Metabolism and Endocrinology

Diabetes

• Canagliflozine

  • Invokana is indicated for the treatment of adults and children aged 10 years and older with insufficiently controlled type 2 diabetes mellitus as an adjunct to diet and exercise.

• Dasiglucagon

  • Zegalogue is indicated for the treatment of severe hypoglycaemia in adults, adolescents, and children aged 6 years and over with diabetes mellitus.
• Empagliflozine / metformine ^IND
• Glibenclamide ^IND

• Glucagon

  • Baqsimi is indicated for the treatment of severe hypoglycaemia in adults, adolescents, and children aged 1 year and over with diabetes mellitus.
• Insulin efsitora alfa
• Insulin icodec / semaglutide
• Teplizumab

• Tirzepatide

  • Extension of indication to include treatment of adolescents and children aged 10 years and above with insufficiently controlled type 2 diabetes mellitus as an adjunct to diet and exercise.

Eye disorders

• Teprotumumab

Liver diseases

• Linerixibat

• Maralixibat

  • Extension of indication to include treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) in patients 3 months of age and older.
• Norucholzuur
• Resmetirom
• Seladelpar

• Semaglutide

  • Treatment of non-cirrhotic metabolic dysfunction-associated steatohepatitis with liver fibrosis.

Metabolic diseases

• Arimoclomol

  • Treatment of Niemann-Pick disease type C (NPC) in patients aged 6 months and older in combination with miglustat.
• Atidarsagene autotemcel ^IND
• BCAA-vrije aminozuren

• Burosumab

  • Extension of indication to include treatment of X-linked hypophosphataemia (XLH) in paediatric patients from birth to less than 1 year of age.
• Clemidsogene lanparvovec
• Coördinatieverbinding van ijzercitraat
• Crinecerfont

• Dasiglucagon

  • Infants and children aged 7 days to 12 years with congenital hyperinsulinism (CHI).
• Dersimelagon
• Efzimfotase alfa

• Elamipretide

  • Elamipretide monotherapy for treatment of Barth syndrome in male adolescents, adults and elderly.
  • Elamipretide monotherapy for treatment of primary Mitochondrial myopathy in adults and elderly with pathogenic nuclear DNA mutations.
• Eliglustat ^IND
• Elinzanetant
• Encaleret
• Eneboparatide

• Govorestat

  • Sorbitol dehydrogenase deficiency

• Hydrocortison

  • Treatment of adrenal insufficiency (AI) in adolescents aged 12 years and over and adults
• L-acetylleucine
• Leriglitazon

• Liraglutide

  • Children (6 to <12 years): Saxenda is indicated as an adjunct to healthy nutrition and increased physical activity for weight management in children from the age of 6 to <12 years with obesity (BMI ≥95th percentile)* and body weight ≥45 kg Treatment with Saxenda should be discontinued and re-evaluated if patients have not lost at least 4% of their BMI or BMI z score after 12 weeks on the 3.0 mg/day or maximum tolerated dose. *CDC BMI cut-off points for obesity (≥95th percentile) by sex between 6 to <12 years (see table 2), in accordance with study design of the Trial 4392, see section 5.1.
• Lonapegsomatropine ^IND

• Migalastat

  • Migalastat monotherapy for treatment of Fabryʼs disease in adolescents, adults and elderly with amenable GLA variants and severe renal impairment not receiving any type of dialysis treatment.
  • Migalastat monotherapy for treatment of Fabry’s disease in adults and elderly with amenable GLA variants and end-stage renal disease who are receiving either standard hemodialysis or hemodiafiltration.
• Nedosiran

• Octreotide

  • Oczyesa is indicated for maintenance treatment in adult patients with acromegaly who have responded to and tolerated treatment with somatostatin analogues.
• Paltusotine
• Pegunigalsidase alfa

• Pegvaliase

  • Extension of indication to include treatment of adolescent patients aged 12 to <16 years with PKU
• Rebisufligene etisparvovec

• Relacorilant

  • Cushingʼs Syndrome
• Sepiapterin

• Somapacitan

  • Somapacitan monotherapy for treatment of Short stature in children and adolescents either born small for gestational age or with Turnerʼs syndrome, Noonan syndrome, or idiopathic short stature.

• Somatrogon

  • Somatrogon monotherapy for treatment of growth hormone deficiency in adults.
• Tiomolibdate choline
• Venglustat

Other metabolism and Endocrinology

• Alpelisib

  • PIK3CA-Related Overgrowth Spectrum (PROS). PROS omvat de onderstaande syndromen: - Goedaardige lichenoïde keratose (BLK) - Dysplastische megalencefalie (DMEG) - CLOVES (aangeboren lipomateuze overgroei, vasculaire malformatie, epidermal naevi-syndroom) - Epidermal naevi (EN) - Fibro-adipeuze overgroei (FAO) - Hemimegalencefalie (HMEG) - Hemihyperplasie - meervoudige lipomatose (HHML) geïsoleerde grote lymfatische malformatie (ILM) - Megaencefalie- capillaire malformatie (MCAP) - Muscular hemihyperplasia (mHH) - Seborrhoïsche keratose (SK)
• Autologous human chondrocytes in vitro expanded
• Carbetocine
• Denatonium Acetate Monohydrate
• Diazoxide choline (controlled-release tablet)
• Ersodetug ^IND
• Estetrol

• Infigratinib

  • Infigratinib monotherapy for treatment of Achondroplasia in children over 3 years of age and adolescents.

• Linzagolix

  • Extension of indication to include treatment of endometriosis-associated pain in adult women of reproductive age.
• Navepegritide

• Pegzilarginase

  • Pegzilarginase monotherapy for treatment of Arginase 1 deficiency in newborns, infants and toddlers.

• Semaglutide

  • Op dit moment heeft oraal semaglutide reeds een indicatie voor diabetes. De aan te vragen indicatieuitbreiding zal slaan op het behandelen van obesitas.

• Setmelanotide

  • Extension of indication to include reduction in hunger (or hyperphagia) and BMI/BMI z-score, improvement of metabolic parameters, and increase in energy expenditure in adults and children 4 years of age and above, following rapid and severe weight gain associated with hypothalamic injury and/or impairment.
  • Extension of indication to include the population of children aged 2 years and above for the treatment of pro-opiomelanocortin (POMC), proprotein convertase subtilisin/kexin Type 1 (PCSK1) deficiency or biallelic leptin receptor (LEPR) deficiency and Bardet-Biedl Syndrome (BBS) for IMCIVREE.
• Setrusumab

• Sparsentan

  • Sparsentan monotherapy for treatment of primary and genetic Focal segmental glomerulosclerosis in children over 8 years of age, adolescents, adults and elderly.
• Xylitol/l-carnitine/glucose

Unknown

• Etelcalcetide

  • Etelcalcetide in combination with standard of care for treatment of Hyperparathyroidism secondary in infants and toddlers, children and adolescents with chronic kidney disease receiving maintenance hemodialysis.
• Mirabegron ^IND
• Vibegron