Metabolism and Endocrinology

Bowel diseases

• Lubiprostone

Diabetes

• Bexagliflozine

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

• Liraglutide

  • Extension of Indication to include treatment of children and adolescents (age 10-17 years) with type 2 diabetes.
• 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.
• 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.
  • 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

• 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.
• Resmetirom
• Seladelpar

• Semaglutide

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

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

• 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.
  • Treatment of adrenal insufficiency (AI) in adolescents aged 12 years and over and adults
• Idursulfase-IT
• Kaliumcitraat / kaliumwaterstofcarbonaat
• L-acetylleucine
• 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
• Palopegteriparatide
• Palovarotene
• Paltusotine
• 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.
  • Extension of indication to include treatment of adolescent patients aged 12 to <16 years with PKU
• Rebisufligene etisparvovec

• Relacorilant

  • Cushingʼs Syndrome
• Reloxaliase
• Sapropterin ^G
• Sebelipase alfa
• Sepiapterin
• 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)
  • 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

  • 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
• 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

  • 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

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

• 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.
  • 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.
• 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.

• 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
• 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