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Indication List

Therapeutic List

AAT Congenital Deficiency
Achondroplasia
Acromegaly
Acute Disseminated Encephalomyelitis
Acute Lymphoblastic Leukemia
Acute Myeloid Leukemia Adult
Acute Radiation Syndrome
Addisons Disease
Adenosine Deaminase Deficiency
Adrenal Hyperplasia, Congenital
Adrenocortical Carcinoma (ACC)
Adrenoleukodystrophy X-Linked
Adult Growth Hormone Deficiency
AL Amyloidosis
Alagille Syndrome
Alexander Disease
Alopecia
Alpha 1 Antitrypsin Deficiency
Alpha Mannosidosis
Alport Syndrome
Altitude Sickness
Amoebiasis
Amyloid Neuropathy
Amyloidosis
Amyotrophic Lateral Sclerosis (ALS)
Anaemia in Chronic Kidney Disease (CKD) (Dialysis)
Anaemia, AIDS Related
Anaemia, Aplastic
Anaemia, Haemolytic
Anaemia, Megaloblastic
Anaemia, Other
Anal Dysplasia
Anaphylaxis
Aniridia
Anthrax Prophylaxis
Anthrax Treatment
Anti Thrombin III Deficiency
Apnoea of Prematurity
Arrhythmia, Ventricular
Arteriovenous Fistula
Arthritis, General
Arthritis, Rheumatoid
Aspergillosis Infections
Asthenia
Ataxia Telangiectasia
Autoimmune Hepatitis
Autosomal Recessive Polycystic Kidney Disease
B Cell Lymphomas
Bacteraemia
Bacterial Meningitis
Basal Cell Carcinoma (BCC)
Becker Muscular Dystrophy
Behcets Syndrome
Benign Hyperphenylalaninemia
Benign Recurrent Intrahepatic Cholestasis 1
Biliary Cancer
Bladder Cancer
Blepharospasm
Blood Substitution
Bone Cancer (Osteosarcoma)
Bone Marrow Transplantation
Botulism Treatment
Bowel Clearance
Brain Cancer
Brain Metastases
Brain Tumor Adult
Bronchiectasis Oligospermia
Bronchiolitis Obliterans
Bronchitis, Chronic
Bronchopulmonary Dysplasia
Buergers Disease
Bullous Pemphigoid
Cachexia (Wasting), AIDS Related
Cancer Related Bone Loss
Candidiasis Familial Chronic Mucocutaneous Autosomal Recessive
Carcinoid Syndrome
Cardiogenic Shock
Cardiopulmonary Bypass Surgery
Carnitine Transporter Deficiency
Castleman Disease
Cataract And Congenital Ichthyosis
Cerebral Oedema
Cerebral Palsy
Cerebrotendinous Xanthomatosis (CTX)
Cervical Cancer
Cervical Dystonia
Cestodes (Tapeworm) Infections
Chagas Disease
Charcot Marie Tooth Disease
Chemotherapy Induced Hyperuricaemia
Childhood Onset Schizophrenia
Cholelithiasis
Cholestasis
Cholesteryl Ester Storage Disease
Choroideremia
Chronic Fatigue Syndrome
Chronic Granulomatous Disease
Chronic Inflammatory Demyelinating Polyneuropathy (CIDP)
Chronic Kidney Disease (CKD)
Chronic Lymphocytic Leukemia
Churg Strauss Syndrome
Cias1 Associated Periodic Syndromes (CAPS)
Circadian Rhythm Sleep Disorders
Clostridium Difficile Associated Diarrhoea (CDAD)
CMV Infection Prophylaxis
CMV Infection, AIDS Related
CMV Infections
Cockayne Syndrome
Colorectal Cancer
Cold Agglutinin Disease
Congenital Adrenal Hyperplasia
Congenital Cytomegalovirus
Congenital Hepatic Fibrosis
Congenital Human Immunodeficiency Virus
Congenital Muscular Dystrophy
Congenital Sucrase Isomaltase Deficiency (CSID)
Congestive Heart Failure (CHF)
Conjunctivitis Ligneous
Conjunctivitis, Allergic
Coronary Artery Bypass Graft (CABG)
Crigler Najjar Syndrome
Crohns Disease
Cryptosporidiosis
Cushings Syndrome
Cutaneous T Cell Lymphoma
Cystic Fibrosis (CF)
Cystinuria
Cystitis

225Ac-lintuzumab (Actimab-A)
40K PEGylated recombinant factor IX (nonacog beta pegol)
AAV1-FS344 (gene therapy)
AAV2-REP1 (gene therapy)
AAV5-hFIX
AB103
AB569
ABBV-8E12/C2N-8E12 (recombinant human anti-tau antibody)
ABT-199 (venetoclax)
ABT-308/RPC-4046
ABT-414
acamprosate
acebilustat (CTX-4430)
aceneuramic acid (UX001)
ACER-001 (reformulated NaPBA)
ACER-002 (celiprolol)
Actemra (tocilizumab)
Actimab-A (225Ac-lintuzumab)
Actimmune (interferon gamma-1b)
Adempas (riociguat)
adenovirus delta 24-RGD (DNX-2401)
A-dmDT390-bisFv (UCHT1) (Resimmune)
Ad-RTS-IL-12
ADS-5102 (amantadine ER)
ADXS11-001
AEM-28
AeroVanc (vancomycin hydrochloride (inhalation))
afamelanotide (Scenesse)
Afinitor (everolimus)
AFM13
AG-120
AG-221
AG-348
aglatimagene besadenovec (AdV-TK gene therapy)
AGT-181
AGT-182/SHP631
AL3818 (anlotinib hydrochloride)
albutropin (TV-1106)
algenpantucel-L
alicaforsen
alisertib
ALK-001 (retinol deuterated)
allantonin (SD-101)
allopregnanolone (SAGE-547)
ALN-AT3SC (fitusiran)
ALN-TTR02 (siRNA inhibtior targeting TTR) (patisiran)
alpha-1 proteinase inhibitor (AAT)
Alpha1-HC
alvocidib
ALX-0081 (caplacizumab)
ALXN-1101
amantadine (OS-320)
amantadine ER (ADS-5102)
amatuximab (MORAb-009)
ambroxol (BEL-0218)
Amiket (amitriptyline and ketamine)
amitriptyline and ketamine (Amiket)
Ampligen (rintatolimid)
andexanet alfa
anetumab ravtansine
ANG1005 (paclitaxel trevatide)
anlotinib hydrochloride (AL3818)
anti-CD45 mAb 131I-BC8
anti-Ebola mAb
anti-myostatin adnectin-IgG2 (BMS-986089)
antineoplaston A10/antineoplaston AS2-2
anti-T-lymphocyte immune globulin
antroquinonol
APL-2 subcutaneous
apremilast (Otezla)
ARA 290
ARC-AAT
ARD-3150 (ciprofloxacin inhalation)
Arenegyr (NGF-hTNF)
ARG201 (type 1 native bovine skin collagen)
AriKayce (liposomal amikacin)
ARM210
ARQ-087
ARQ092
ARRY-797
arylsulfatase A (SHP611)
ASP0113
AST-OPC1
AT1001 (migalastat)
ATR-01
ATYR 1940 (recombinant human histidyl tRNA synthetase)
avatrombopag (E5501)
avelumab (PF-06834635)
AVI-7288
AVI-7537
avoralstat
AVXS-101
axalimogen filolisbac
AZD-1775
Azedra (iobenguane I-131)
Azixa (verobulin)
BAF312 (siponimod)
bardoxolone methyl (BARD)
BAX335
BAX-930 (recombinant disintegrin and metalloprotease)
beclomethasone (SGX203)
beclomethasone dispropionate (OrbeShield)
BEL-0218 (ambroxol)
BEL-0222 (firtecan pegol)
benzoate (NaBen)
BG00011 (STX-100)
BGB324
BI-505
bimagrumab (BYM338)
binimetinib/MEK162
BIO 300 (radioprotective)
bis-choline tetrathiomolybdate (WTX-101)
BL-8040
BLU-285
BLU-554
BLZ-100
BMN044
BMN045
BMN053
BMN111
BMN190 (rhTPP1)
BMN701 (reveglucosidase alfa)
BMS-986089 (anti-myostatin adnectin-IgG2)
BMS-986168
Bosulif (bosutinib)
bosutinib (Bosulif)
BP1001 (liposomal Grb-2)
BPX-501
bromelain topical (Debrase)
BT-062 (indatuxuimab ravtansine)
budesonide (SHP621)
bupivacaine transdermal
BYM338 (bimagrumab)
CA4P (fosbretabulin)
cannabidiol
CAP-1002
caplacizumab (ALX-0081)
Carbaglu (carglumic acid)
carbon dioxide intranasal
carglumic acid (Carbaglu)
CAT-1004
Cavatak (coxsackievirus type A21)
CB-5083
CC-90001
CD101 IV
celiprolol (ACER-002)
CEM-102 (sodium fusidate)
CEQ508
chenodeoxycholic acid (Xenbilox)
chenodiol
Cipro-DPI (ciprofloxacin dry powder inhaler)
ciprofloxacin dry powder inhaler (Cipro-DPI)
ciprofloxacin inhalation (ARD-3150)
Cirara (glibenclamide)
Civacir
clofazimine
CLR 131 radiopharmaceutical
CLR-1404I-124
CMB305 (G305/LV305 combination)
CMD-003
CN-105
CNDO-109
COR-003
Cotara
COTI-2
coxsackievirus type A21 (Cavatak)
CPP-115
CPP-1X (eflornithine)
CPP-1X/sul (eflornithine plus sulindac)
CPX-351 (cytarabine:daunorubicin)
crenolanib
cridanimod
CRLX101 (poly-CD-PEG-camptothecin)
cromolyn sodium for inhalation (PA101B)
CTX-4430 (acebilustat)
CUDC-907
Cyramza (ramucirumab)
cysteamine (RP103)
cytarabine:daunorubicin (CPX-351)

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Therapeutic List

Dementia, Frontotemporal
Dengue Fever Treatment
Diarrhoea
Diffuse Systemic Sclerosis
Dilated Cardiomyopathy
Dravet Syndrome
Drug Toxicity
Duchenne Muscular Dystrophy
Dupuytrens Contracture
Dyskinesia Drug Induced
Ebola Haemorrhagic Fever (EHF)
Ebola Virus Disease
Eclampsia
Ehlers Danlos Syndrome
Emesis
Emphysema
Endophthalmitis
Enterovirus Antenatal Infection
Eosinophilic Oesophagitis
Epidermolysis Bullosa (EB)
Epilepsy
Erythromelalgia Primary
Esophageal Ulcer
Essential Thrombocythemia
Ewings Sarcoma
Fabry Disease
Facioscapulohumeral Muscular Dystrophy (FSHD)
Facioscapulohumeral Muscular Dystrophy 1A
Factor X Deficiency
Factor XI Deficiency
Factor XIII Deficiency
Faecal Incontinence
Fallopian Tube Cancer
Familial Adenomatous Polyposis (FAP)
Familial Colorectal Cancer
Familial Lipoprotein Lipase Deficiency
Familial Mediterranean Fever
Familial Pulmonary Arterial Hypertension Leucopenia And Atrial Septal Defect
Familial Transthyretin Amyloidosis
Fibrodysplasia Ossificans Progressiva
Focal Segmental Glomerulosclerosis (FSGS)
Follicular Lymphoma
Fragile X Syndrome
Friedreich Ataxia

dabrafenib and trametinib (Tafinlar + Mekinist)
dacomitinib
DCC-2618
DCVax-L
DE-109 (sirolimus ophthalmic)
Debio 8206 (triptorelin pamoate)
Debrase (bromelain topical)
defactinib (VS-6063)
defactinib (VS-6063)
delamanid (OPC-67683)
demcizumab
dianhdrogalactitol (NSC-132313)
diazepam (intranasal) (NRL-1)
diazepam (intranasal) (Plumiaz)
diazoxide choline controlled release
Dificid (fidaxomicin)
DigiBind (digitoxin immune fab (ovine))
digitoxin immune fab (ovine) (DigiBind)
dipraglurant
DNX-2401 (adenovirus delta 24-RGD)
DOTA-Tyr3 octreotide (Lutathera)
DPX-Survivac
drisapersen
DTX101
duvelisib
E5501 (avatrombopag)
EBV-CTL
ecopipam hydrochloride
EDI200
eflornithine (CPP-1X)
eflornithine plus sulindac (CPP-1X/sul)
EG-1962 (intraventricular nimodipine)
ELAD
eleclazine
eltoprazine
emicizumab (RG6013)
emricasan
encorafenib/LGX818
ENMD-2076
ensitiximab
entolimod
epacadostat
epratuzumab
eprodisate (Kiacta)
EPZ-5676 (DOT1L inhibitor) (pinometostat (DOT1L inhibitor))
EPZ-6438 (tazemetostat)
ERC-1671
EryDex (erythrocyte-encapsulated dexamethasone)
erythrocyte-encapsulated dexamethasone (EryDex)
esuberaprost
eteplirsen/AVI-4658 (exon 51)
ethylenediamine (SQ109)
etirinotecan pegol (NKTR-102)
everolimus (Afinitor)
evinacumab
EX-404
EX-RAD (recilisib sodium)
EZN-2279 (recombinant adenosine deaminase)
farletuzumab (MORAb-003)
FCR001
fenfluramine low-dose (ZX-008)
fenretinide intravenous
FG-3019 (pamrevlumab)
fibroblast growth factor 20 (NGM282)
fidaxomicin (Dificid)
filanesib
fingolimod (Gilenya)
firtecan pegol (BEL-0222)
fitusiran (ALN-AT3SC)
fludarabine phosphate (Gedeptin)
fosbretabulin (CA4P)
fostamatinib disodium
FPA008
FT1050 (Prohema)
funapide (TV-45070)

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Therapeutic List

Gallstones
Gastro Intestinal Adenocarcinoma
Gastro Intestinal Fistula
Gastro Intestinal Stromal Tumours (GIST)
Gaucher Disease
Germ Cell Tumour
Gi Spasm
Giardiasis
Glaucoma
Glioblastoma Multiforme
Glioma
Globoid Cell Leukodystrophy (GLD)
Glomerulonephritis
Glucose Transporter Type 1 Deficiency Syndrome
Glycogen Storage Disease Type 2 (Pompe Disease)
Gout
Graft vs Host Disease (Gvhd)
Gram Negative Infections
Granulomatosis with Polyangiitis
Graves Disease
Growth Hormone Deficiency
Guillain Barre Syndrome
Haemolytic Uremic Syndrome
Haemorrhagic Fever with Renal Syndrome (HFRS) Prophylaxis
Heart Transplantation
Hemophagocytic Lymphohistiocytosis
Hemophilia A Congenital
Hemophilia B
Hepatic Encephalopathy
Hepatitis B Prophylaxis
Hepatitis B Treatment
Hepatitis C Prophylaxis
Hepatitis C Treatment
Hepatitis D Treatment
Hepatoma, Liver Cancer
Hereditary Angioedema
Hereditary Cerebral Hemorrhage with Amyloidosis
Hidradenitis Suppurativa
HIV Associated Nephropathy
Hodgkin Lymphoma
Homocystinuria
Homozygous Familial Hypercholesterolemia
Hunter Syndrome (Mucopolysaccharidosis II Or MPS II)
Huntington Disease
Hurlers Syndrome (Mucopolysaccharidosis I Or MPS I)
Hutchinson Gilford Progeria Syndrome
Hyperammonaemia
Hypercalcaemia
Hyperimmunoglobulinemia D Syndrome (HIDS)
Hyperinsulinemic Hypoglycemia Familial 2
Hyperinsulinemic Hypoglycemia Familial 2 & 3
Hyperlipoproteinemia Type 1
Hyperoxaluria
Hyperparathyroidism
Hyperphosphataemia
Hyperprolactinaemia
Hypersomnia
Hypertrophic Cardiomyopathy
Hyperuricaemia
Hypoglycaemia
Hypogonadism
Hypolipoproteinemia
Hypoparathyroidism
Hypophosphatasia
Hypophosphatemic Rickets
Hypotension
Hypoxia
Idiopathic Pulmonary Fibrosis
Idiopathic Thrombocytopenic Purpura
Iga Nephropathy
Immune Thrombocytopenia
Immunoglobulin Deficiency
Inclusion Body Myopathy 2
Inclusion Body Myositis
Infantile Spasms Broad Thumbs
Iron Poisoning

G17DT immunogen (polyclonal antiboby stimulator)
G305/LV305 combination (CMB305)
galinpepimut-S (WT1)
gallium (68Ga) edotreotide (SomaKit-TOC)
gallium-68 DOTATATE (Somakit-TATE)
galunisertib
ganaxolone
Gazyva (obinutuzumab)
GBT440
GC4419
Gedeptin (fludarabine phosphate)
GEN-1
Gilenya (fingolimod)
GL-0817
glibenclamide (Cirara)
glucagon
glucagon injection (G-Pen)
glufosfomide
GM06
GMI-1070 (pan-selectin antagonist) (rivipansel (pan-selectin antagonist))
GMI-1271
golimumab (Simponi Aria)
golimumab (Simponi)
G-Pen (glucagon injection)
GPX-150
GS010
GS-4997
GS-5734
GS-5745
GSK2398852 + GSK2315698
GSK2696273
GSK2696274
GSK2696275
GSK3377794 (NY-ESO T-lymphocyte cell therapy)
GZ402666 (neoGAA)
GZ402671
H.P. Acthar Gel (repository corticortropin injection)
halofuginone hydrobromide (HT-100)
hepatocyte growth factor-X7 (VM202)
HSP70 heat shock protein (Minnelide 001)
HT-100 (halofuginone hydrobromide)
human plasminogen
human retinal progenitor cells (hRPC)
hydrocortisone modified release capsules
hydroxyprogesterone (LPCN 1107)
Hyqvia (immune globulin infusion 10% (human) with recombinant human hyaluronidase)
ibalizumab (TMB-355)
Iclusig (ponatinib)
ICT-107
idebenone (Raxone)
IMA901
imetelstat
immune globulin infusion 10% (human) with recombinant human hyaluronidase (Hyqvia)
IMO-8400
indatuxuimab ravtansine (BT-062)
inhaled xenon gas
INOpulse (nitric oxide)
inotuzumab ozogamicin
interferon gamma-1b (Actimmune)
intravenous carbamazepine
intraventricular nimodipine (EG-1962)
iobenguane I-131 (Azedra)
IONIS-DMPK-2.5RX
IONIS-HTTRX
IONIS-SMNRX (nusinersen)
IONIS-SOD111RX
IONIS-TTRRX
ipatasertib
IRX-2 immunotherapy
isatuximab
isotretinoin (PAT-001)
ixazomib (MLN9708)
ixmyelocel-T

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Therapeutic List

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Therapeutic List

Malignant Ascites
Malignant Mesothelioma
Mantle Cell Lymphoma
Maple Syrup Urine Disease
Marburg Haemorrhagic Fever
Marfan Syndrome
Maroteaux Lamy Disease (Mucopolysaccharidosis VI Or MPS VI)
Mastocytosis
Melanoma
Meningitis, Cryptococcal AIDS Related
Mesothelioma
Metachromatic Leukodystrophy (MLD)
Methaemoglobinaemia
Methicillin Resistant Staphylococcus Aureus (MRSA) Infections
Methylmalonic Acidaemia (MMA)
Mitochondrial Complex Deficiency I V
Mitochondrial Disease
Mitochondrial Encephalopathy, Lactic Acidosis, And Stroke Like Episodes (MELAS)
Mitochondrial Oxidative Phosphorylation Disorders
Molybdenum Cofactor Deficiency
Morquio Syndrome (Mucopolysaccharidosis IV Or MPS IV)
Mucocutaneous Dryness
Mucopolysaccharidosis Type I
Mucopolysaccharidosis Type II
Mucopolysaccharidosis Type IIIA
Mucopolysaccharidosis Type IIIB
Mucopolysaccharidosis Type VII
Multifocal Motor Neuropathy (MMN)
Multiple Myeloma
Multiple Pterygium Syndrome X-Linked
Muscle Spasticity
Muscular Dystrophy
Musculoskeletal Inflammation
Myasthenia Gravis
Mycobacterium Avium Complex Infections
Mycosis Fungoides
Myelodysplastic Syndromes
Myelofibrosis
Myocardial Infarction, Acute (AMI)
Myotonic Dystrophy
Narcolepsy
Necrotizing Enterocolitis
Necrotizing Fasciitis
Nematode (Roundworm) Infections
Neonatal Ovarian Cyst
Neoplastic Meningitis
Nephropathic Cystinosis
Nephrotic Syndrome
Neuroblastoma
Neuroendocrine Tumour
Neuromyelitis Optica Spectrum Disorder
Neuronal Ceroid Lipofuscinoses
Neutropaenia
Neutropenia And Thrombocytopenia And Anemia
Niemann Pick Disease Type C1
Noma
Non Hodgkin Lymphoma (NHL)
Non Hodgkin Lymphoma Childhood
Non Small Cell Lung Cancer (NSCLC)
Non Tuberculosis Mycobacterial Lung Disease and Cystic Fibrosis
Oesophageal Cancer
Oesophageal Varices
Optic Neuritis
Optic Neuromyelitis
Optic Neuropathy Anterior Ischemic
Organ Rejection
Organic Acidemia
Osteoporosis
Ovarian Cancer

MA09-hRPE cells
macimorelin acetate (Macrilen)
Macrilen (macimorelin acetate)
ManNAc (N-acetyl-D-mannosamine)
maribavir (SHP620)
marizomib IV
MASP-2 (OMS-721)
MBX-8025
mecasermin rinfabate (SHP607)
MEDI-551
melapuldencel-T
melphalan
melphalan flufenamide
MENK (met5-enkephalin)
mepolizumab
mesalamine controlled release (Lialda)
met5-enkephalin (MENK)
metadoxine
metronidazole 10% ointment
mibefradil
midazolam intranasal spray
midostaurin (PKC412)
migalastat (AT1001)
milciclib
Minnelide 001 (HSP70 heat shock protein)
mipsagargin
mirvetuximab soratansine
mitomycin (Mitosol)
Mitosol (mitomycin)
MLN4924 (pevonedistat)
MLN9708 (ixazomib)
MN-001 (tipelukast)
mocetinostat
MOD-5014 (long-acting recombinant factor VIIa-CTP4)
mogamulizumab
momelotinib
MOR208
MORAb-003 (farletuzumab)
MORAb-004 (ontuxizumab)
MORAb-009 (amatuximab)
MORAb-066
motolimid
moxetumomab pasudotox
MP-376 (levofloxacin inhalation)
MST188 (poloxamer 188, purified)
Multikine
Multistem
N91115 (S-nitrosoglutathione)
NaBen (benzoate)
N-acetyl-D-mannosamine (ManNAc)
naloxone topical
necuparanib
NEO100 (perillyl alcohol)
NEOD001
neoGAA (GZ402666)
neuroblastoma bivalent vaccine
NGF-hTNF (Arenegyr)
NGM282 (fibroblast growth factor 20)
NI-0501 (mAb)
nifurtimox
niraparib
nitric oxide (INOpulse)
Nitric oxide (Thiolanox)
NK-8228 (letermovir)
NKTR-102 (etirinotecan pegol)
NKTT120
NLX-101
NNZ-2566 (trafinetide)
nonacog beta pegol (40K PEGylated recombinant factor IX)
NP001 (sodium chlorite)
NRL-1 (diazepam (intranasal))
NSC-132313 (dianhdrogalactitol)
NSI-566
NT-100 (rhGCSF)
NT-501
NurOwn
nusinersen (IONIS-SMNRX)
NVX-108
NY-ESO T-lymphocyte cell therapy (GSK3377794)
obinutuzumab (Gazyva)
OCT-002 (L-ornithine phenylacetate)
octreotide (oral)
olaratumab
olipudase alfa (recombinant human acid sphingomyelinase (rhASM))
ombitasvir/paritaprevir/ritonavir (ViekiraPak)
omeprazole-lansoprazole with buffer
omigapil
OMS-721 (MASP-2)
OMS824
ONO-01210 (recilisib sodium)
ontuxizumab (MORAb-004)
OPC-67683 (delamanid)
Opdivo nivolumab phalan
oprozomib
OrbeShield (beclomethasone dispropionate)
oregovomab
OS-320 (amantadine)
OSE2101
osilodrostat (LCI699)
Otezla (apremilast)
OTL38
otlertuzumab
Oxabact (oxalobacter formigenes)
oxalobacter formigenes (Oxabact)
OXi4503

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Pachyonychia Congenita
Pagets Disease
Pancreatic Cancer
Pancreatic Islet Transplantation
Pancreatitis
Pantothenate Kinase Associated Neurodegeneration
Panuveitis
Parkinson Disease
Paroxysmal Nocturnal Haemoglobinuria (PNH)
Patent Ductus Arteriosus
Pcdh19 Epilepsy
Pediatric Crohns Disease
Pediatric Ulcerative Colitis
Pemphigus Vulgaris
Peripheral Neuropathy
Peripheral T Cell Lymphoma
Peripheral Vascular Disease (PVD)
Pertussis
Peyronies Disease
Phenylketonuria (PKU)
Plague Treatment
Pleural Effusion
Pneumocystis Jiroveci (Pneumocystis Carinii) Infections
Polio Prophylaxis
Polyarthritis Systemic
Polycystic Kidney Disease (PKD)
Polycystic Liver Disease
Polycystic Ovarian Syndrome
Polycythaemia Vera
Polymyositis
Polymyositis and Dermatomyositis
Porphyria
Post Transplant Lymphoproliferative Disease
Postherpetic Neuralgia
Pouchitis
Prader Willi Syndrome
Pre Eclampsia
Precocious Puberty
Primary Biliary Cirrhosis (PBC)
Primary Sclerosing Cholangitis
Progressive Multifocal Leukoencephalopathy (PML)
Progressive Supranuclear Palsy
Prostate Cancer
Proteus Syndrome
Pruritus
Pseudomonas Infections
Pulmonary Alveolar Proteinosis
Pulmonary Fibrosis, Idiopathic
Pulmonary Hypertension
Pyruvate Kinase Deficiency
Rabies
Renal Cell Carcinoma (RCC)
Renal Osteodystrophy
Respiratory Distress Syndrome (RDS)
Retinal Detachment
Retinal Telangiectasia Hypogammaglobulinemia
Retinal Vein Occlusion
Retinitis Pigmentosa
Retinoblastoma
Retinopathy of Prematurity
Rett Syndrome
Rosacea
RSV Infections

P1101 (pegylated proline interferon alpha-2b)
PA101B (cromolyn sodium for inhalation)
paclitaxel trevatide (ANG1005)
palovarotene
pamrevlumab (FG-3019)
pancreatic cancer cell therapy
pantothenate phosphate (RE-024)
PAT-001 (isotretinoin)
patisiran (ALN-TTR02 (siRNA inhibitor targeting TTR))
PB1046
PBT2
PCO371
PEG-PAL (pegvaliase)
PEG-PH20 (PEGylated recombinant human hyaluronidase PH20)
pegvaliase (PEG-PAL)
pegylated carboxyhemoglobin bovine (Sanguinate)
pegylated proline interferon alpha-2b (P1101)
PEGylated recombinant human hyaluronidase PH20 (PEG-PH20)
pembrolizumab (Keytruda)
perillyl alcohol (NEO100)
Perjeta (pertuzumab)
pertuzumab (Perjeta)
pevonedistat (MLN4924)
pexastimogene devacirepvec
pexidartinib (PLX3397)
PF-02545920 (PDE10 inihibitor)
PF-04447943
PF-05230907
PF-06252616
PF-06463922
PF-06834635 (avelumab)
PF-06836922 (MOD-4023) (HGH agonist)
pinometostat (DOT1L inhibitor) (EPZ-5676 (DOT1L inhibitor))
PKC412 (midostaurin)
plasmin (human)
plasminogen intravenous
pleconaril
plitidepsin
Plumiaz (diazepam (intranasal))
PLX3397 (pexidartinib)
PM1183 (lurbinectedin)
Pneumostem
PNT2258 (rosomidnar)
poloxamer 188, purified (MST188)
poly-CD-PEG-camptothecin (CRLX101)
polyclonal antiboby stimulator (G17DT immunogen)
ponatinib (Iclusig)
pracinostat
pradigastat (LCQ908)
pridopidine (TV-7820)
PRM-151 (rhPentraxin-2/rhSerum Amyloid P)
PRM-151 (rhPTX-2 for injection)
Prohema (FT1050)
prolactin receptor antagonist
prophage cancer vaccine
propranolol/etodolac (VT-122)
PRT-201 (vonapanitase)
PRTX-100 (staphylococcal aureus protein A)
PTI-111 (Systebryl)
PV-10 (rose bengal disodium)
PVX-410
Q203
QLT091001
QPI-1002
QPI-1007
QR-010
quizartinib
rAAV1-CB-hAAT (gene therapy)
rAAV2-CB-hRPE65 (gene therapy)
ralinepag
ramucirumab (Cyramza)
Raxone (idebenone)
RBX2660
RE-024 (pantothenate phosphate)
recilisib sodium (EX-RAD)
recilisib sodium (ONO-01210)
recombinant adenosine deaminase (EZN-2279)
recombinant disintegrin and metalloprotease (BAX-930)
recombinant human acid sphingomyelinase (rhASM) (olipudase alfa)
recombinant human beta-glucuronidase (UX003)
recombinant human histidyl tRNA synthetase (ATYR 1940)
recombinant human nerve growth factor (rhNGF)
refanalin (BB3) (HGF mimetic)
reformulated NaPBA (ACER-001)
regorafenib (Stivarga)
REMUNE
reparixin
repository corticortropin injection (H.P. Acthar Gel)
Resimmune (A-dmDT390-bisFv (UCHT1))
retinol deuterated (ALK-001)
reveglucosidase alfa (BMN701)
revusiran
RG6013 (emicizumab)
RG7800
RGI-2001 (liposomal galactosylceramide)
RGN-259 (thymosin beta 4 (TB4))
Rhenium 188 P2045 (somatostatin analog) (Tozaride)
rhGCSF (NT-100)
rhNAGLU (SBC-103)
rhPentraxin-2/rhSerum Amyloid P (PRM-151)
rhPTX-2 for injection (PRM-151)
rhTPP1 (BMN190)
riboflavin ophthalmic solution ultraviolet-A (UVA) irradiation
rigosertib
rintatolimid (Ampligen)
riociguat (Adempas)
Rituxan (rituximab)
rituximab (Rituxan)
RiVax
rivipansel (pan-selectin antagonist) (GMI-1070 (pan-selectin antagonist))
rose bengal disodium (PV-10)
rosomidnar (PNT2258)
rovalpituzumab tesirine
RP103 (cysteamine)
RST100
rucaparib
ruxolitinib (Jakafi)
RX-0201

A B C D E F G H I J K L M N O P Q R S T U V W X Y Z

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Indication List

Therapeutic List

Sanfilippo Syndrome (Mucopolysaccharidosis III or MPS III)
Sarcoidosis
Schizophrenia
Scleroderma
Sclerosing Cholangitis
Severe Combined Immunodeficiency Disease (SCID)
Short Bowel Syndrome
Sickle Cell Anemia
Sjogrens Syndrome
Skin Cancer, Non Melanoma
Slys Syndrome (Mucopolysaccharidosis VII Or MPS VII)
Small Cell Lung Cancer (SCLC)
Smallpox
Smallpox Prophylaxis
Soft Tissue Sarcoma
Solid Tumour Indications
Sphingolipidosis
Spinal Cord Injury
Spinal Muscular Atrophy
Spinocerebellar Ataxia
Squamous Cell Carcinoma of The Head And Neck
Staphylococcal Infections
Stargardt Disease
Status Epilepticus
Stiff Person Syndrome
Stomach Carcinoma
Stomatitis (Mouth Ulcers)
Strabismus (Crossed Eyes)
Stroke, Acute
Subarachnoid Haemorrhage
Surgical Adhesions
Systemic Lupus Erythematosus (SLE)
T Cell Lymphoma 1A
Tay Sachs and Sandhoffs Disease
Testicular Cancer
Testosterone Deficiency
Thalassaemia
Thromboangiitis Obliterans
Thrombocythaemia
Thrombocytopaenia
Thrombosis
Thrombosis, Deep Vein (DVT)
Thrombotic Thrombocytopenic Purpura Congenital
Thyroid Cancer
Tourette Syndrome (Pediatric)
Tourettes Syndrome
Toxoplasmosis
Toxoplasmosis, AIDS Related
Traumatic Brain Injury
Trematode (Fluke) Infections
Trypanosomiasis
Tuberous Sclerosis
Tumor Necrosis Factor Receptor Associated Periodic Syndrome (TRAPS)
Turners Syndrome
Type 1 Plasminogen Deficiency
Tyrosinaemia
Ulcerative Colitis
Ulcers, Chronic Skin
Ulcers, Corneal
Upper Respiratory Tract Infections
Urea Cycle Disorders
Urinary Calculi
Urticaria
Usher Syndrome
Uterine Cancer
Uveitis

SA237
SAGE-547 (allopregnanolone)
Samsca (tolvaptan)
Sanguinate (pegylated carboxyhemoglobin bovine)
sapacitabine
SAR156597
SAR339375
SAR422459
Sarasar (lonafarnib)
SBC-103 (rhNAGLU)
SBP-101
Scenesse (afamelanotide)
SD-101 (allantonin)
SelG1
SER-109
setmelanotide
seviprotimut-L
SGI-110
SGN-CD33A (vadastuximab talirine)
SGX203 (beclomethasone)
SGX301
SHAPE
SHP607 (mecasermin rinfabate)
SHP610 (sulfamidase)
SHP611 (arylsulfatase A)
SHP620 (maribavir)
SHP621 (budesonide)
SHP625
silibinin
siltuximab (Sylvant )
Simponi (golimumab)
Simponi Aria (golimumab)
simtuzumab
siponimod (BAF312)
sirolimus ophthalmic (DE-109)
skin tissue replacement therapy
SL-401
S-nitrosoglutathione (N91115)
SNS-595 (vosaroxin)
sodium chlorite (NP001)
sodium fusidate (CEM-102)
sodium thiosulfate (STS)
Somakit-TATE (gallium-68 DOTATATE)
SomaKit-TOC (gallium (68Ga) edotreotide)
Somatuline (lanreotide acetate)
somavaratan
sonidegib (LDE225)
SOR-C13
sotatercept
sparsentan
SPI-026 (tacrolimus)
Spiriva Handihaler (tiotropium bromide)
SPK-CHM
SPK-FIX
SPK-RPE65
SQ109 (ethylenediamine)
SRP-4053 (exon 53)
staphylococcal aureus protein A (PRTX-100)
Stivarga (regorafenib)
STM 434
STP-206
STX-100 (BG00011)
sulfamidase (SHP610)
Sylvant (siltuximab)
Systebryl (PTI-111)
tacrolimus (SPI-026)
tafamidis meglumine (Vyndaqel)
tafenoquine
Tafinlar + Mekinist (dabrafenib and trametinib)
tarextumab
tazemetostat (EPZ-6438)
TD-101
tecovirimat
telatinib
telotristat etiprate
TEN-010
teprotumumab
ThermoDox (lyso-thermosensitive lipsomal doxorubicin)
Thiolanox (Nitric oxide)
thymosin beta 4 (TB4) (RGN-259)
tiotropium bromide (Spiriva Handihaler)
tipelukast (MN-001)
tirasemtiv
tisagenlecleucel-T
tivantinib
TMB-355 (ibalizumab)
TNT009
Toca 511 + Toca FC
tocilizumab (Actemra)
tolvaptan (Samsca)
topiramate injection
tosedostat
Tozaride (Rhenium 188 P2045 (somatostatin analog))
TPI 287
TPIV200
trafinetide (NNZ-2566)
trans sodium crocetinate (TSC)
TRC105
trehalose
tremelimumab
Trevyent
triheptanoin (UX007)
triptorelin pamoate (Debio 8206)
TV-1106 (albutropin)
TV-45070 (funapide)
TV-7820 (pridopidine)
TXA127
type 1 native bovine skin collagen (ARG201)
TZ101
ulocuplumab
UshStat
UX001 (aceneuramic acid)
UX003 (recombinant human beta-glucuronidase)
UX007 (triheptanoin)

A B C D E F G H I J K L M N O P Q R S T U V W X Y Z

Request Added Indication or Therapeutic: One Day Turnaround

Indication List

Therapeutic List

Varicella Infections
Vasculitis
Veno Occlusive Disease (VOD)
Von Hippel Lindau Disease
Von Willebrand Disease (VWD)
Vulvar Dysplasia
Waldenstrom Macroglobulinemia
Wegeners Granulomatosis
West Nile Virus (WNV) Disease
Whooping Cough
Wilson Disease
Wiskott Aldrich Syndrome
Wolmans Disease
X-Linked Hypohidrotic Ectodermal Dysplasia