Emergence of Orphan Drugs in Cancer Therapy |
Aug-2022 |
Global Orphan Drug Rare Disease Clinical Trials Market USD 350 Billion Opportunity |
Nov-2021 |
HyBryte Receives Orphan Drug Designation From FDA For Treatment of T Cell Lymphoma |
Oct-2021 |
AOC 1001 Receives Orphan Drug Designation in Europe |
Oct-2021 |
ZYNLONTA Granted Orphan Designation In Europe |
Oct-2021 |
Nadunolimab Drug Granted Orphan Drug Designation |
Oct-2021 |
FDA Granted Orphan Designation For Gavo-Cel To Treat Cholangiocarcinoma |
Oct-2021 |
CAM2029 Drug Granted Orphan Drug Designation To Treat Polycystic Liver Disease |
Oct-2021 |
PLX-200 Drug Granted Orphan Drug Designation To Treat Krabbe Disease Therapy |
Oct-2021 |
LP-184 DRUG For Glioblastoma Multiforme Gets Orphan Drug Designation |
Oct-2021 |
Opelconazole (PC945) Drug Granted Orphan Designation By U.S FDA |
Oct-2021 |
VS01 Earns Orphan Drug Designation To Treat Hyperammonemia |
Oct-2021 |
US FDA Granted Orphan Designation To DB-OTO |
Oct-2021 |
FLT201 Drug Wins Orphan Drug Designation From FDA |
Oct-2021 |
Octagam Drug Granted Orphan Designation From U.S FDA |
Oct-2021 |
FDA Grants Orphan Drug Designation For PLX-200 Drug To Treat GM2 Gangliosidoses |
Oct-2021 |
DAY101 Wins Orphan Drug Designation By European Commission |
Sep-2021 |
U.S. FDA Grants Orphan Drug Designation for Tesomet |
Sep-2021 |
Multiple Myeloma Drug ALLO-715 Granted Orphan Designation |
Sep-2021 |
European Medicines Agency (EMA) Grants Orphan Drug Designation For Bomedemstat |
Sep-2021 |
FDA Grants Orphan Designation To CycloSam For Treatment of Osteosarcoma |
Sep-2021 |
FDA Grants Orphan Drug Designation To CFT7455 For Treatment of Multiple Myeloma |
Sep-2021 |
NNZ-2591 Receives Orphan Drug Designation For Treatment Of Prader-Willi |
Sep-2021 |
European Commission Grants TASHA-102 Orphan Designation For Treatment Of Rett Syndrome |
Sep-2021 |
Orphan Drug Designates EPX-200 For Treatment Of Lennox-Gastaut Syndrome |
Sep-2021 |
VS-01 Got Orphan Drug Designation To Treat Hyperammonemia |
Sep-2021 |
Lantern Pharma Grants Orphan Drug Designation For LP-184 |
Sep-2021 |
RLF-100 (aviptadil) Receives U.S. FDA Orphan Drug Designation |
Sep-2021 |
FDA Grants Orphan Drug Designation for NUZYRA (omadacycline) |
Sep-2021 |
Imatinib Received US FDA Orphan Drug Designation |
Sep-2021 |
Ketamine Earns FDA Orphan Drug Designation To Treat Amyotrophic Lateral Sclerosis |
Sep-2021 |
European Commission Grants Orphan Drug Designatipon For AK-OTOF |
Sep-2021 |
Orphan Drug Designation Grants Ketamine To Treat ALS |
Sep-2021 |
Marinus Pharmaceuticals Earns Orphan Drug Designation For Ganaxolon |
Sep-2021 |
Sotigalimab (APX005M) Receives Orphan Drug Designation From US FDA |
Sep-2021 |
NFX-179 Receives Orphan Drug Designation For Treatment Of Cutaneous Neurofibromatosis Type 1 |
Sep-2021 |
Aldeyra Therapeuticss Receives Orphan Drug Designation Granted For ADX-2191 |
Sep-2021 |
ABX-002 Earns Orphan Drug Designation |
Sep-2021 |
U.S FDA Grants Orphan Drug Designation for AER-901 (Inhaled Imatinib) |
Sep-2021 |
VBX-100 Prodrug Earns Orphan Drug Designation |
Sep-2021 |
Relief Announces Orphan Drug Designation To Use Rlf-100 (Aviptadil) |
Sep-2021 |
FDA Grants Orphan Drug Designation For VO659 |
Sep-2021 |
FDA Grants Orphan Drug Designation To ADX-2191 (Intravitreal Methotrexate) |
Sep-2021 |
Vactosertib Receives FDA Orphan Drug Designation For Osteosarcoma |
Sep-2021 |
Alrizomadlin (APG-115) Wins FDAs Orphan Drug Designation |
Sep-2021 |
LX1004 Earns FDAs Orphan Drug Designation |
Sep-2021 |
ADX-2191 Wins FDAs Orphan Drug Designation |
Sep-2021 |
INZ-701 Gets Orphan Drug Designation From European Medicines Agency |
Sep-2021 |
US FDA Grants Orphan Drug Designation For ONCOFID-P |
Sep-2021 |
FDA & European Commission Grants Orphan Drug Designation to UX053 |
Sep-2021 |
Orphan Drug Designation Grants European Medicines Agency (EMA) For Saroglitazar Mg |
Sep-2021 |
Pridopidine Wins Orphan Drug Designation To Treat ALS |
Sep-2021 |
INTEPLA (Fenfluramine) Receives Orphan Drug Designation in Japan |
Sep-2021 |
ALS001 Earns Orphan Drug Designation from the U.S FDA |
Sep-2021 |
VICO Therapeutics Receives Orphan Drug Designation For VO659 |
Sep-2021 |
CTD401 Earns Orphan Drug Designation To Treat T-ALL |
Sep-2021 |
FDA Grants Orphan Drug Designation For TP-317 To Treat Pancreatic Cancer |
Sep-2021 |
US FDA Grants Orphan Drug Designation For Etrasimod To Treat Eosinophilic Esophagitis Eoe |
Sep-2021 |
To Treat Stem Cell Transplant FDA Grants Orphan Drug Designation For JSP191 |
Sep-2021 |
Diazoxide Choline Receives Orphan Drug Designation To Treat Glycogen Storage Disease Type 1a |
Sep-2021 |
SPG302 Wins Orphan Drug Designation From U.S FDA |
Sep-2021 |
European Medicines Agency Grants Orphan Drug Designation For Elamipretide |
Sep-2021 |
VectivBio Earns FDA Orphan Drug Designation for Apraglutide |
Sep-2021 |
CPI-613® (Devimistat) Receives Orphan Drug Designation From U.S FDA |
Sep-2021 |
Atsena Therapeutics Earns Orphan Drug Designation from US FDA for Novel Gene Therapy |
Sep-2021 |
US FDA Grants Orphan Drug Designation For Xywav |
Sep-2021 |
BNL-101 Wins Orphan Drug Designation To Treat Malignant Gliomas |
Sep-2021 |
US FDA Grants Orphan Drug Designation for GS-100 Drug |
Sep-2021 |
EMA Grants Orphan Drug Designation to CLN5 Batten Disease Gene Therapy |
Sep-2021 |
EMA Grants Orphan Drug Designation To SLS-005 |
Sep-2021 |
PT001 Receives Orphan Drug Designation To Treat Pulmonary Arterial Hypertension |
Sep-2021 |
EU grants orphan drug status to Strekin's STR001 for Sudden Sensorineural Hearing Loss |
Apr-2017 |
NantCell receives FDA orphan drug status for Ganitumab Monoclonal Antibody Therapy in Ewing Sarcoma |
Apr-2017 |
Tasquinimod from Active Biotech receives orphan drug status for multiple myeloma |
Apr-2017 |
FDA orphan drug status given to Exelixis, Inc. (EXEL) for the Treatment of Hepatocellular Carcinoma |
Apr-2017 |
Annamycin from Moleculin receives orphan drug status for the Treatment of Acute Myeloid Leukemia |
Apr-2017 |
FDA grants orphan drug designation to SB-913 Genome Editing Treatment for MPS II |
Apr-2017 |
DLBCL receives FDA orphan drug designation for eFT508 |
Apr-2017 |
AstraZeneca NMOSD Drug receives orphan drug designation |
Apr-2017 |
FDA grants orphan drug status to Sapience Therapeutics (ST-36) |
Apr-2017 |
FDA orphan status for VAL001 |
Apr-2017 |
Opko receives orphan drug designation for CUR-1916 in Dravet Syndrome |
Apr-2017 |
European Union grants orphan drug designation to LSK BioPharma Apatinib |
Apr-2017 |
EMA grants orphan drug designation to aTyr Pharma for the Treatment of Limb Girdle Muscular Dystrophy with Resolaris |
Apr-2017 |
FDA grants orphan drug designation to Miragen Therapeutics |
Apr-2017 |
Prometic PBI-4050 receives FDA orphan drug status for Alström Syndrome |
Apr-2017 |
FDA announces orphan drug status for aNK Natural Killer Cell Therapy in Merkel Cell Carcinoma |
Apr-2017 |
FDA grants orphan drug designation to EnGeneIC for Targeted EDV™ Nanocells to Treat Glioblastoma Multiforme |
Apr-2017 |
Gamida Cell Announces orphan drug designation for NiCord® |
Apr-2017 |
EU Commission grants orphan drug status to ZX008 in Lennox Gastaut Syndrome |
Apr-2017 |
EMA grants orphan drug designation to Epidiolex® from GW Pharmaceuticals |
Apr-2017 |
CAEL-101 from Fortress Biotech (FBIO) Receives Orphan Drug Designations for Amyloidosis |
Apr-2017 |
Dynport’s Plague vaccine receives orphan drug status from FDA |
Apr-2017 |
Boehringer Ingelheim's investigational anti-CD33 monoclonal antibody BI 836858 receives FDA orphan drug status |
Apr-2017 |
ChemoCentryx announces FDA orphan drug designation for Avacopan in the Treatment of Debilitating Kidney Disease C3 Glomerulopathy (C3G) |
Apr-2017 |
EU Commission grants orphan drug designation to Abeona’s EB101 Gene Therapy |
Apr-2017 |
Yeliva receives FDA orphan drug status for cholangiocarcinoma |
Apr-2017 |
Minoryx therapeutics announces orphan drug designation for MIN-102 |
Mar-2017 |
Protalex receives orphan drug designation for ITP |
Mar-2017 |
FDA grants orphan drug status to ZW25 for Gastric Cancer Treatment |
Mar-2017 |
Immune design receives orphan drug status for G100 Intratumoral therapy C&idate |
Mar-2017 |
FDA grants orphan drug designation to Sangamo’s SB-318 |
Feb-2017 |
FDA grants orphan drug status to Vicore |
Feb-2017 |
ONCURIOUS NV receives orphan drug status for TB-403 for Medulloblastoma |
Feb-2017 |
EC grants orphan drug designation to Therabron Therapeutic’s CG367 |
Feb-2017 |
Arthritis in Children gets orphan drug by FDA |
Feb-2017 |
Abeona receives orphan drug designation for ABO-101 to treat Sanfilippo Syndrome Type- B |
Feb-2017 |
Idogen receives orphan drug status for Hemophilia A |
Feb-2017 |
FDA grants orphan drug designation to sickle cell treatment |
Feb-2017 |
FDA grants orphan drug designation to lysogene’s LYSGM101 for GM1 Gangliosidosis |
Feb-2017 |
Angiosarcoma gets orphan drug as Propranolol by EC |
Feb-2017 |
EMA grants orphans drug status to Therabron Therapeutics’ CG367 |
Feb-2017 |
Viralym-C receives orphan status to treat CMV |
Feb-2017 |
ProMetic's PBI-4050 recieves orphan drug status for Alström Syndrome: EC |
Feb-2017 |
GenSight Biologics announces orphan drug status for GS030 for Retinitis Pigmentosa |
Feb-2017 |
EC Orphan Drug Designation for Cabiralizumab (FPA008) for PVNS |
Feb-2017 |
Concert’s CTP-656 receives orphan drug status for Cystic Fibrosis |
Feb-2017 |
FDA grants orphan drug designation to TG Therapeutics to treat Diffuse Large B-cell Lymphoma |
Feb-2017 |
Benitec’s BB-301 receives Orphan drug status to treat OMPD |
Feb-2017 |
Nintedanib receives orphan drug award for mesothelioma |
Jan-2017 |
Orphan status for Manchester Biotech’s antifungal in EU |
Jan-2017 |
FDA grants orphan status to Marinus Pharma |
Jan-2017 |
Hansa Medical receives orphan drug status for Ides in Europe |
Jan-2017 |
European orphan drug award for Agilis Bio therapeutics for AADC Deficiency |
Jan-2017 |
MeiraGTx receives Orphan Drug Designation for A004 Gene Therapy for Retinitis Pigmentosa |
Jan-2017 |
EU Commission grants orphan drug designation to Nefecon® For Treatment Of Iga Nephropathy |
Jan-2017 |
FDA grants orphan designation for ABTL0812 in Pancreatic Cancer |
Jan-2017 |
Escend’s ES3000 receives FDA orphan Drug status for AML |
Jan-2017 |
FDA orphan drug status for Cellspan Esophageal Implant by Biostage |
Jan-2017 |
Eiger Bio's exendin 9-39 receives orphan drug status for hyperinsulinemic hypoglycemia |
Jan-2017 |
FDA grants orphan drug status to Profounda Inc. to treat PAM |
Jan-2017 |
EMA grants orphan drug status to F2G’s antifungal drug |
Jan-2017 |
US FDA grants orphan drug status to MacroGenics’ MGD006 |
Jan-2017 |
Yisheng Biopharma's PIKA Rabies Vaccine receives orphan drug status from FDA |
Jan-2017 |
FDA orphan drug status to Acucela for Stargardt Disease |
Jan-2017 |
Senhwa Biosciences CX-4945 receives orphan drug status in Cholangiocarcinoma |
Jan-2017 |
Alxion Pharma receives FDA orphan drug designation for PNH |
Jan-2017 |
FDA grants orphan drug status to Sangamo’s SB-318 Genome editing treatment for MPS-I |
Jan-2017 |
FDA grants orphan drug designation to PTCT’s RG-7916 |
Jan-2017 |
EMA grants orphan drug designation to AB-201 for juvenile batten disease |
Jan-2017 |
TG-1101 & TG-1202 Combo receives orphan drug designation: FDA |
Jan-2017 |
FDA grants orphan drug status to Marinus’ Ganaxolone |
Jan-2017 |
Cellceutix announces orphan drug status for Kevetrin |
Jan-2017 |
Neurotrope receives orphan drug designation for bryostatin-1 by FDA |
Jan-2017 |
FDA grants orphan drug designation for Biogen’s Spinraza Spinal Muscular Atrophy |
Jan-2017 |
FDA grants orphan drug status to CymaBay’s MBX-8025 |
Jan-2017 |
Sickle Cell Disease gets its orphan drug by Global Blood in Europe |
Jan-2017 |
FDA grants orphan drug designation to Viking Therapeutics’ VK0214 to treat X-ALD |
Jan-2017 |
FDA declares BHV-0223 as orphan drug to treat ALS |
Jan-2017 |
Amryt receives orphan drug status for Episalvan |
Jan-2017 |
EU Union grants orphan drug designation to ARA 290 |
Dec-2016 |
FDA grants orphan drug status to Keystone’s ceramides |
Dec-2016 |
Novel PAH treatment receives FDA orphan drug designation |
Dec-2016 |
Omeros Corp. files for orphan drug status for OMS721 in Immunoglobin A Nephropathy |
Dec-2016 |
Novel Hepatocellular Carcinoma Tx receives orphan drug status by FDA |
Dec-2016 |
EU Commission granted orphan drug status for SENS-401 |
Dec-2016 |
FDA grants orphan drug status to Diazepam Buccal soluble film |
Dec-2016 |
FDA grants orphan drug status to ARMO’s AM0010 |
Dec-2016 |
AbbVie receives orphan status for ABBV-006 in U.S |
Dec-2016 |
FDA grants orphan drug status to PRX-OTC by PhaseRx |
Dec-2016 |
Ofev receives orphan drug status for Potential Scleroderma |
Dec-2016 |
EMA grants BioMarin’s Brinuera orphan drug status for CLN2 disease |
Dec-2016 |
FDA grants orphan drug status to Singh Biotechnology |
Dec-2016 |
Ra’s RX101494 receives orphan drug designation for PNH by EU |
Dec-2016 |
GX-H9 receives orphan designation for growth hormone deficiency |
Dec-2016 |
European Union grants orphan drug status to Crenolanib |
Dec-2016 |
ELX-02 receives orphan drug status for MPS I |
Dec-2016 |
EU grants orphan drug status to Respiratorius’s VAL001 |
Dec-2016 |
Cytori’s ECCS- 50 granted as orphan drug status for scleroderma |
Dec-2016 |
Resunab by Corbus Pharma receives Orphan drug status by EC |
Dec-2016 |
SGT-001 declared orphan drug for DMD |
Nov-2016 |
Iomab-B receives EMA orphan drug status for AML |
Nov-2016 |
DPX-Survivac receives orphan drug status from EMA |
Nov-2016 |
Eloxx pharmaceuticals receives orphan drug designation for ELX-02 in Mucopolysacchardisis Type 1 (MPS 1) in US |
Nov-2016 |
Lundbeck’s carnexiv receives FDA orphan drug status |
Nov-2016 |
FDA grants orphan drug status to SER100 in PAH |
Nov-2016 |
EC grants orphan drug designation to Philogen SpA for the treatment of soft tissue sarcoma. |
Nov-2016 |
Invasive Aspergillus infections recieves orphan drug as SCY-078 by SCYNEXIS |
Nov-2016 |
Solid Biosciences receives orphan drug designation from FDA & EMA for Duchenne Muscular Dystrophy |
Nov-2016 |
Theranexus’s THN102 receives orphan drug designation from FDA for treatment of Narcolepsy |
Nov-2016 |
FDA grants orphan drug designation to Synlogic Proprietary Synthetic Biotic |
Nov-2016 |
Yisheng Biopharma's Biological Product receives orphan drug status for Hepatocellular Carcinoma |
Nov-2016 |
True North’s TNT009 receives orphan drug status for Autoimmune Hemolytic Anemia, including Cold Agglutinin Disease (CAD) |
Nov-2016 |
FDA grants orphan drug designation to AAV1-FS344 for Inclusion Body Myositis |
Nov-2016 |
CD4CAR therapy receives FDA approval for orphan drug designation for peripheral T-cell Lymphoma |
Nov-2016 |
EMA announces orphan drug designation for BP1001 for AML treatment |
Nov-2016 |
FDA announces orphan drug designation for Veliparib as NSCLC treatment |
Nov-2016 |
FDA approves orphan drug status for Dusquetide for Macrophage Activation Syndrome |
Nov-2016 |
FDA announces orphan drug designation for DTX401 for the treatment of Glycogen Storage Disease Type Ia (GSDIa) |
Nov-2016 |
MediciNova declares FDA approval for MN-166 as orphan drug |
Nov-2016 |
EU Union grants orphan drug status to GLPG1690 for idiopathic pulmonary fibrosis |
Nov-2016 |
Firdapse gets orphan drug status for treatment of Myasthenia Gravis |
Nov-2016 |
FDA announces orphan drug designation for TG-1101 for NMO & NMOSD |
Nov-2016 |
TiGenix’s Cx601 granted as orphan drug designation in Switzerl& |
Nov-2016 |
EMA announces orphan drug designation for ABO-102 gene therapy |
Nov-2016 |
EMA declares orphan drug designation to Masitinib for Lou Gehrig's disease |
Oct-2016 |
Regenicin's NovaDerm designated as Orphan drug for skin grafting: FDA |
Oct-2016 |
FDA grants VX15 from Vaccinex Inc. Orphan Drug designation for Huntington’s disease |
Oct-2016 |
Tivorsan receives orphan drug designation for Human Recombinant Biglycan for DMD |
Oct-2016 |
FDA grants Biotie’s BTT1023 orphan drug designation |
Oct-2016 |
First orphan drug introduced for Friedreich’s Ataxia: FDA |
Oct-2016 |
European commission declares orphan drug status for Alexion’s ALXN1007 |
Oct-2016 |
FDA grants TGR1202 orphan drug designation for Chronic Lymphocytic Leukemia |
Oct-2016 |
Ovarian Cancer gets Orphan Drugs as ZW25 & ZW33; approves FDA |
Oct-2016 |
TGA grants orphan drug status to Raxone for DMD |
Oct-2016 |
First orphan drug approved for Duchenne muscular dystrophy |
Oct-2016 |
Singh Biotechnology receives orphan drug designation for STAT3 |
Oct-2016 |
FDA declares Firdapse as orphan drug for treatment of Myasthenia Gravis |
Oct-2016 |
Singh Biotechnology receives orphan drug designation for STAT3 |
Oct-2016 |
GLPG1690 from Galapogas, receives orphan drug designation for idiopathic pulmonary fibrosis |
Oct-2016 |
FDA grants orphan drug designation to OV101 for Angelman Syndrome |
Oct-2016 |
FDA grants orphan drug designation to BIV201 for treatment of Acsites |
Oct-2016 |
Orphan drug designation plea for Delta-9-Tetrahydrocannibinol & Cannabidiol |
Oct-2016 |
SB- Fix receives orphan drug designation for Hemophilia B |
Oct-2016 |
FDA grants BioXcel’s BXCL101 orphan drug designation for Neurofibromatosis Type 2 (NF2) |
Oct-2016 |
FDA approves Soricimed's SOR-C13 as orphan drug for pancreatic cancer |
Oct-2016 |
Coversin by Akari therapeutics receives Orphan drug Designation from FDA |
Oct-2016 |
FDA approves N-Methanocarbathymidine as orphan drug in U.S |
Oct-2016 |
FDA orphan drug designation for ProTmune given to Fate Therapeutics |
Oct-2016 |