Medicines advice

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

In combination with gemcitabine and cisplatin for the first-line treatment of adults with locally advanced, unresectable, or metastatic biliary tract cancer (BTC).

Long-term enzyme replacement therapy (ERT) in adult patients with confirmed diagnosis of Fabry disease (FD) (deficiency of alpha galactosidase).

Prevention of preterm birth in women with a singleton pregnancy who have a short cervix (mid-trimester sonographic cervix ≤25 mm) and/or a history of spontaneous preterm birth.

For treatment of people 16 years and older with moderately to severely active CD who have had an inadequate response to, lost response to, or were intolerant to conventional therapy or a biologic therapy, or if such therapies are not advisable

For the treatment of wild‑type or hereditary transthyretin amyloidosis in adult patients with cardiomyopathy (ATTR-CM)

Treatment of proven nephropathic cystinosis (NC). Mercaptamine reduces cystine accumulation in some cells (e.g. leukocytes, muscle and liver cells) of NC patients and, when treatment is started early, it delays the development of renal failure.

In combination with a rituximab or cyclophosphamide regimen for the treatment of adult patients with severe, active granulomatosis with polyangiitis (GPA) or microscopic polyangiitis (MPA).

As monotherapy for the treatment of adults with advanced RET fusion‑positive non‑small cell lung cancer (NSCLC) not previously treated with a RET inhibitor.

Alone or in combination with methotrexate (MTX), is anticipated to be indicated for the treatment of active psoriatic arthritis (PsA) in adults who have had an inadequate response or who have been intolerant to one or more disease-modifying antirheumatic drugs (DMARDs).

Treatment of adults with metastatic hormone-sensitive prostate cancer (mHSPC) in combination with docetaxel

As monotherapy for the treatment of adult patients with chronic lymphocytic leukaemia (CLL)

Prophylaxis of migraine in adults who have at least four migraine days per month.

The treatment of cytomegalovirus (CMV) infection and/or disease that are refractory (with or without resistance) to one or more prior therapies, including ganciclovir, valganciclovir, cidofovir or foscarnet in adult patients who have undergone a haematopoietic stem cell transplant (HSCT) or solid organ transplant (SOT).

In combination with mycophenolate mofetil for the treatment of adult patients with active class III, IV or V (including mixed class III/V and IV/V) lupus nephritis (LN).

Treatment of seizures associated with Dravet syndrome as an add-on therapy to other anti-epileptic medicines for patients 2 years of age and older.

As monotherapy for the adjuvant treatment of adult patients with germline BRCA1/2-mutations who have HER2-negative, high-risk early breast cancer who have previously been treated with neoadjuvant or adjuvant chemotherapy.

Treatment of adult patients with prostate-specific membrane antigen (PSMA)-positive metastatic castration-resistant prostate cancer (mCRPC) who have been treated with androgen receptor (AR) pathway inhibition and taxane-based chemotherapy or who are not medically suitable for taxanes.

Treatment of adult patients with von Hippel-Lindau (VHL) disease who require therapy for VHL associated renal cell carcinoma (RCC), central nervous system (CNS) hemangioblastomas, or pancreatic neuroendocrine tumours (pNET), and for whom localised procedures are unsuitable or undesirable.

Treatment of adult patients 26 years of age and above with relapsed or refractory B-cell precursor acute lymphoblastic leukaemia (ALL).

Symbicort Turbohaler 200/6 as reliever therapy for adults and adolescents ≥12 years with mild asthma 

• for treatment of high-risk localised and locally advanced hormone dependent prostate cancer in combination with radiotherapy
  
  
• as neo-adjuvant treatment prior to radiotherapy in patients with high-risk localised or locally advanced hormone dependent prostate cancer

Adults with active non-radiographic axial spondyloarthritis with objective signs of inflammation as indicated by elevated C-reactive protein (CRP) and/or magnetic resonance imaging (MRI) who have responded inadequately or are intolerant to non-steroidal anti-inflammatory drugs (NSAIDs)
and
adults with active ankylosing spondylitis who have responded inadequately or are intolerant to conventional therapy.

Long-term enzyme replacement therapy used in combination with the enzyme stabiliser miglustat for the treatment of adults with late-onset Pompe disease (LOPD; acid α-glucosidase [GAA] deficiency.

As monotherapy for the treatment of adult patients with metastatic or locally advanced cutaneous squamous cell carcinoma (CSCC) who are not candidates for curative surgery or curative radiation.

As monotherapy for the treatment of adult patients with unresectable or metastatic HER2‑low breast cancer who have received prior chemotherapy in the metastatic setting or developed disease recurrence during or within 6 months of completing adjuvant chemotherapy.

Treatment of moderate to severe plaque psoriasis in adults who are candidates for systemic therapy.

In combination with platinum-based chemotherapy for the neoadjuvant treatment of resectable (tumours ≥ 4 cm or node positive) non-small cell lung cancer in adults.

Treatment of X-linked hypophosphataemia in children and adolescents aged 1 to 17 years with radiographic evidence of bone disease.

Monotherapy for the treatment of multiple myeloma in adult patients, who have received at least four prior therapies and whose disease is refractory to at least one proteasome inhibitor, one immunomodulatory agent, and an anti-CD38 monoclonal antibody, and who have demonstrated disease progression on the last therapy.

As monotherapy for the treatment of adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and high-grade B-cell lymphoma (HGBL), after two or more lines of systemic therapy.

Treatment of adults with moderate-to-severe prurigo nodularis (PN) who are candidates for systemic therapy.

As monotherapy for the treatment of adult patients with locally advanced or metastatic differentiated thyroid carcinoma (DTC), refractory or not eligible to radioactive iodine (RAI) who have progressed during or after prior systemic therapy.

as monotherapy for the treatment of human leukocyte antigen (HLA)-A*02:01-positive adult patients with unresectable or metastatic uveal melanoma

For 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 active moderate to severe hidradenitis suppurativa (HS) in adults with an inadequate response to conventional systemic HS therapy.

Treatment of advanced levodopa-responsive Parkinson’s disease (PD) with severe motor fluctuations and hyperkinesia or dyskinesia when available combinations of Parkinson medicinal products have not given satisfactory results.

As monotherapy for the treatment of adult patients with germline BRCA1/2 mutations, who have HER2-negative locally advanced or metastatic breast cancer. Patients should have been previously treated with an anthracycline and/or a taxane in the (neo)adjuvant, locally advanced or metastatic setting unless patients were not suitable for these treatments. Patients with hormone receptor (HR)-positive breast cancer should have been treated with a prior endocrine-based therapy, or be considered unsuitable for endocrine-based therapy.

Treatment of severe alopecia areata in adults and adolescents aged 12 years and over.

Treatment of adult patients with insomnia characterised by symptoms present for at least 3 months and considerable impact on daytime functioning.