Metabolism and Endocrinology

Bowel diseases

• Apraglutide

Diabetes

• Cagrilintide / semaglutide

• Ertugliflozin

  • Ertugliflozin in combination with metformin with or without insulin for treatment of Type 2 diabetes mellitus in children over 10 years of age and adolescents.
• Glibenclamide ^IND
• Insulin efsitora alfa

• Insulin icodec / semaglutide

  • Treatment of adults with type 2 diabetes mellitus insufficiently controlled on basal insulin or glucagon-like peptide 1 (GLP-1) receptor agonists.
  • Extension of indication to include treatment of adults with insufficiently controlled type 2 diabetes mellitus as an adjunct to diet and exercise.
• Insulin oral
• Orforglipron

• Tarcocimab tedromer

  • Moderately severe to severe non-proliferative diabetic retinopathy in adults and elderly with type 1 or 2 diabetes mellitus.
• Teplizumab

• Tirzepatide

  • Mounjaro is indicated for the treatment of adults, adolescents and children aged 10 years and above with insufficiently controlled type 2 diabetes mellitus as an adjunct to diet and exercise - as monotherapy when metformin is considered inappropriate due to intolerance or contraindications - in addition to other medicinal products for the treatment of diabetes.

Eye disorders

• Teprotumumab

Liver diseases

• Linerixibat
• Norucholzuur

• Odevixibat

  • Odevixibat behandeling van galgangatresie (biliary atresia) bij kinderen die een Kasai-operatie hebben gehad. Indicatiestelling is inschatting op basis van lopende fase 3 studie BOLD:
• Resmetirom
• Saroglitazar
• Seladelpar

• Semaglutide

  • Kayshild is indicated in conjunction with diet and exercise for the treatment of adults with non-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) with moderate to advanced liver fibrosis (fibrosis stages F2 to F3).

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

  • Crysvita is indicated for the treatment of X-linked hypophosphataemia in infants from 1 month to 1 year of age with hypophosphataemia (see section 5.1)
• Clemidsogene lanparvovec
• Coördinatieverbinding van ijzercitraat

• 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.
• Elinzanetant
• Encaleret
• Eneboparatide
• Garetosmab

• Govorestat

  • Sorbitol dehydrogenase deficiency.
• Hydrocortison slow-release ^IND

• L-acetylleucine

  • Aqneursa is indicated for the treatment of neurological manifestations of Niemann-Pick type C (NPC) disease, in combination with miglustat, or as a monotherapy in patients where miglustat is not tolerated, in adults and children aged 6 years and older and weighing at least 20 kg.
• 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.

• Octreotide

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

• Osilodrostat

  • Treatment of endogenous Cushing’s syndrome in adolescents and children aged 6 years and older.
• Paltusotine

• Pegvaliase

  • Palynziq is indicated for the treatment of patients with phenylketonuria (PKU) aged 12 years and older who have inadequate blood phenylalanine control (blood phenylalanine levels greater than 600 micromol/l) despite prior management with available treatment options.
• RGX-111
• Rebisufligene etisparvovec

• Relacorilant

  • Cushingʼs Syndrome.
• Sepiapterine

• Somapacitan

  • Sogroya is indicated for treatment of growth failure in children and adolescents in the following indications: - Growth disturbance (current height SDS < -2.5 and parental adjusted height SDS < -1) in short children born small for gestational age (SGA), with a birth weight and/or length below -2 SD, who failed to show catch-up growth (HV SDS < 0 during the last year) by 4 years of age or later, - Noonan syndrome (NS).

• Triheptanoin

  • Treatment of patients with Long chain fatty acid oxidation disorder (LC-FAOD).
• Venglustat

Other metabolism and Endocrinology

• Alpelisib

  • Vijoice is indicated for the treatment of adult and paediatric patients aged 2 years and older with severe or life-threatening manifestations of PIK3CA-related overgrowth spectrum (PROS) who require systemic therapy.
• 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

  • Extension application to introduce a new pharmaceutical form (tablet), associated with four new strengths (1,5 mg, 4 mg, 9mg and 25 mg) and a new route of administration (oral use).

• Setmelanotide

  • Imcivree is indicated for the treatment of obesity and the control of hunger in adults and children 4 years of age and above with acquired hypothalamic obesity (aHO) due to hypothalamic injury or impairment.
• Setrusumab

• Sparsentan

  • Sparsentan monotherapy for treatment of primary and genetic Focal segmental glomerulosclerosis in children over 8 years of age, adolescents, adults and elderly.
• Tradipitant
• 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