosphamide, doxorubicin, dacarbazine, dexamethasone (BrECADD).

For treatment of adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion ≥1.0 g/day (or urine protein-to-creatinine ratio ≥0.8 g/g).

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.

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.

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

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.

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).

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

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.

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.

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.