|
Drug
|
Condition
|
Approvals
|
Metabolic
|
|
|
|
|
|
Aminoacid Disorders
|
|
|
Benzoate and phenylacetate
|
Hyperammonemia of urea cycle disorders
|
FDA
|
|
Sodium phenylbutyrate
|
Urea cycle disorders
|
FDA, EMA
|
|
Carglumic acid
|
N-acetylglutamate synthetase deficiency
|
FDA, EMA
|
|
Betaine
|
Homocystinuria
|
FDA, EMA
|
|
Sapropterin
|
Hyperphenylalaninemia, Tetrahydrobiopterin deficiency
|
FDA, EMA, China
|
|
Pegvaliase
|
Phenylketonuria
|
FDA, EMA
|
|
Nitisinone
|
Tyrosinemia type 1
|
FDA, EMA
|
|
|
|
|
|
|
Lysosomal Storage Diseases
|
|
|
Miglustat
|
Gaucher disease
|
FDA, EMA, China
|
|
Eliglustat
|
Gaucher disease type 1
|
FDA, EMA
|
|
Velaglucerase alfa
|
Gaucher disease type 1
|
FDA, EMA
|
|
Imiglucerase
|
Gaucher disease type 1 or Type 3
|
FDA, EMA, China
|
|
Taliglucerase
|
Gaucher disease
|
FDA, EMA
|
|
Agalsidase beta
|
Fabry disease (alphagalactosidase A deficiency)
|
EMA, China
|
|
Agalsidase alfa
|
Fabry disease (alphagalactosidase A deficiency)
|
EMA, China
|
|
Migalastat
|
Fabry disease
|
FDA, EMA
|
|
Sebelipase alfa
|
Lysosomal acid lipase deficiency, Wolman disease, Cholesteryl ester storage disease
|
FDA, EMA
|
|
Alglucosidase alfa
|
Pompe disease
|
FDA, EMA, China
|
|
Velmanase alfa
|
Alpha mannosidosis
|
EMA
|
|
Laronidase
|
Mucopolysaccharidosis I (Iduronidase deficiency)
|
EMA, China
|
|
Idursulfase
|
Hunter syndrome (Mucopolysaccharidosis II)
|
FDA, EMA, China
|
|
Elosulfase alfa
|
Mucopolysaccharidosis IV (Morquio A syndrome)
|
FDA, EMA, China
|
|
Galsulfase
|
Mucopolysaccharidosis VI (Maroteaux-Lamy syndrome)
|
EMA
|
|
Vestronidase alfa
|
Mucopolysaccharidosis VII (Sly syndrome)
|
FDA, EMA
|
|
Cerliponase alfa
|
Neuronal ceroid lipofuscinosis type 2
|
FDA, EMA
|
|
Cysteamine
|
Nephropathic cystinosis
|
FDA, EMA
|
|
Cysteamine (enteric coated)
|
Nephropathic cystinosis
|
FDA, EMA
|
|
Cysteamine hydrochloride eyedrops
|
Corneal crystal accumulation in cystinosis
|
FDA, EMA
|
|
|
|
|
|
|
Cholesterol, Lipid, Fatty Acid Disorders
|
|
|
Evolocumab
|
Homozygous familial hypercholesterolemia
|
FDA, EMA, China
|
|
Rosuvastatin calcium
|
Homozygous hypercholesterolemia
|
FDA, China
|
|
Lomitapide
|
Homozygous familial hypercholesterolemia
|
FDA, EMA
|
|
Cholic acid
|
Cholesterol and bile acid synthesis defects
|
FDA, EMA, China
|
|
Chenodeoxycholic acid
|
Cerebrotendinous xanthomatosis
|
EMA
|
|
Volanesorsen
|
Familial chylomicronemia syndrome
|
EMA
|
|
Tocofersolan
|
Congenital or hereditary chronic cholestasis
|
EMA
|
|
|
|
|
|
|
Other Metabolic Disorders
|
|
|
Asfotase alfa
|
Pediatric onset hypophosphatasia
|
FDA, EMA
|
|
Burosumab-twza
|
Hypophosphatemic rickets (x-Linked)
|
FDA, EMA
|
|
Calcium acetate
|
Hyperphosphatemia in renal failure
|
FDA
|
|
Alendronate
|
Osteogenesis imperfecta
|
China
|
|
Ascorbic acid
|
Scurvy
|
FDA
|
|
Thiamine
|
Metabolic acidosis
|
EMA
|
|
Trisodium citrate
|
Metabolic acidosis
|
EMA
|
|
Levocarnitine
|
Genetic carnitine deficiency
|
FDA, China
|
|
Triheptanoin
|
Fatty acid oxidation disorders
|
FDA
|
|
Riboflavin
|
Acyl Coenzyme A dehydrogenase deficiency
|
EMA
|
|
Uridine triacetate
|
Hereditary orotic aciduria
|
FDA
|
|
Potassium citrate
|
Prevention of uric acid nephrolithiasis.
|
FDA
|
|
Tiopronin
|
Prevention of cystine nephrolithiasis (cystinuria)
|
FDA
|
|
Penicillamine
|
Wilson disease
|
China
|
|
Trientine HCl
|
Wilson disease intolerant of penicillamine
|
FDA, EMA
|
|
Zinc acetate
|
Wilson disease
|
FDA, EMA
|
|
Hydroxocobalamin
|
Acute cyanide poisoning, Cobalamin defects
|
FDA
|
|
Sodium nitrite/sodium thiosulfate
|
Cyanide poisoning, Calciphylaxis
|
FDA
|
|
Acetylcysteine
|
Acetaminophen overdose
|
FDA
|
|
Allopurinol sodium
|
Preservation of cadaveric kidneys for transplantation
|
FDA
|
|
|
|
|
Neurologic
|
|
|
|
|
|
General
|
|
|
Inotersen
|
Hereditary transthyretin amyloidosis polyneuropathy
|
FDA, EMA
|
|
Tafamidis
|
Transthyretin amyloidosis
|
FDA, EMA
|
|
Patisiran sodium
|
Hereditary transthyretin amyloidosis
|
FDA, EMA
|
|
Teriflunomide
|
Multiple Sclerosis
|
EMA, China
|
|
Fingolimod HCl
|
Multiple Sclerosis
|
EMA, China
|
|
Siponimod
|
Multiple Sclerosis
|
China
|
|
Rasagiline
|
Parkinson Disease (Young and Early-onset)
|
EMA, China
|
|
Selegiline
|
Parkinson Disease (Young and Early-onset)
|
FDA, EMA, China
|
|
Pramipexole
|
Parkinson Disease (Young and Early-onset)
|
EMA, China
|
|
Carbidopa/Levodopa
|
Parkinson Disease (Young and Early-onset); biopterin defects
|
FDA, EMA
|
|
Pitolisant
|
Narcolepsy with or without cataplexy
|
FDA, EMA
|
|
Sodium oxybate
|
Narcolepsy with cataplexy
|
FDA, EMA
|
|
Deutetrabenazine
|
Huntington Disease
|
China
|
|
Tetrabenazine
|
Huntington Disease
|
FDA, EMA
|
|
Baclofen
|
Dystonia, Spasticity
|
FDA
|
|
Capsaicin
|
Postherpetic neuralgia
|
FDA
|
|
Naltrexone
|
Postherpetic neuralgia
|
FDA
|
|
Ziconotide
|
Chronic pain requiring intrathecal analgesia
|
EMA
|
|
Everolimus
|
Tuberous Sclerosis Complex
|
FDA, EMA
|
|
Folic acid
|
Spina bifida (prevention)
|
EMA
|
|
Biotin
|
Biotinidase deficiency
|
China
|
|
|
|
|
|
|
Epilepsy
|
|
|
Vigabatrin
|
Infantile spasms
|
FDA, EMA
|
|
Rufinamide
|
Lennox-Gastaut syndrome
|
FDA, EMA
|
|
Cannabidiol
|
Lennox-Gastaut syndrome and Dravet syndrome
|
FDA, EMA
|
|
Stiripentol
|
Severe myoclonic epilepsy in infancy (Dravet syndrome)
|
FDA, EMA
|
|
Midazolam
|
Status epilepticus
|
FDA
|
|
Levetiracetam
|
Juvenile myoclonic epilepsy, Generalized epilepsy
|
EMA
|
|
Clobazam
|
Complex and rare disease epilepsy
|
FDA
|
|
Lamotrigine
|
Complex and rare disease epilepsy
|
FDA
|
|
Topiramate
|
Complex and rare disease epilepsy
|
FDA
|
|
|
|
|
|
|
Neuromuscular Diseases
|
|
|
Gabapentin
|
Amyotrophic lateral sclerosis
|
FDA
|
|
Riluzole
|
Amyotrophic lateral sclerosis
|
FDA, EMA, China
|
|
Radicava
|
Amyotrophic lateral sclerosis
|
China
|
|
Pyridostigmine Bromide
|
Myasthenia gravis
|
China
|
|
Amifampridine
|
Lambert-Eaton myasthenic syndrome
|
EMA
|
|
Mexiletine hcl
|
Non-dystrophic myotonic disorders
|
EMA
|
|
Nusinersen sodium
|
5q Spinal Muscular Atrophy
|
FDA, EMA, China
|
|
|
|
|
Hematologic
|
|
|
|
|
|
Coagulation Defects
|
|
|
Octocog alpha
|
Hemophilia A (Factor VIII deficiency)
|
EMA
|
|
Rurioctocog alfa pegol
|
Hemophilia A (Factor VIII deficiency)
|
EMA
|
|
Lonoctocog alfa
|
Hemophilia A (Factor VIII deficiency)
|
EMA
|
|
Emicizumab
|
Hemophilia A (Factor VIII deficiency)
|
FDA, EMA, China
|
|
Damoctocog alfa pegol
|
Hemophilia A (Factor VIII deficiency)
|
EMA
|
|
Turoctocog alpha
|
Hemophilia A (Factor VIII deficiency)
|
EMA
|
|
Simoctocog alfa
|
Hemophilia A (Factor VIII deficiency)
|
EMA
|
|
Moroctocog alpha
|
Hemophilia A (Factor VIII deficiency)
|
EMA
|
|
Desmopressin acetate
|
Hemophilia
|
FDA, EMA
|
|
Recombinant Factor VIII
|
Hemophilia A (Factor VIII deficiency)
|
EMA, China
|
|
Efmoroctocog alfa
|
Hemophilia A (Factor VIII deficiency)
|
EMA
|
|
Factor VIII/ von Willebrand factor
|
von Willebrand disease, Hemophilia A
|
EMA
|
|
Vonicog alfa
|
von Willebrand disease
|
EMA
|
|
Eftrenonacog alfa
|
Hemophilia B
|
EMA
|
|
Albutrepenonacog alfa
|
Hemophilia B
|
EMA
|
|
Nonacog alpha
|
Hemophilia B (Factor IX deficiency)
|
EMA
|
|
Human coagulation factor IX
|
Hemophilia B (Factor IX deficiency)
|
EMA
|
|
Nonacog beta pegol
|
Hemophilia B (Factor IX deficiency)
|
EMA
|
|
Nonacog gamma
|
Hemophilia B (Factor IX deficiency)
|
EMA
|
|
Recombinant Factor IX
|
Hemophilia B (Factor IX deficiency)
|
EMA, China
|
|
Eptacog alpha (activated)
|
Hemophilia (Factor VII deficiency)
|
EMA
|
|
Recombinant Factor VIIa
|
Hemophilia (Factor VII deficiency)
|
EMA
|
|
Human coagulation factor X
|
Factor X deficiency
|
EMA
|
|
Catridecacog
|
Factor XIII A-subunit deficiency
|
EMA
|
|
Human protein c
|
Protein C deficiency
|
EMA
|
|
|
|
|
|
|
Anemias
|
|
|
Hydroxyurea
|
Sickle cell anemia
|
FDA
|
|
Epoetin alfa
|
Anemia of end-stage renal disease
|
FDA
|
|
Eltrombopag
|
Idiopathic thrombocytopenic purpura, Aplastic anemia
|
FDA, EMA
|
|
Deferasirox
|
Beta thalassemia major
|
FDA, EMA, China
|
|
|
|
|
|
|
Other Hematologic Disorders
|
|
|
Methylene blue injection
|
Congenital and acquired methemoglobinemia
|
FDA
|
|
Hemin
|
Acute intermittent porphyria
|
FDA
|
|
Afamelanotide
|
Erythropoietic protoporphyria
|
FDA, EMA
|
|
Siltuximab
|
Multicentric Castleman’s disease
|
FDA, EMA
|
|
Anagrelide hydrochloride
|
Essential thrombocythemia
|
FDA, EMA
|
|
Ravulizumab
|
Paroxysmal nocturnal hemoglobinuria
|
FDA, EMA
|
|
Macapegfilgrastim
|
Severe congenital neutropenia
|
China
|
|
Busulfan
|
Conditioning for hematopoietic stem cell transplant
|
FDA, EMA
|
|
Thiotepa
|
Conditioning for hematopoietic stem cell transplant
|
FDA, EMA
|
|
Deferiprone
|
Iron overload
|
FDA, EMA
|
|
Caplacizumab
|
Acquired thrombotic thrombocytopenic purpura
|
FDA, EMA
|
|
Romiplostim
|
Immune (idiopathic) thrombocytopenic purpura
|
FDA, EMA
|
|
Ropeginterferon alfa-2b
|
Polycythemia vera
|
EMA
|
|
Ruxolitinib
|
Polycythemia vera
|
FDA, EMA
|
|
Immunoglobulin infusion
|
Agammaglobulinemia
|
China
|
|
|
|
|
Anti-inflammatory
|
|
|
|
|
Rheumatoid Arthritis
|
|
|
Methotrexate
|
Juvenile rheumatoid arthritis
|
FDA, EMA
|
|
Etanercept
|
Juvenile rheumatoid arthritis
|
FDA, EMA
|
|
Methylprednisolone
|
Juvenile rheumatoid arthritis
|
EMA
|
|
Adalimumab
|
Juvenile rheumatoid arthritis, Pediatric ulcerative colitis
|
FDA
|
|
Infliximab
|
Crohn's disease, Juvenile rheumatoid arthritis, Sarcoidosis
|
FDA
|
|
Tocilizumab
|
Pediatric polyarticular juvenile arthritis
|
FDA, EMA
|
|
Abatacept
|
Polyarticular juvenile idiopathic arthritis
|
EMA
|
|
Golimumab
|
Polyarticular juvenile idiopathic arthritis
|
FDA, EMA
|
|
|
|
|
|
|
Gastrointestinal Inflammation
|
|
|
Mesalamine; 5-aminosalicylic acid
|
Ulcerative colitis
|
FDA
|
|
Obeticholic acid
|
Primary biliary cholangitis
|
FDA, EMA
|
|
Tocofersolan
|
Hereditary chronic cholestasis
|
EMA
|
|
|
|
|
|
|
Angioedema
|
|
|
C1 inhibitor(human)
|
Hereditary angioedema
|
EMA
|
|
Icatibant acetate
|
Hereditary angioedema
|
FDA, EMA
|
|
Lanadelumab
|
Hereditary angioedema
|
FDA, EMA, China
|
|
Danazol
|
Hereditary angioedema
|
China
|
|
Tranexamic acid
|
Hereditary angioedema
|
FDA, China
|
|
C1-esterase-inhibitor, human
|
Angioedema due to C1 esterase inhibitor deficiency
|
FDA
|
|
Conestat alfa
|
Angioedema due to C1 esterase inhibitor deficiency
|
EMA
|
|
|
|
|
|
|
Other Inflammatory Disorders
|
|
|
Colchicine
|
Multiple sclerosis, Behcet's disease, Familial Mediterranean fever
|
FDA, China
|
|
Eculizumab
|
Dermatomyositis, Atypical hemolytic uremic syndrome, Neuromyelitis Optica, Paroxysmal nocturnal hemoglobinuria, Myasthenia gravis
|
FDA, EMA, China
|
|
Rituximab
|
Anti-neutrophil vasculitis, Wegener’s granulomatosis, Churg-Strauss Syndrome
|
FDA
|
|
Canakinumab
|
Familial Mediterranean fever, Cryopyrin fevers
|
FDA, EMA, China
|
|
IL-1 Receptor antagonist anakinra
|
Still's disease, Systemic juvenile arthritis
|
FDA, EMA
|
|
Cenegermin
|
Neurotrophic keratitis
|
FDA, EMA
|
|
Ciclosporin
|
Vernal keratoconjunctivitis
|
EMA
|
|
Dexamethasone
|
Non-infectious uveitis
|
FDA, EMA
|
|
Rilonacept
|
Cryopyrin-associated periodic syndromes
|
FDA, EMA
|
|
|
|
|
Endocrine
|
|
|
|
|
Somatropin for injection
|
Growth hormone deficiency in children
|
FDA, EMA
|
|
Octreotide
|
Acromegaly
|
FDA
|
|
Lanreotide
|
Acromegaly
|
FDA
|
|
Pegvisomant
|
Acromegaly
|
FDA, EMA
|
|
Pasireotide
|
Acromegaly and Cushing's syndrome
|
FDA, EMA
|
|
Osilodrostat
|
Endogenous Cushing’s syndrome
|
FDA, EMA
|
|
Ketoconazole
|
Endogenous Cushing’s syndrome
|
EMA
|
|
Hydrocortisone
|
Adrenal insufficiency
|
FDA, EMA, China
|
|
Human chorionic gonadotropin
|
Idiopathic Hypogonadotropic Hypogonadism
|
EMA, China
|
|
Gonadotropin-releasing hormone
|
Idiopathic Hypogonadotropic Hypogonadism
|
EMA, China
|
|
Mecasermin
|
Primary insulin-like growth factor-1 deficiency
|
FDA, EMA
|
|
Calcitonin-human for injection
|
Paget's disease (osteitis deformans)
|
FDA
|
|
Parathyroid hormone
|
Hypoparathyroidism
|
FDA, EMA
|
|
Tasimelteon
|
Non-24-hour sleep-wake disorder
|
FDA, EMA
|
|
Metreleptin
|
Leptin deficiency in lipodystrophy patients
|
FDA, EMA
|
|
Metreleptine
|
Familial partial lipodystrophy
|
EMA
|
|
|
|
|
Pulmonary
|
|
|
|
|
|
Pulmonary Arterial Hypertension
|
|
|
Macitentan
|
Pulmonary arterial hypertension
|
FDA, EMA, China
|
|
Tadalafil
|
Pulmonary arterial hypertension
|
FDA, EMA
|
|
Ambrisentan
|
Pulmonary arterial hypertension
|
FDA, EMA, China
|
|
Nitric oxide
|
Pulmonary arterial hypertension
|
FDA, EMA
|
|
Sildenafil
|
Pulmonary arterial hypertension
|
EMA, China
|
|
Bosentan monohydrate
|
Pulmonary arterial hypertension, systemic sclerosis
|
FDA, EMA, China
|
|
Selexipag
|
Pulmonary arterial hypertension
|
FDA, EMA, China
|
|
Iloprost
|
Pulmonary arterial hypertension
|
FDA, EMA, China
|
|
Parenteral treprostinil
|
Pulmonary arterial hypertension
|
FDA, EMA, China
|
|
Riociguat
|
Thromboembolic pulmonary hypertension and Pulmonary arterial hypertension
|
FDA, EMA, China
|
|
|
|
|
|
|
Cystic Fibrosis
|
|
|
Mannitol
|
Cystic fibrosis
|
FDA, EMA
|
|
Ivacaftor
|
Cystic fibrosis
|
FDA, EMA
|
|
Tezacaftor/ivacaftor
|
Cystic fibrosis
|
FDA, EMA
|
|
Tobramycin
|
Cystic fibrosis
|
FDA, EMA
|
|
Aztreonam
|
Cystic fibrosis
|
FDA, EMA
|
|
Colistimethate sodium
|
Cystic fibrosis
|
EMA
|
|
Lumacaftor / ivacaftor
|
Cystic fibrosis
|
FDA, EMA
|
|
Levofloxacin
|
Cystic fibrosis
|
EMA
|
|
|
|
|
|
|
Other Pulmonary Disorders
|
|
|
Pirfenidone
|
Idiopathic Pulmonary Fibrosis
|
FDA, EMA
|
|
Nintedanib
|
Idiopathic Pulmonary Fibrosis
|
FDA, EMA, China
|
|
Caffeine citrate
|
Primary apnea of premature newborns
|
FDA, EMA
|
|
|
|
|
Immunologic
|
|
|
|
|
Pegademase bovine
|
Enzyme replacement for Adenosine deaminase deficiency (ADA)
|
FDA
|
|
CD34+ cells transduced with ADA cDNA
|
Severe combined immunodeficiency, Adenosine deaminase deficiency (ADA)
|
EMA
|
|
Interferon gamma 1-b
|
Chronic granulomatous disease
|
FDA
|
|
Tacrolimus
|
Prophylaxis of graft-versus-host-disease, Graft rejection
|
FDA
|
|
Sirolimus
|
Lymphangioleiomyomatosis, Tuberous sclerosis
|
FDA, EMA
|
|
|
|
|
Miscellaneous
|
|
|
|
|
Pentamidine isethionate
|
Pneumocystis carinii pneumonia
|
FDA
|
|
Cromolyn sodium
|
Mastocytosis
|
FDA
|
|
Amiodarone
|
Ventricular tachycardia
|
FDA
|
|
Autologous human corneal stem cells
|
Limbal stem cell deficiency
|
EMA
|
|
Voretigene neparvovec
|
Inherited retinal dystrophy
|
FDA, EMA
|
|
Teduglutide
|
Short bowel syndrome
|
FDA, EMA
|
|
Defibrotide
|
Hepatic veno-occlusive disease, Sinusoidal obstruction
|
FDA, EMA
|
|
Proteolytic enzymes with bromelain
|
Deep partial- and full-thickness thermal burns
|
EMA
|
|
Tolvaptan
|
Autosomal dominant polycystic kidney disease
|
FDA, EMA
|
|
Ibuprofen
|
Patent ductus arteriosus
|
FDA, EMA
|
|
Dexrazoxane
|
Anthracycline extravasation
|
FDA, EMA
|