Deferiprone monotherapy is indicated for the treatment of iron overload in patients with thalassaemia major when current chelation therapy is contraindicated or inadequate.
Deferiprone in combination with another chelator is indicated in patients with thalassaemia major when monotherapy with any iron chelator is ineffective, or when prevention or treatment of life-threatening consequences of iron overload (mainly cardiac overload) justifies rapid or intensive correction
Miglustat is indicated for the oral treatment of adult patients with mild to moderate type 1 Gaucher disease. Miglustat may be used only in the treatment of patients for whom enzyme replacement therapy is unsuitable.
Miglustat is indicated for the treatment of progressive neurological manifestations in adult patients and paediatric patients with Niemann-Pick type C disease.
Severe forms of acne (such as nodular or conglobate acne or acne at risk of permanent scarring) resistant to adequate courses of standard therapy with systemic anti-bacterials and topical therapy.
Prophylaxis of clinical manifestations of cytomegalovirus infection in patients subjected to immunosuppressive therapy, particularly in transplant recipients.
The concomitant use of adequate virostatic agents should be considered for CMV-prophylaxis.
Reversal of neuromuscular blockade induced by rocuronium or vecuronium in adults.
For the paediatric population: sugammadex is only recommended for routine reversal of rocuronium induced blockade in children and adolescents aged 2 to 17 years.
Ambrisentan is indicated for treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III, including use in combination treatment. Efficacy has been shown in idiopathic PAH (IPAH) and in PAH associated with connective tissue disease.
Reversal of neuromuscular blockade induced by rocuronium or vecuronium in adults.
For the paediatric population: Sugammadex is only recommended for routine reversal of rocuronium induced blockade in children and adolescents aged 2 to 17 years.
Deferasirox is indicated for the treatment of chronic iron overload due to frequent blood transfusions (≥7 ml/kg/month of packed red blood cells) in patients with beta thalassaemia major aged 6 years and older.
Deferasirox is also indicated for the treatment of chronic iron overload due to blood transfusions when deferoxamine therapy is contraindicated or inadequate in the following patient groups:
- in paediatric patients with beta thalassaemia major with iron overload due to frequent blood transfusions (≥7 ml/kg/month of packed red blood cells) aged 2 to 5 years,
- in adult and paediatric patients with beta thalassaemia major with iron overload due to infrequent blood transfusions (<7 ml/kg/month of packed red blood cells) aged 2 years and older,
- in adult and paediatric patients with other anaemias aged 2 years and older.
Deferasirox is also indicated for the treatment of chronic iron overload requiring chelation therapy when deferoxamine therapy is contraindicated or inadequate in patients with nontransfusion-dependent thalassaemia syndromes aged 10 years and older.
