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Title Author Type Date Words
US FDA grants Orphan Drug Designation to OBI Pharma's OBI-999. Dec 27, 2019 206
US FDA grants Orphan Drug Designation to OBI Pharma's OBI-999. Dec 27, 2019 210
Amryt Pharma: US deal set to transform the orphan drug company. Dec 20, 2019 715
European Commission grants Dicerna Pharmaceuticals' DCR-A1AT orphan drug designation. Dec 19, 2019 158
European Commission grants Dicerna Pharmaceuticals' DCR-A1AT orphan drug designation. Dec 19, 2019 154
Eagle Pharmaceuticals Granted Orphan Drug Designation for Ryanodex for Treatment of Organophosphate Exposure. Dec 18, 2019 323
The Daily Biotech Pulse: Roche Gets US Antitrust Clearance For Spark Purchase, An Orphan Drug Designation For Prevail, Dynavax Names CEO. Shanthi Rexaline Dec 17, 2019 1468
Savara Names van Es-Johansson to Board of Directors. Dec 6, 2019 326
APR Applied Pharma Research's APR-TD011 granted US FDA Orphan Drug Designation. Dec 5, 2019 232
APR Applied Pharma Research's APR-TD011 granted US FDA Orphan Drug Designation. Dec 5, 2019 228
CNS Pharmaceuticals signs license agreement with Houston Pharmaceuticals. Nov 21, 2019 243
CNS Pharmaceuticals signs license agreement with Houston Pharmaceuticals. Nov 21, 2019 239
Global Orphan Drugs Market Analysis and Forecast 2016-2026: ~$400 Billion Market Breakdown by Drug Type, Therapy Class, Drug, Disease Type, Indication, Phase, Distribution Channel & Geography. Nov 20, 2019 764
United States : Drug Manufacturer Prevented from Abusing System After Shaheen Calls on FDA to Act. Nov 15, 2019 686
Autolus Therapeutics receives FDA orphan drug designation for AUTO1. Nov 5, 2019 147
Deal-Making Trends in Pharma: 2019 Thematic Research Report. Report Oct 29, 2019 817
Crispr Therapeutics expects data from Phase 1/2 study of CTX001 in late 2019. Oct 28, 2019 118
Verastem Shares Rally After Getting Orphan Drug Designation For COPIKTRA; Compliments Fast Track Designation To Treat Peripheral T-cell Lymphoma (Nasdaq:VSTM). Oct 7, 2019 696
Matinas BioPharma receives orphan drug designation from FDA for MAT2203. Oct 7, 2019 162
Flavocure Biotech Announces Orphan Designation Granted by FDA for Caflanone (FBL-03G) in Pancreatic Cancer. Oct 3, 2019 916
Exclusive Bionical EMAS Speaker Interview Released for the Orphan Drugs & Rare Diseases Conference. Conference news Sep 29, 2019 600
United States : Cumberland Pharmaceuticals Announces FDA Orphan Drug Grant Award For New Phase II Clinical Program. Clinical report Sep 28, 2019 364
Amryt Pharma: US deal set to transform the orphan drug company. Sep 26, 2019 540
Reneo Pharmaceuticals's REN001 granted Orphan Drug Designation by US FDA. Sep 24, 2019 214
Reneo Pharmaceuticals's REN001 granted Orphan Drug Designation by US FDA. Sep 24, 2019 210
SpringsWorks Therapeutics: EC grants Orphan Drug Designation for nirogacestat. Sep 24, 2019 138
Reneo Pharmaceuticals receives FDA orphan drug designation for REN001 to treat fatty acid oxidation disorders. Sep 23, 2019 228
Reneo Pharmaceuticals receives FDA orphan drug designation for REN001 to treat fatty acid oxidation disorders. Sep 23, 2019 224
Chugai Pharmaceutical Co., Ltd. - Chugai Receives Orphan Drug Designation for Satralizumab in Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorder from the Ministry of Health, Labour and Welfare -- 13/9/2019. Sep 19, 2019 686
India,United States : Glenmark Receives Orphan Drug Designation for GBR 1342, a Bispecific Antibody Candidate Under Evaluation for the Treatment of Multiple Myeloma. Sep 19, 2019 438
Glenmark Pharmaceuticals wins the FDA orphan drug designation for GBR 1342 to treat multiple myeloma. Sep 17, 2019 258
Glenmark Pharmaceuticals wins the FDA orphan drug designation for GBR 1342 to treat multiple myeloma. Sep 17, 2019 254
Japan : Chugai Receives Orphan Drug Designation for Satralizumab in Neuromyelitis Optica and Neuromyelitis Optica Spectrum Disorder from the Ministry of Health, Labour and Welfare. Sep 17, 2019 579
Akari Therapeutics' nomacopan granted U.S. orphan drug designation for BP. Sep 13, 2019 208
United States : Shaheen Calls on FDA to Stop Drug Manufacturer from Abusing System Amid Opioid Epidemic & Allow More Affordable Options for Medication-Assisted Treatment. Sep 12, 2019 591
US FDA grants Orphan Drug designation to Acceleron Pharma's Sotatercept. Sep 11, 2019 220
US FDA grants Orphan Drug designation to Acceleron Pharma's Sotatercept. Sep 11, 2019 216
Acceleron's Pulmonary Arterial Hypertension Asset Receives Orphan Drug Designation. Sep 9, 2019 283
Acceleron Receives FDA Orphan Drug Designation for Sotatercept in Pulmonary Arterial Hypertension. Sep 9, 2019 529
Puma's Breast Cancer Drug Accorded Orphan Drug Designation For Brain Metastases. Sep 4, 2019 347
BioXcel Therapeutics receives Orphan Drug Designation for BXCL701. Sep 4, 2019 187
FDA grants orphan status to Puma's Nerlynx for brain metastases. Sep 3, 2019 193
CARsgen Therapeutics Inc wins US FDA orphan drug designation for investigational ct053 for multiple myeloma treatment. Sep 2, 2019 195
CARsgen Therapeutics Inc wins US FDA orphan drug designation for investigational ct053 for multiple myeloma treatment. Sep 2, 2019 191
US FDA grants orphan drug designation to CARsgen Therapeutics' multiple myeloma therapy. Sep 2, 2019 218
US FDA grants orphan drug designation to CARsgen Therapeutics' multiple myeloma therapy. Sep 2, 2019 214
Mike Page, Alexion Pharmaceuticals Invites You to SMi's Orphan Drugs & Rare Diseases Conference. Conference news Sep 1, 2019 444
La Jolla Pharmaceutical Company was granted an Orphan Drug Designation from the FDA for the investigational drug, LJPC-0118 (artesunate), for the treatment of malaria. Clinical report Sep 1, 2019 157
US FDA grants orphan drug designation to AstraZeneca's Fasenra. Aug 30, 2019 152
US FDA grants orphan drug designation to AstraZeneca's Fasenra. Aug 30, 2019 148
CrystalGenomics Receives Orphan Drug Designation from the US FDA for CG-745 in Pancreatic Cancer. Aug 30, 2019 483
The Daily Biotech Pulse: Genmab-Novartis Multiple Sclerosis Drug Aces Late-Stage Studies, Akari Nets Orphan Drug Designation. Aug 30, 2019 587
AstraZeneca's Fasenra granted Orphan Drug Designation by US FDA for eosinophilic oesophagitis. Aug 29, 2019 236
AstraZeneca's Fasenra granted Orphan Drug Designation by US FDA for eosinophilic oesophagitis. Aug 29, 2019 232
Orphan Drugs Market Is Booming With Top Key Players: Bayer, Bristol-Myers Squibb, Novartis, Pfizer, Roche / Radiant Insights, Inc. Aug 27, 2019 818
Chrysalis BioTherapeutics wins FDA and EMA orphan drug status for TP508 for ARS. Aug 22, 2019 157
Chrysalis BioTherapeutics wins FDA and EMA orphan drug status for TP508 for ARS. Aug 22, 2019 153
Orphan Drugs: Global Market Outlook Through 2019-2023 with Bristol-Myers Squibb, Roche, Johnson & Johnson Services, Novartis, and Takeda Pharmaceutical Leading. Aug 19, 2019 719
Neuropore Receives Orphan Drug Designation for NPT520-34 for the Treatment of Amyotrophic Lateral Sclerosis. Aug 16, 2019 315
US FDA grants orphan drug designation to Stoke Therapeutics' STK-001. Aug 8, 2019 275
Stoke Therapeutics Granted FDA Orphan Drug Designation for STK-001, an Investigational New Treatment for Dravet Syndrome. Aug 8, 2019 558
US FDA grants orphan drug designation to Stoke Therapeutics' STK-001. Aug 8, 2019 279
Global Orphan Drugs Market Growth, Trends and Forecasts 2019-2024. Aug 2, 2019 623
APB-102 RECEIVES ORPHAN DRUG DESIGNATION FROM FDA. Aug 1, 2019 504
European Commission grants orphan drug designation to SpringWorks Therapeutics' mirdametinib. Aug 1, 2019 150
European Commission grants orphan drug designation to SpringWorks Therapeutics' mirdametinib. Aug 1, 2019 146
Global Pulmonary Arterial Hypertension (PAH) Market Forecast to 2025: Focus on Prostacyclin & Prostacyclin Analogs, SGC Stimulators, ERA, and PDE-5 Drug Classes. Aug 1, 2019 889
AstraZeneca- Imfinzi granted US Orphan Drug Designation for small cell lung cancer -- 12/7/2019. Aug 1, 2019 285
European Commission Grants Orphan Drug Designation for SpringWorks Therapeutics' MEK Inhibitor, Mirdametinib, for the Treatment of Neurofibromatosis Type 1. Jul 31, 2019 851
Biohaven enrolls first patient in Phase 3 trial for Multiple System Atrophy. Jul 31, 2019 166
Cervical Dystonia Treatment Market Size, Share and Consumption Analysis Report 2028. Jul 31, 2019 874
Cervical Dystonia Treatment Market Size, Share and Consumption Analysis Report 2028. Jul 31, 2019 859
X4 Pharmaceuticals Receives Orphan Drug Designation from European Commission for Mavorixafor for Treatment of WHIM Syndrome. Jul 31, 2019 756
SpringWorks awarded EC orphan drug designation for Mirdametinib for the treatment of Neurofibromatosis Type 1. Jul 30, 2019 227
SpringWorks awarded EC orphan drug designation for Mirdametinib for the treatment of Neurofibromatosis Type 1. Jul 30, 2019 223
Oncotelic to provide clinical development updates for OT101, OXi4503. Jul 30, 2019 444
Orphan Drugs Market is expected to reach $169 billion from $106 billion and register a CAGR of 6.8% by 2022. Jul 29, 2019 758
Mustang Bio receives orphan drug designation for MB-102. Jul 24, 2019 130
Aridis Pharmaceuticals' AR-501 receives Orphan Drug Designation. Jul 22, 2019 252
Aridis Pharmaceuticals' AR-501 receives Orphan Drug Designation. Jul 22, 2019 248
AstraZeneca's Imfinzi Granted orphan drug designation. Jul 17, 2019 143
AstraZeneca's Imfinzi Granted orphan drug designation. Jul 17, 2019 139
Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-ANG3. Jul 17, 2019 387
Arrowhead receives orphan drug designation for ARO-ANG3. Jul 16, 2019 149
AstraZeneca - Imfinzi granted US Orphan Drug Designation for small cell lung cancer -- 12/7/2019. Jul 16, 2019 285
AstraZeneca announces grant by US FDA of Orphan Drug Designation to Imfinzi. Jul 15, 2019 286
AstraZeneca announces grant by US FDA of Orphan Drug Designation to Imfinzi. Jul 15, 2019 282
TOP NEWS: AstraZeneca Gets US FDA Orphan Drug Designation For Imfinzi. Jul 12, 2019 183
Pfizer and Sangamo reveal details of phase 1/2 Alta study of SB-525 haemophilia A gene therapy. Jul 9, 2019 203
Pfizer and Sangamo reveal details of phase 1/2 Alta study of SB-525 haemophilia A gene therapy. Jul 9, 2019 199
FDA approves Xpovio and dexamethasone for refractory multiple myeloma. Jul 4, 2019 262
FDA approves Xpovio and dexamethasone for refractory multiple myeloma. Jul 4, 2019 258
Alexion Pharmaceuticals and Minoryx Therapeutics to Present at SMi's Orphan Drugs & Rare Diseases Event. Jun 29, 2019 676
LifeMax Receives Orphan Drug Designation from the United States Food and Drug Administration For LM-030 For The Treatment of Netherton Syndrome. Jun 28, 2019 303
Aridis Pharmaceuticals awarded FDA's ODD for AR-501 for cystic fibrosis. Jun 27, 2019 192
Aridis Pharmaceuticals awarded FDA's ODD for AR-501 for cystic fibrosis. Jun 27, 2019 188
Rick Thompson, CEO of Findacure Invites Delegates to SMi's Orphan Drugs & Rare Diseases Conference 2019. Conference news Jun 27, 2019 760
Arrowhead Pharmaceuticals Receives Orphan Drug Designation for ARO-APOC3. Jun 27, 2019 348
Orphan Drugs Market Size, Share, Trends and Future Growth Predictions and Forecast 2017 -- 2025. Jun 27, 2019 901
Orphan Drugs Market Size, Share, Trends and Future Growth Predictions and Forecast 2017 -- 2025. Jun 27, 2019 888
XW Laboratories Inc passes US FDA orphan drug designation for XWL-008 to treat narcolepsy patients. Jun 24, 2019 234
XW Laboratories Inc passes US FDA orphan drug designation for XWL-008 to treat narcolepsy patients. Jun 24, 2019 230
NuCana says FDA grants orphan drug designation for Acelarin. Jun 12, 2019 166
US FDA grants orphan drug designation to Reata Pharmaceuticals' bardoxolone methyl. Jun 7, 2019 209
US FDA grants orphan drug designation to Reata Pharmaceuticals' bardoxolone methyl. Jun 7, 2019 205
Palatin Technologies awarded FDA's orphan drug designation for PL-8177 for treatment of non-infectious uveitis. Jun 7, 2019 243
Palatin Technologies awarded FDA's orphan drug designation for PL-8177 for treatment of non-infectious uveitis. Jun 7, 2019 239
Palatin granted FDA orphan drug designation for PL-8177. Jun 6, 2019 122
Biosight awarded US FDA orphan drug designation for BST-236 to treat AML. Jun 5, 2019 206
Biosight awarded US FDA orphan drug designation for BST-236 to treat AML. Jun 5, 2019 202
Proteostasis receives orphan drug designation for PTI-428 in Europe. Jun 4, 2019 111
The Orphan Promise: New policy on rare disease treatments likely soon. Harachand, S. Jun 1, 2019 834
Ridgeback Biotherapeutics wins FDA orphan drug designation for mAb114 for for Ebola. May 31, 2019 248
Ridgeback Biotherapeutics wins FDA orphan drug designation for mAb114 for for Ebola. May 31, 2019 244
FDA Grants Orphan Drug Designation to Ayala's AL101 for Potential Treatment of Adenoid Cystic Carcinoma. May 17, 2019 370
Registration Opens for SMi's 9th Annual Orphan Drugs & Rare Diseases Conference. Conference news May 10, 2019 671
Corcept says EMA recommends orphan drug designation for relacorilant. May 2, 2019 210
MY ROAD TO THE RARE DISEASE WORLD. Meyers, Abbey S. May 1, 2019 1869
TG Therapeutics, Inc. was granted. May 1, 2019 358
Knopp Biosciences Receives FDA Orphan Drug Designation for Dexpramipexole for Treatment of Hypereosinophilic Syndrome. Apr 25, 2019 287
US FDA grants Orphan Drug Designation to Viracta Therapeutics' cancer drug nanatinostat. Apr 17, 2019 189
US FDA grants Orphan Drug Designation to Viracta Therapeutics' cancer drug nanatinostat. Apr 17, 2019 185
FibroGen receives orphan drug designation from FDA for pamrevlumab. Apr 15, 2019 133
Synspira Therapeutics wins EMA's orphan designation for SNSP113 for treating cystic fibrosis in the EU. Apr 11, 2019 253
Synspira Therapeutics wins EMA's orphan designation for SNSP113 for treating cystic fibrosis in the EU. Apr 11, 2019 249
Imbrium Therapeutics Gains US FDA Orphan Drug Designation for Tinostamustine for the Treatment of T-cell Prolymphocytic Leukemia. Apr 2, 2019 592
Catalyst Biosciences receives orphan drug desgination for MarzAA in EU. Apr 1, 2019 202
United Kingdom,United States : US FDA grants saracatinib Orphan Drug Designation for idiopathic pulmonary fibrosis. Mar 25, 2019 263
US FDA grants Orphan Drug Designation to AstraZeneca's saracatinib for idiopathic pulmonary fibrosis. Mar 19, 2019 241
US FDA grants Orphan Drug Designation to AstraZeneca's saracatinib for idiopathic pulmonary fibrosis. Mar 19, 2019 237
United Kingdom : US FDA grants saracatinib Orphan Drug Designation for idiopathic pulmonary fibrosis. Mar 18, 2019 267
Orphan Drugs Market Projections & Future Opportunities Recorded for the Period until 2025. Mar 12, 2019 696
Orphan Drugs Market Projections & Future Opportunities Recorded for the Period until 2025. Mar 12, 2019 972
Orphan Drugs Market Projections & Future Opportunities Recorded for the Period until 2025. Mar 12, 2019 959
Acceleron Receives FDA Orphan Drug Designation for ACE-083 in Charcot-Marie-Tooth Disease. Mar 6, 2019 524
Acceleron receives FDA Orphan Drug Designation for ACE-083. Mar 5, 2019 103
United States : Provectus Announces Grant of Orphan Drug Designation in U.S. to PV-10 for Treatment of Ocular Melanoma. Feb 23, 2019 365
Omeros Streamlines Path to Biologics License Application for OMS721 in HSCT-TMA Following Meeting with FDA. Feb 19, 2019 987
Calliditas Therapeutics wins US FDA orphan drug designation for primary biliary cholangitis. Feb 13, 2019 215
Calliditas Therapeutics wins US FDA orphan drug designation for primary biliary cholangitis. Feb 13, 2019 211
Amplyx Pharmaceuticals passes US FDA orphan drug designation for APX001 to treat cryptococcosis. Feb 12, 2019 292
Amplyx Pharmaceuticals passes US FDA orphan drug designation for APX001 to treat cryptococcosis. Feb 12, 2019 288
AstraZeneca announces grant of Orphan Drug Designation to Fasenra for hypereosinophilic syndrome. Feb 7, 2019 304
Equillium granted FDA orphan drug designation for EQ001 for aGVHD. Feb 7, 2019 107
Calliditas Therapeutics announces grant by US FDA of orphan drug designation for Autoimmune hepatitis. Feb 6, 2019 309
Calliditas Therapeutics announces grant by US FDA of orphan drug designation for Autoimmune hepatitis. Feb 6, 2019 305
Pharm-Olam Enrolls Patients in Zealand Pharma Orphan Drug Study. Clinical report Feb 6, 2019 241
BioLineRx wins US FDA orphan drug designation for BL-8040 to treat pancreatic cancer. Feb 5, 2019 239
Pharm-Olam enrolls patients in Zealand Pharma orphan drug study. Clinical report Feb 5, 2019 119
Apellis receives orphan drug designation for C3 complement inhibitor APL-2. Feb 5, 2019 226
Japan : A Nucleic Acid Drug, TRK-250, Receives Orphan Drug Designation from the FDA. Feb 4, 2019 299
Merck KGaA passes US FDA orphan drug designation for M7824 to treat biliary tract cancer. Dec 11, 2018 240
Merck KGaA passes US FDA orphan drug designation for M7824 to treat biliary tract cancer. Dec 11, 2018 236
Merck KGaA announces FDA orphan drug designation for M7824. Dec 10, 2018 290
FDA GRANTS ORPHAN DRUG DESIGNATION TO NPC DRUG. Dec 1, 2018 496
AstraZeneca announces grant of Orphan Drug Designation by US FDA for Fasenra. Nov 27, 2018 207
AstraZeneca announces grant of Orphan Drug Designation by US FDA for Fasenra. Nov 27, 2018 203
United Kingdom : US FDA grants Fasenra Orphan Drug Designation for Eosinophilic Granulomatosis with Polyangiitis. Nov 26, 2018 226
Pliant Therapeutics wins US FDA orphan drug designation for PLN-74809 for primary sclerosing cholangitis. Nov 21, 2018 246
OBI Pharma passes US FDA orphan drug designation for OBI-888 to treat pancreatic cancer. Nov 21, 2018 241
Pliant Therapeutics wins US FDA orphan drug designation for PLN-74809 for primary sclerosing cholangitis. Nov 21, 2018 242
OBI Pharma passes US FDA orphan drug designation for OBI-888 to treat pancreatic cancer. Nov 21, 2018 237
FDA Grants Orphan Drug Designation Granted to 4D-110 for the Treatment of Choroideremia. Nov 20, 2018 239
REGENXBIO Inc awarded US FDA's orphan drug designation for RGX-181 for treating CLN2 disease. Nov 15, 2018 242
REGENXBIO Inc awarded US FDA's orphan drug designation for RGX-181 for treating CLN2 disease. Nov 15, 2018 238
Gilead announces GS-9674 Phase 2 trial resutls, confirms Orphan Drug Designation. Nov 9, 2018 213
Provectus says FDA grants orphan drug designation to PV-10 for neuroblastoma. Nov 5, 2018 131
SpringWorks Therapeutics Receives FDA Orphan Drug Designation for MEK Inhibitor (PD-0325901) for the Treatment of Neurofibromatosis Type 1. Nov 5, 2018 629
Vertex receives EU authorization for CF medicine Symkevi. Nov 1, 2018 102
Orphan Drug Designation For Treatment Of Stem Cell Transplant-Related Disorder. Oct 29, 2018 365
Orphan Drug Designation For Treatment Of Stem Cell Transplant-Related Disorder. Oct 29, 2018 365
Eidos Therapeutics receives orphan drug designation for AG10 from EMA. Oct 26, 2018 108
Hansa Medical announces receipt of positive opinion for Orphan Drug Designation in the EU for Imlifidase for anti-GBM disease. Oct 25, 2018 361
Hansa Medical announces receipt of positive opinion for Orphan Drug Designation in the EU for Imlifidase for anti-GBM disease. Oct 25, 2018 362
Hansa Medical announces receipt of positive opinion for Orphan Drug Designation in the EU for Imlifidase for anti-GBM disease. Oct 25, 2018 357
Omeros granted FDA Orphan Drug Designation for OMS721. Oct 23, 2018 164
Y-mAbs Therapeutics receives recommendation from COMP for OMPD for naxitamab. Oct 23, 2018 187
Khon2btreat: Late clinical development of kh176: An innovative orphan drug to reach mitochondrial disease patients & Market. Oct 18, 2018 380
AstraZeneca and Merck announce grant of Orphan Drug Designation by US FDA for pancreatic cancer medicine Lynparza. Oct 17, 2018 342
AstraZeneca and Merck announce grant of Orphan Drug Designation by US FDA for pancreatic cancer medicine Lynparza. Oct 17, 2018 338
US FDA Grants AstraZeneca and Merck's Lynparza Orphan Drug Designation for Pancreatic Cancer. Oct 17, 2018 331
AstraZeneca, Merck: FDA grants LYNPARZA Orphan Drug Designation. Oct 16, 2018 103
X4 Pharmaceuticals Receives Orphan Drug Designation from the US FDA for X4P-001-RD for the Treatment of WHIM Syndrome. Oct 15, 2018 478
Adult Malignant Glioma Therapeutics Market to Expand at a Significant CAGR of 9.2% from 2018 to 2026. Oct 11, 2018 122
Preliminary Attendee List Released for Orphan Drugs & Rare Diseases Conference in London. Conference notes Oct 10, 2018 484
Access to Orphan Drugs is a Challenge for Sustainable Management of Cystinosis in China. Li, Xiao-Qiao; Peng, Xiao-Xia; Gong, Chun-Xiu Report Oct 5, 2018 1155
Esanex Concludes End-of-Phase 2 Meeting for SNX-5422 in Neuroendocrine Tumors. Oct 5, 2018 258
Daiichi Sankyo's axicabtagene ciloleucel granted Orphan Drug designation by Japanese Ministry. Oct 4, 2018 205
Daiichi Sankyo Company wins Japanese orphan drug designation for Axicabtagene Ciloleucel for treating B-Cell Lymphoma. Oct 4, 2018 227
Daiichi Sankyo's axicabtagene ciloleucel granted Orphan Drug designation by Japanese Ministry. Oct 4, 2018 201
Daiichi Sankyo Company wins Japanese orphan drug designation for Axicabtagene Ciloleucel for treating B-Cell Lymphoma. Oct 4, 2018 223
Bertilimumab granted orphan drug status, studies underway. Mechcatie, Elizabeth Oct 1, 2018 204
The US FDA grants orphan drug designation to Myelo Therapeutics' Myelo001. Sep 27, 2018 192
The US FDA grants orphan drug designation to Myelo Therapeutics' Myelo001. Sep 27, 2018 188
Amag Pharmaceuticals acquires orphan drug candidate for preeclampsia. Sep 26, 2018 322
Pluristem PLX cell therapy granted FDA Orphan Drug Designation. Sep 25, 2018 136
Fibrocell Science receives $1.4M grant for FCX-007 from FDA's OOPD. Sep 25, 2018 127
FDA Grants Myelo Therapeutics Orphan Drug Designation for Myelo001 as Treatment for Acute Radiation Syndrome. Sep 25, 2018 577
Pharma Giants Collaboration Accelerates Access to Rare Disease Treatment - Hear More at Orphan Drugs & Rare Diseases 2018. Sep 21, 2018 547
OBI Pharma Inc wins US FDA's orphan drug designation for OBI-3424 for Acute Lymphoblastic Leukemia. Sep 20, 2018 239
OBI Pharma Inc wins US FDA's orphan drug designation for OBI-3424 for Acute Lymphoblastic Leukemia. Sep 20, 2018 235
Corbus Pharmaceuticals receives orphan drug designation in EU for Lenabasum. Sep 18, 2018 166
Belgium : Committee for Orphan Medicinal Products (COMP) elects new chair. Sep 18, 2018 302
Cytori Therapeutics receives FDA orphan drug designation for ATI-1123. Sep 17, 2018 346
Syntimmune Granted FDA Orphan Drug Designation for SYNT001 in Treatment of Pemphigus. Sep 13, 2018 495
XORTX seeks orphan designation for potential new polycystic kidney disease treatment. Sep 10, 2018 222
XORTX seeks orphan designation for potential new polycystic kidney disease treatment. Sep 10, 2018 218
Invitation from Carina Schey, Researcher at University of Groningen and Chair of This Year's Orphan Drugs & Rare Diseases Conference. Conference news Sep 8, 2018 555
ARMGO Pharma wins US FDA's orphan drug designation for ARM210/S48168 for Ryanodine Receptor Type 1 Related Myopathies. Sep 6, 2018 249
ARMGO Pharma wins US FDA's orphan drug designation for ARM210/S48168 for Ryanodine Receptor Type 1 Related Myopathies. Sep 6, 2018 253
ARMGO Pharma Receives FDA Orphan Drug Designation for ARM210/S48168 for the Treatment of Ryanodine Receptor Type 1 Related Myopathies. Sep 6, 2018 404
Maryland biopharm gets orphan drug designation. Sep 5, 2018 115
Gene Therapy Specialist ReflectionBio Receives Orphan Drug Designation from US FDA for the Treatment of Bietti's Crystalline Dystrophy. Aug 31, 2018 354
Trovagene awarded European Commission's Orphan Drug Designation for Onvansertib for treating Acute Myeloid Leukemia in Europe. Aug 30, 2018 311
Trovagene awarded European Commission's Orphan Drug Designation for Onvansertib for treating Acute Myeloid Leukemia in Europe. Aug 30, 2018 307
European Commission Grants Orphan Drug Designation to Onvansertib for Treatment of Acute Myeloid Leukemia in Europe. Aug 30, 2018 755
European Commission Designates OMS721 as an Orphan Medicinal Product for Treatment in Hematopoietic Stem Cell Transplantation. Aug 29, 2018 730
Omeros: OMS721 designated Orphan Medicinal Product in EU for HSCT. Aug 28, 2018 104
United States : Bertilimumab Granted Orphan Drug Designation for the Treatment of Bullous Pemphigoid. Aug 27, 2018 205
HealthTech Healx Raise $10m to Accelerate Drug Discovery, Prior to Speaking at Orphan Drugs and Rare Diseases Conference. Conference news Aug 16, 2018 585
USFDA grants orphan drug designation to Onspira Therapeutics' OSP-101. Aug 16, 2018 174
USFDA grants orphan drug designation to Onspira Therapeutics' OSP-101. Aug 16, 2018 170
Alteogen Gets FDA Approval for Orphan Drug Designation with an Antibody-Drug Conjugate for Gastric Cancer. Aug 9, 2018 368
Neuralstem announces NSI-189 granted orphan drug designation from FDA. Aug 9, 2018 106
PharmaMar wins US FDA orphan drug designation for lurbinectedin for small cell lung cancer. Aug 6, 2018 196
PharmaMar wins US FDA orphan drug designation for lurbinectedin for small cell lung cancer. Aug 6, 2018 192
How Challenges Including Brexit, Inadequate Funding & Insufficient Patient Sample Sizes Hinder Orphan Drugs and Rare Diseases Research. Aug 4, 2018 715
Trovagene's AML candidate recommended for Orphan Drug designation in EU. Aug 1, 2018 122
The European Medicines Agency's committee adopts positive opinion on Omeros' OMS721 orphan drug designation. Jul 27, 2018 230
The European Medicines Agency's committee adopts positive opinion on Omeros' OMS721 orphan drug designation. Jul 27, 2018 226
Oasmia Pharmaceutical gives update on Apealea's EU orphan designation status. Jul 27, 2018 254
Oasmia Pharmaceutical withdraws status of Orphan designation of Apealea in the EU. Jul 27, 2018 306
Oasmia Pharmaceutical withdraws status of Orphan designation of Apealea in the EU. Jul 27, 2018 307
Oasmia Pharmaceutical gives update on Apealea's EU orphan designation status. Jul 27, 2018 250
Oasmia Pharmaceutical withdraws status of Orphan designation of Apealea in the EU. Jul 27, 2018 302
Omeros says OMS721 receives European Orphan Drug designation positive opinion. Jul 25, 2018 254
FDA, EMA Grant Inozyme Pharma Orphan Drug Designation for INZ-701. Jul 23, 2018 236
US FDA Awards Orphan Drug Status to OBI Pharma's OBI-3424 for HCC. Jul 12, 2018 209
Acceleron announces orphan drug designation for ACE-083 by FDA. Jul 12, 2018 148
Dicerna receives orphan designation in U.S., recommended orphan by EMA. Jul 11, 2018 148
OBI Pharma awarded US FDA orphan drug designation for Hepatocellular Carcinoma (HCC) treatment OBI-3424. Jul 10, 2018 248
Hansa Medical announces grant of orphan drug designation by the FDA to its lead candidate imlifidase (IdeS). Jul 10, 2018 365
Hansa Medical announces grant of orphan drug designation by the FDA to its lead candidate imlifidase (IdeS). Jul 10, 2018 366
OBI Pharma awarded US FDA orphan drug designation for Hepatocellular Carcinoma (HCC) treatment OBI-3424. Jul 10, 2018 244
Cellectar Biosciences receives FDA ODD for CLR 131. Jul 9, 2018 119
Patient Organisations Role in Orphan Drugs Clinical Development. Jul 6, 2018 543
EMA Orphan Drug Status Granted to Oral Candidate for Treatment of Stargardt Disease. Jul 2, 2018 223
Wave Life Sciences receives positive opinion for ODD for WVE-210201. Jul 2, 2018 284
Cemiplimab impresses in advanced cutaneous SCC. Jancin, Bruce Jul 1, 2018 1137
Lin BioScience wins EMA's orphan drug status for LBS-008 for Stargardt Disease. Jun 28, 2018 257
Lin BioScience wins EMA's orphan drug status for LBS-008 for Stargardt Disease. Jun 28, 2018 253
MeiraGTx receives positive opinion in for orphan drug designation for AAV-CNGA3. Jun 26, 2018 102
FDA Orphan Drug Designation For Treatment of Limbal Stem Cell Deficiency. Jun 25, 2018 444
FDA Orphan Drug Designation For Treatment Or Limbal Stem Cell Deficiency. Jun 25, 2018 444
FDA Grants Orphan Drug Designation for AMO Pharma's Rett Syndrome Treatment. Jun 18, 2018 195
Samus Therapeutics passes US FDA's orphan drug designation for PU-H71 for myelofibrosis, with first patient dosed under Phase 1b study. Jun 14, 2018 252
Samus Therapeutics passes US FDA's orphan drug designation for PU-H71 for myelofibrosis, with first patient dosed under Phase 1b study. Jun 14, 2018 248
FDA Grants Orphan Drug Designation to Ironwoods's Olinciguat for the Treatment of Sickle Cell Disease. Jun 12, 2018 197
United States : Horizon Pharma plc and the National Organization for Rare Disorders (NORD) Illustrate 35 years of Progress in Rare Disease at the Biotechnology Innovation Organization (BIO) International Convention. Jun 11, 2018 470
Official Programme Released for SMi's 8th Annual Orphan Drugs and Rare Diseases Conference. Conference news Jun 11, 2018 347
FDA Grants Orphan Drug Designation to Alnylam's Investigational RNAi Therapeutic for the Treatment of Transthyretin-Mediated Amyloidosis. Jun 8, 2018 257
Ironwood says FDA grants orphan drug designation to olinciguat. Jun 7, 2018 151
Ascendis Pharma announces orphan drug designation from FDA for TransCon PTH. Jun 6, 2018 131
BioCryst's BCX7353 receives European regulatory designations. May 24, 2018 193
Cellectar granted Orphan Drug Designation for CLR 131. May 9, 2018 111
Bavarian Nordic announces receipt FDA orphan drug designation for BN-Brachyury for the treatment of Chordoma. May 3, 2018 348
Bavarian Nordic announces receipt FDA orphan drug designation for BN-Brachyury for the treatment of Chordoma. May 3, 2018 349
FDA Grants Stealth BioTherapeutics Orphan Drug Designation for Elamipretide for Treatment of Leber's Hereditary Optic Neuropathy. Clinical report May 2, 2018 235
Stealth BioTherapeutics passes US FDA orphan drug designation for elamipretide to treat Leber's hereditary optic neuropathy. May 1, 2018 232
Stealth BioTherapeutics passes US FDA orphan drug designation for elamipretide to treat Leber's hereditary optic neuropathy. May 1, 2018 228
Lin BioScience passes US FDA orphan drug designation for LBS-007 to treat acute lymphoblastic leukaemia. Apr 25, 2018 227
Lin BioScience passes US FDA orphan drug designation for LBS-007 to treat acute lymphoblastic leukaemia. Apr 25, 2018 223
Abeona Therapeutics granted Orphan Drug Designation for ABO-202 in Europe. Apr 20, 2018 250
NeuroVive secures US FDA's orphan drug designation for KL1333 for treating mitochondrial diseases. Apr 19, 2018 311
NeuroVive secures US FDA's orphan drug designation for KL1333 for treating mitochondrial diseases. Apr 19, 2018 315
US FDA Grants Stealth BioTherapeutics Orphan Drug Designation for Elamipretide for Treatment of Barth Syndrome. Apr 16, 2018 232
US FDA Grants Stealth BioTherapeutics Orphan Drug Designation for Elamipretide for Treatment of Barth Syndrome. Apr 12, 2018 232
Stealth BioTherapeutics passes US FDA orphan drug designation for elamipretide to treat Barth Syndrome. Apr 10, 2018 207
Stealth BioTherapeutics passes US FDA orphan drug designation for elamipretide to treat Barth Syndrome. Apr 10, 2018 203
FDA Grants Orphan Drug Designation To MSC Product. Apr 9, 2018 323
Tetra Discovery Partners Receives FDA Orphan Drug Designation for BPN14770 for Treatment of Fragile X Syndrome. Apr 9, 2018 238
EMA Grants Orphan Drug Designation to Sancilio's Altemia for Treatment of Pediatric Patients with Sickle Cell Disease. Apr 9, 2018 229
Tetra Discovery Partners Receives FDA Orphan Drug Designation for BPN14770 for Treatment of Fragile X Syndrome. Apr 6, 2018 242
EMA Grants Orphan Drug Designation to Sancilio's Altemia for Treatment of Pediatric Patients with Sickle Cell Disease. Apr 6, 2018 233
Tetra Discovery Partners Receives FDA Orphan Drug Designation for BPN14770 for Treatment of Fragile X Syndrome. Apr 6, 2018 207
EMA Grants Orphan Drug Designation to Sancilio's Altemia for Treatment of Pediatric Patients with Sickle Cell Disease. Apr 6, 2018 197
US FDA Grants MaxiNovel Pharmaceuticals Orphan Drug Designation for MAX-40279 for Treatment of AML. Apr 4, 2018 185
FDA Grants Orphan Drug Designation to Scholar Rock for SRK-015 for Treatment of SMA. Apr 4, 2018 245
FDA Grants Martin Pharmaceuticals Orphan Drug Designation for Livantra for Treatment of Liver Failure. Apr 4, 2018 141
FDA Grants Orphan Drug Designation to PharmAbcine's TTAC-0001 for Glioblastoma Multiforme. Apr 4, 2018 137
FDA Grants Elysium Health Orphan Drug Designation for EH301 for Treatment of ALS. Clinical report Apr 2, 2018 256
FDA Grants Orphan Drug Designation to MSC Product. Apr 2, 2018 328
FDA Grants Orphan Drug Designation to MSC Product. Apr 2, 2018 328
European Commission grants orphan drug designation to Soligenix's recombinant modified ricin toxin A-chain subunit. Mar 27, 2018 175
European Commission grants orphan drug designation to Soligenix's recombinant modified ricin toxin A-chain subunit. Mar 27, 2018 171
USFDA grants orphan drug designation to Proteostasis Therapeutics for PTI-428. Mar 16, 2018 192
USFDA grants orphan drug designation to Proteostasis Therapeutics for PTI-428. Mar 16, 2018 188
Protagonist Therapeutics Receives Orphan Drug Designation from FDA for PTG-300 for Treatment of Beta-Thalassemia. Mar 9, 2018 257
Supply Of Orphan Drugs And For The Treatment Of Rare Diseases. Mar 9, 2018 117
Protagonist Therapeutics passes US FDA orphan drug designation for PTG-300 to treat beta-thalassemia. Mar 7, 2018 197

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