Metabolism and Endocrinology

Bowel diseases

• Apraglutide
• Lubiprostone

Diabetes

• Bexagliflozine
• Cagrilintide / semaglutide

• Canagliflozine

  • Invokana is indicated for the treatment of adults 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 and in addition to other medicinal products for the treatment of diabetes
  • 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.
• Canagliflozine / metformine ^IND
• Dapagliflozin / saxagliptin / metformin

• Dapagliflozine

  • Extension of indication for Forxiga / Edistride to include treatment of children aged 10 years and adolescents with T2DM based on the results from studies
  • Extension of Indication to include type 1 diabetes mellitus as an adjunct to insulin in patients with BMI ≥ 27 kg/m2, when insulin alone does not provide adequate glycaemic control despite optimal insulin therapy.

• Dasiglucagon

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

• Empagliflozine

  • Extension of indication for Jardiance to include treatment of children aged 10 years and above with type 2 diabetes.
  • diabetes mellitus type 1, adjunct to insulin
• Empagliflozine / metformine ^IND

• Ertugliflozin

  • Steglatro is geïndiceerd bij volwassenen van 18 jaar en ouder met type 2-diabetes mellitus als aanvulling op een dieet en lichaamsbeweging ter verbetering van de bloedglucoseregulatie: 1) als monotherapie bij patiënten bij wie het gebruik van metformine ongeschikt wordt geacht vanwege intolerantie of contra-indicaties, 2) als aanvulling op andere geneesmiddelen voor de behandeling van diabetes.
  • 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.
• Ertugliflozin / metformin
• Ertugliflozin / sitagliptin

• Exenatide

  • Extension of indication to include the treatment of type 2 diabetes in adolescents and children aged 10 years and above
  • Bydureon is a diabetes medicine used together with other diabetes medicines including long-acting insulin to treat adults with type 2 diabetes whose blood glucose (sugar) levels are not adequately controlled with the other medicines
  • Diabetes type 2

• Finerenon

  • Kerendia is indicated for the treatment of chronic kidney disease (stage 3 and 4 with albuminuria) associated with type 2 diabetes in adults.
  • Extension of indication to include the treatment of chronic kidney disease (CKD) and for the prevention of cardiovascular (CV) events in adults with CKD (regardless of the stage of albuminuria) associated with type 2 diabetes
• Glibenclamide ^IND

• Glucagon

  • Behandeling van ernstige hypoglykemie bij volwassenen, adolescenten en kinderen in de leeftijd van 4 jaar en ouder met diabetes mellitus.
  • Baqsimi is indicated for the treatment of severe hypoglycaemia in adults, adolescents, and children aged 1 year and over with diabetes mellitus.

• Insulin aspart

  • The treatment of diabetes mellitus in adults, adolescents and children aged 1 year and above.
  • The treatment of diabetes mellitus in adults, adolescents and children aged 1 year and above.
• Insulin efsitora alfa
• Insulin glargine ^IND
• Insulin icodec

• 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 lispro ^IND
• Insulin lispro (ultra rapid formulation)
• Insulin oral
• Insuline voor inhalatie

• Liraglutide

  • Extension of Indication to include treatment of children and adolescents (age 10-17 years) with type 2 diabetes.
• Orforglipron
• Polyethylene glycol loxenatide
• Regulatoire T-cellen (TREGS)
• Reparixin

• Semaglutide

  • Rybelsus is indicated for the treatment of adults with insufficiently controlled type 2 diabetes mellitus to improve glycaemic control as an adjunct to diet and exercise as monotherapy when metformin is considered inappropriate due to intolerance or contraindications or in combination with other medicinal products for the treatment of diabetes.
  • Ozempic is geïndiceerd voor de behandeling van volwassenen met onvoldoende gereguleerde diabetes mellitus type 2 als toevoeging aan dieet en lichaamsbeweging als monotherapie wanneer metformine ongeschikt wordt geacht als gevolg van intolerantie of contra-indicaties en in aanvulling op andere geneesmiddelen voor de behandeling van diabetes.

• Sotagliflozine

  • Patients With Type 2 Diabetes Mellitus and Moderate Renal Impairment Who Have Inadequate Glycemic Control.
  • Zynquista is indicated as an adjunct to insulin therapy to improve glycaemic control in adults with type 1 diabetes mellitus with a Body Mass Index (BMI) ≥ 27 kg/m2, who have failed to achieve adequate glycaemic control despite optimal insulin therapy.

• Tarcocimab tedromer

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

• Tirzepatide

  • Voor de behandeling van volwassenen met onvoldoende gereguleerde diabetes mellitus type 2, als aanvulling op dieet en lichaamsbeweging • als monotherapie wanneer metformine niet geschikt blijkt te zijn vanwege intolerantie of contra-indicaties • in aanvulling op andere geneesmiddelen voor de behandeling van diabetes.
  • 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

• Mercaptamine

  • Cystadrops is geïndiceerd voor gebruik bij volwassenen en kinderen vanaf 2 jaar met cystinose voor de behandeling van afzettingen van cystinekristallen in het hoornvlies.
• Pegpleranib
• Teprotumumab

Hormonal disorders

• Cinacalcet
• Elagolix
• Enclomifene
• Macimorelin
• Pasireotide LAR
• Prasterone
• Recombinant humaan parathyroid hormoon

• Relugolix

  • Treatment of uterine fibroids.

Liver diseases

• Arachidyl amido cholanoic acid
• Cenicriviroc
• Elafibranor
• Givosiran
• Linerixibat

• Maralixibat

  • Treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) 2 months of age and older.
  • Extension of indication to include treatment of Progressive Familial Intrahepatic Cholestasis (PFIC) in patients 3 months of age and older.
  • Progressive Familial Intrahepatic Cholestasis Type 2 Treatment of Progressive Familial Intrahepatic Cholestasis Type 2
• Norucholzuur

• Obeticholic acid

  • OCALIVA is geïndiceerd voor de behandeling van primaire biliaire cholangitis (ook primaire biliaire cirrose genaamd) in combinatie met ursodeoxycholzuur (UDCA) bij volwassenen met een ontoereikende respons op UDCA of als monotherapie bij volwassenen die UDCA niet kunnen verdragen.
  • Fibrosis due to Non-Alcoholic Steatohepatitis (NASH)

• Odevixibat

  • Treatment of progressive familial intrahepatic cholestasis (PFIC) in patients aged 6 months or older.
  • Kayfanda is indicated for the treatment of cholestatic pruritus in Alagille syndrome (ALGS) in patients aged 6 months or older.
  • 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

• Afamelanotide

  • Extension of indication for the prevention of phototoxicity in adolescent patients (12 to under 18 years of age) with erythropoietic protoporphyria (EPP).

• Arimoclomol

  • Treatment of Niemann-Pick disease type C (NPC) in patients aged 6 months and older in combination with miglustat.
• Asfotase alfa
• Atidarsagene autotemcel ^IND
• Autologous CD34+ cell enriched population that contains hematopoietic stem and progenitor cells transduced ex vivo using a lentiviral vector encoding the human arylsulfatase A gene
• Avalglucosidase alfa
• BCAA-vrije aminozuren
• Bardoxolone Methyl
• Betaine ^G

• Burosumab

  • Crysvita is geïndiceerd voor de behandeling van X-gebonden hypofosfatemie (XLH) met radiografisch bewijs van botziekte bij kinderen in de leeftijd van 1 jaar en ouder en adolescenten met een groeiend skelet.
  • Crysvita is indicated for the treatment of X-linked hypophosphataemia, in children and adolescents aged 1 to 17 years with radiographic evidence of bone disease, and in adults.
  • 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)
• Cerliponase alfa
• Chenodeoxycholzuur
• Cholic acid
• Cipaglucosidase alfa
• Clemidsogene lanparvovec
• Coördinatieverbinding van ijzercitraat
• Crinecerfont
• Cyclodextrine (Hydroxypropyl betadex)

• Dasiglucagon

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

• Elamipretide

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

• Empagliflozine

  • Jardiance is indicated in adults for the treatment of chronic kidney disease.
• Encaleret
• Eneboparatide
• Fexapotide
• Fezolinetant
• Fosdenopterin
• Garetosmab

• Govorestat

  • Patients with Classic Galactosaemia aged 2 to 65 years.
  • Sorbitol dehydrogenase deficiency.

• Hydrocortison

  • Treatment of congenital adrenal hyperplasia (CAH) in adolescents aged 12 years and over and adults.
• Hydrocortison slow-release ^IND
• Idursulfase-IT
• Kaliumcitraat / kaliumwaterstofcarbonaat

• 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
• Leriglitazone
• Levamisole (hydrochloride)
• Levoketoconazole

• 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.
  • Extension of indication to include treatment as an adjunct to a healthy nutrition and physical activity counselling for weight management in adolescent patients from the age of 12 years and above with body weight above 60kg and obesity (BMI corresponding to ≥30kg/m2 for adults).
• Lixivaptan

• Lonafarnib

  • Zokinvy is indicated for the treatment of patients 12 months of age and older with a genetically confirmed diagnosis of Hutchinson-Gilford progeria syndrome or a processing-deficient progeroid laminopathy associated with either a heterozygous LMNA mutation with progerin-like protein accumulation or a homozygous or compound heterozygous ZMPSTE24 mutation.
• Lonapegsomatropine ^IND
• Metreleptin

• Migalastat

  • Ziekte van Fabry voor patiënten van 16 jaar of ouder.
  • Galafold is indicated for long-term treatment of adults and adolescents aged 12-16 years and older with a confirmed diagnosis of Fabry disease (α-galactosidase A deficiency) and who have an amenable mutation.
  • 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
• Neod001
• Nitisinone ^IND

• Octreotide

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

• Osilodrostat

  • Treatment of endogenous Cushing’s syndrome in adolescents and children aged 6 years and older.
• Palopegteriparatide
• Palovarotene
• Paltusotine
• Pariglasgene brecaparvovec
• Patiromer
• Pegunigalsidase alfa

• Pegvaliase

  • Palynziq is geïndiceerd voor de behandeling van patiënten met fenylketonurie (PKU) van 16 jaar en ouder met onvoldoende onder controle gebrachte fenylalanineconcentraties in het bloed (concentraties fenylalanine in het bloed hoger dan 600micromol/l) ondanks voorgaande behandeling met beschikbare behandelopties.
  • 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.
• Reloxaliase
• Sapropterin ^G
• Sebelipase alfa
• Sepiapterine
• Sodium benzoate
• Sodium zirconium cyclosilicate

• Somapacitan

  • Sogroya is indicated for the replacement of endogenous growth hormone (GH) in children aged 3 years and above and adolescents with growth failure due to growth hormone deficiency (paediatric GHD), and in adults with growth hormone deficiency (adult GHD)
  • 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).

• Somatrogon

  • Ngenla is geïndiceerd voor de behandeling van kinderen en adolescenten vanaf 3 jaar met een groeistoornis als gevolg van onvoldoende secretie van groeihormoon.
  • Somatrogon monotherapy for treatment of growth hormone deficiency in adults.

• Sparsentan

  • Filspari is indicated for the treatment of adults with primary immunoglobulin A nephropathy (IgAN) with a urine protein excretion >1.0 g/day (or urine protein-to-creatinine ratio ≥0.75 g/g).

• Tenapanor

  • Hyperphosphatemia
• Tiomolibdate choline
• Trientine

• Triheptanoin

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

• Volanesorsen

  • Familial Partial Lipodystrophy

Other metabolism and Endocrinology

• Abaloparatide

  • Treatment of osteoporosis in postmenopausal women at increased risk of fracture.
  • Treatment of osteoporosis in postmenopausal women at increased risk of fracture

• 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.
• Autologous human chondrocytes in vitro expanded

• Burosumab

  • Extension of indication to include treatment of FGF23-related hypophosphataemia in tumour-induced osteomalacia (TIO) associated with phosphaturic mesenchymal tumours that cannot be curatively resected or localised in patients aged 1 year and over.
• Carbetocine
• Corifollitropin alfa ^IND
• Denatonium Acetate Monohydrate
• Diazoxide choline (controlled-release tablet)
• Drospirenon / estetrol, monohydraat
• Ersodetug ^IND
• Estetrol

• Etelcalcetide

  • Etelcalcetide in combination with standard of care for treatment of Hyperparathyroidism secondary in children and adolescents with chronic kidney disease receiving maintenance hemodialysis.
• Ganirelix ^G

• Infigratinib

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

• Linzagolix

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

• Pegzilarginase

  • Loargys is indicated for the treatment of arginase 1 deficiency (ARG1‑D), also known as hyperargininemia, in adults, adolescents and children aged 2 years and older.
  • Pegzilarginase monotherapy for treatment of Arginase 1 deficiency in newborns, infants and toddlers.
• Relugolix / estradiol / norethisterone acetaat ^IND
• Romosozumab

• Semaglutide

  • Wegovy is indicated as an adjunct to a reduced-calorie diet and increased physical activity for weight management in adolescents ages 12 years and above with obesity and body weight above 60 kg. Treatment with Wegovy should be discontinued and re-evaluated if adolescent patients have not reduced their BMI by at least 5% after 12 weeks on the 2.4 mg or maximum tolerated dose.
  • For weight management in people with obesity or who are overweight and have other related conditions.
  • Extension of indication to include risk reduction of major adverse cardiovascular events (cardiovascular death, non-fatal myocardial infarction, or non-fatal stroke) in adults with established cardiovascular disease and BMI ≥27 kg/m2.
  • 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 associated with genetically confirmed loss-of-function biallelic pro-opiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years of age and above.
  • IMCIVREE is indicated for the treatment of obesity and the control of hunger associated with genetically confirmed Bardet‑Biedl syndrome (BBS), loss-of-function biallelic pro-opiomelanocortin (POMC), including PCSK1, deficiency or biallelic leptin receptor (LEPR) deficiency in adults and children 6 years of age and above.
  • 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.
  • 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

• Somapacitan

  • Sogroya is indicated for the replacement of endogenous growth hormone (GH) in adults with growth hormone deficiency (AGHD).

• Sparsentan

  • Sparsentan monotherapy for treatment of primary and genetic Focal segmental glomerulosclerosis in children over 8 years of age, adolescents, adults and elderly.
• Spheroids of human autologous matrix-associated chondrocytes ^IND

• Tirzepatide

  • Uitbreiding van de indicatie: voor gewichtsbeheersing bij volwassenen met obesitas of overgewicht met gewicht gerelateerde co-morbide aandoeningen.
• Tradipitant

• Vosoritide

  • Voxzogo is indicated for the treatment of achondroplasia in patients 4 months of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing.
  • Voxzogo is indicated for the treatment of achondroplasia in patients 2 years of age and older whose epiphyses are not closed. The diagnosis of achondroplasia should be confirmed by appropriate genetic testing.
• Xylitol/l-carnitine/glucose

Unknown

• Dapagliflozine

  • Forxiga is indicated in adults for the treatment of chronic kidney disease.
• Levonorgestrel ^G

• Linzagolix

  • Yselty is indicated for treatment of moderate to severe symptoms of uterine fibroids in adult women of reproductive age.
• Lonapegsomatropin
• Lumasiran

• Osilodrostat

  • Isturisa is geïndiceerd voor de behandeling van het endogene syndroom van Cushing bij volwassenen.
• Relugolix / estradiol / norethisteronen
• Sofpironium bromide

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