Orphan Drug Intelligence

Somewhere, something incredible in Orphan Drug is waiting to be known. We offer market research capabilities to help you see what everybody else has seen, and to think what nobody else has thought.

Rare diseases, also known as orphan diseases, are difficult to be cared. As per the global estimates, over 80 million people suffer from unexplored medical conditions. However, given the marginal number of patients per disease, not many mainstream pharmaceutical companies picked it as their R&D focus area until recent years when the orphan drug market emerged as an unexplored billion dollar opportunity. These companies, now realizing the revenue potential of unexplored drugs, are souring their resources to develop and market medicinal products to treat them.

KuicK Research provides high-end intelligence, research and consulting services to companies and individuals. We help you gain reliable insight into the current orphan drug market with the information on latest drug development, clinical trials and trends in the international and regional markets. We look at these markets from every angle, use cutting-edge analytics and advance technology methods to collect complex data from suppliers, medical transactions, medical journals, pharmaceutical companies, CRO, global health reports and expert resources on orphan diseases and drugs to be truly of good use to you.

The research outcome of our collaboration lets you harness the power of accurate information and gain strategic knowledge on business continuity. Clear comparison, contrast and analyzation of the current market scenario and ongoing clinical researches of the leading business groups in the segment facilitate critical decisions, such as opening new clinical research fronts, launching fresh drugs or engaging in orphan drug development.
To Know More About How We Can Help You To Influence The Future Of Orphan Drugs, Please Contact Us Today.
Title Date
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

Need custom market research solution? We can help you with that too.