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Lava Therapeutics receives orphan drug designation for LAVA-051. Oct 15, 2021 231
Cantargia receives positive EMA opinion for orphan designation of nadunolimab in pancreatic cancer. Oct 14, 2021 176
Cantargia receives positive EMA opinion for orphan designation of nadunolimab in pancreatic cancer. Oct 14, 2021 177
Cantargia receives positive EMA opinion for orphan designation of nadunolimab in pancreatic cancer. Oct 14, 2021 178
PharmaTher granted orphan drug designation for ketamine to treat CRPS. Oct 13, 2021 176
Tezepelumab Granted Orphan Drug Designation in the US for Eosinophilic Esophagitis. Oct 11, 2021 773
Taysha Gene Therapies Receives Orphan Drug Designation from the European Commission for TSHA-101 for the Treatment of Infantile GM2 Gangliosidosis. Sep 30, 2021 432
Catalyst receives 'positive' decision from Appeals Court over Firdapse. Sep 30, 2021 344
Taysha Gene Therapies gets orphan drug designation from EC for TSHA-101. Sep 29, 2021 316
Bluebird Bio submits BLA to FDA for beti-cel gene therapy. Sep 21, 2021 155
Camurus granted orphan drug designation in the US for CAM2029 in polycystic liver disease. Sep 16, 2021 195
Camurus granted orphan drug designation in the US for CAM2029 in polycystic liver disease. Sep 16, 2021 196
Camurus granted orphan drug designation in the US for CAM2029 in polycystic liver disease. Sep 16, 2021 197
Jaguar Health unit Napo gets ODD for crofelemer to treat SBS-IF. Sep 15, 2021 179
ADC Therapeutics receives orphan drug designation in Europe for Zynlonta. Sep 13, 2021 175
ADC Therapeutics Receives Orphan Drug Designation in Europe for Zynlonta. Sep 13, 2021 670
Versantis Receives US FDA Orphan Drug Designation for VS-01. Sep 8, 2021 401
HIRA offers conditional approval for Xospata. Sep 3, 2021 186
HIRA offers conditional approval for Xospata. Sep 3, 2021 185
Polaryx Therapeutics wins FDA ODD for PLX-200 to treat GM2 Gangliosidoses. Aug 30, 2021 221
Polaryx Therapeutics receives Orphan Drug Designation for PLX-200. Aug 30, 2021 169
Polaryx Therapeutics wins FDA ODD for PLX-200 to treat GM2 Gangliosidoses. Aug 30, 2021 222
Polaryx Therapeutics receives Orphan Drug Designation for PLX-200. Aug 30, 2021 170
Octapharma's Octagam 10% Receives Seven Years of Market Exclusivity for Adult Dermatomyositis with FDA Orphan Drug Approval. Aug 30, 2021 453
Taysha Gene Therapies Receives Orphan Drug Designation for TSHA-105 for the Treatment of Epilepsy Caused by SLC13A5 Deficiency from the European Commission. Aug 27, 2021 231
Zogenix receives Orphan Drug Designation for FINTEPLA in Japan. Aug 26, 2021 157
US FDA Grants NFlection Therapeutics Orphan Drug Designation for NFX-179 for the Treatment of Cutaneous Neurofibromatosis Type 1. Aug 19, 2021 421
Swissmedic Accepts Marketing Authorization Application for Brukinsa in WaldenstrA[pilcrow sign]m's Macroglobulinaemia. Aug 19, 2021 343
Marinus Pharmaceuticals Reports Topline Ganaxolone Phase 2 Open-Label Results in Tuberous Sclerosis Complex and Receives FDA Orphan Drug Designation. Aug 18, 2021 685
Lantern Pharma's LP-184 receives US FDA Orphan Drug Designation. Aug 12, 2021 195
Lantern Pharma's LP-184 receives US FDA Orphan Drug Designation. Aug 12, 2021 196
C4 Therapeutics gets ODD from FDA for multiple myeloma treatment CFT7455. Aug 11, 2021 247
Aldeyra Therapeutics Receives Orphan Drug Designation from the US Food and Drug Administration for ADX-2191 to Treat Retinitis Pigmentosa. Aug 5, 2021 338
PharmaTher granted orphan drug designation for ketamine to treat ALS. Aug 4, 2021 221
US Orphan Drug Designation Granted to Autobahn Therapeutics' ABX-002 for the Treatment of X-linked Adrenoleukodystrophy. Aug 3, 2021 331
Aldeyra Therapeutics Receives Orphan Drug Designation from the US Food and Drug Administration for ADX-2191 to Treat Primary Vitreoretinal Lymphoma. Jul 21, 2021 303
FDA Grants Orphan Drug Designation for Pridopidine for the Treatment of Amyotrophic Lateral Sclerosis. Jul 14, 2021 321
TriAct Therapeutics wins FDA's orphan drug designation for iniparib for malignant glioma. Jul 8, 2021 197
TriAct Therapeutics wins FDA's orphan drug designation for iniparib for malignant glioma. Jul 8, 2021 198
Can-Fite done preparing for start of patient enrollment in Namodenoson study. Jul 8, 2021 202
VICO Therapeutics awarded FDA's ODD for VO659 for spinocerebellar ataxia. Jun 29, 2021 183
VICO Therapeutics awarded FDA's ODD for VO659 for spinocerebellar ataxia. Jun 29, 2021 182
EMA Grants Orphan Drug Designation to CLN5 Batten Disease Gene Therapy. Jun 29, 2021 459
Tracon Pharmaceuticals announces orphan drug designation for envafolimab. Jun 29, 2021 156
Grace Science Receives US FDA Orphan Drug Designation for GS-100, an AAV9 Gene Therapy for NGLY1 Deficiency. Jun 22, 2021 315
LATEST COMPANY NEWS. Jun 22, 2021 15620
Soleno gets FDA Orphan Drug Designation for Diazoxide Choline. Jun 2, 2021 222
Tenaya Therapeutics Receives Orphan Drug Designation and Presents Pre-Clinical Data for its Most Advanced Gene Therapy Product Candidate for Genetic Hypertrophic Cardiomyopathy. May 20, 2021 404
Everest Medicines receives South Korean Orphan Drug Designation for breast cancer drug sacituzumab govitecan-hziy. May 6, 2021 152
Everest Medicines receives South Korean Orphan Drug Designation for breast cancer drug sacituzumab govitecan-hziy. May 6, 2021 153
PharmaDrug becomes first company to receive Orphan Drug Designation for DMT from the US FDA. Apr 29, 2021 300
Revive Therapeutics files Orphan Drug Designation application with FDA for Psilocybin to treat moderate to severe traumatic brain injury (TBI). Apr 28, 2021 691
Revive Therapeutics has repurposed gout drug to fight coronavirus and is pushing into psychedelic medicine. Apr 23, 2021 1164
IQ-AI to finance brain tumour trial. Apr 14, 2021 314
Syndax Pharmaceuticals' axatilimab receives US FDA Orphan Drug Designation. Apr 9, 2021 229
Syndax Pharmaceuticals' axatilimab receives US FDA Orphan Drug Designation. Apr 9, 2021 228
Passage Bio receives Orphan designation from European Commission for PBKR03. Apr 5, 2021 192
EMA grants Orphan Drug Designation to JR-171 for the Treatment of Mucopolysaccaridosis Type I. Mar 31, 2021 295
Intellia Therapeutics treatment NTLA-2001 receives EU orphan drug designation. Mar 30, 2021 237
Precigen's PRGN-2012 receives US FDA orphan drug designation. Mar 19, 2021 190
Precigen's PRGN-2012 receives US FDA orphan drug designation. Mar 19, 2021 189
US FDA grants CiRC Biosciences's chemically induced photoreceptor-like cells Orphan Drug Designation. Mar 11, 2021 193
US FDA grants CiRC Biosciences's chemically induced photoreceptor-like cells Orphan Drug Designation. Mar 11, 2021 192
Nuvation Bio Granted Orphan Drug Designation for NUV-422 for the Treatment of Patients with Malignant Gliomas. Mar 11, 2021 462
PharmaDrug files Orphan Drug Designation with US FDA for the use of DMT in kidney transplants. Feb 26, 2021 487
PharmaDrug pursuing Orphan Drug Designation with FDA to use DMT in kidney transplants. Feb 26, 2021 160
PharmaDrug files Orphan Drug Designation application with the US Food and Drug Administration for use of N,N-Dimethyltryptamine (DMT) in the treatment of acute ischemic stroke patients. Feb 12, 2021 786
Revive Therapeutics provides a corporate update on its cannabinoid pharmaceuticals program. Feb 12, 2021 1042
Polaryx Therapeutics' PLX-300 receives US FDA Rare Pediatric Disease and Orphan Drug Designations. Feb 11, 2021 231
Polaryx Therapeutics' PLX-300 receives US FDA Rare Pediatric Disease and Orphan Drug Designations. Feb 11, 2021 227
Elevar Therapeutics receives US FDA orphan drug designation for rivoceranib (apatinib). Feb 9, 2021 192
Elevar Therapeutics receives US FDA orphan drug designation for rivoceranib (apatinib). Feb 9, 2021 191
Ascentage Pharma receives US FDA Orphan Drug Designation for APG-2575. Jan 29, 2021 219
Ascentage Pharma receives US FDA Orphan Drug Designation for APG-2575. Jan 29, 2021 223
Immunoforge's 'PF1801' Is Designated as Orphan Drug by FDA. Jan 29, 2021 283
Q BioMed drug receives US Food & Drug Administration orphan drug designation. Jan 28, 2021 339
US FDA's Office of Orphan Products Development grants Q Biomed's Uttroside-B Orphan Drug Designation. Jan 28, 2021 180
US FDA's Office of Orphan Products Development grants Q Biomed's Uttroside-B Orphan Drug Designation. Jan 28, 2021 184
Immunicum granted FDA Orphan Drug Designation for ilixadencel in STS. Jan 26, 2021 203
Immunicum granted FDA Orphan Drug Designation for ilixadencel in STS. Jan 26, 2021 204
Immunicum granted FDA Orphan Drug Designation for ilixadencel in STS. Jan 26, 2021 199
Xspray Pharma commences study with modified formulation of HyNap-Dasa. Jan 22, 2021 184
Xspray Pharma commences study with modified formulation of HyNap-Dasa. Jan 22, 2021 180
Xspray Pharma commences study with modified formulation of HyNap-Dasa. Jan 22, 2021 185
FDA Grants Orphan Drug Designation for PVSRIPO for the Treatment of Advanced Melanoma. Jan 11, 2021 493
Antengene submits Korean New Drug Application for ATG-010 (Selinexor). Jan 4, 2021 217
Antengene submits Korean New Drug Application for ATG-010 (Selinexor). Jan 4, 2021 213
Antengene submits ATG-010 (Selinexor) NDA in South Korea's MFDS for rrMM and rrDLBCL. Jan 4, 2021 292
Antengene submits ATG-010 (Selinexor) NDA in South Korea's MFDS for rrMM and rrDLBCL. Jan 4, 2021 288
USFDA grants Orphan Drug Designation to Moleculin Biotech's Annamycin. Dec 30, 2020 166
USFDA grants Orphan Drug Designation to Moleculin Biotech's Annamycin. Dec 30, 2020 162
Annamycin Receives FDA Orphan Drug Designation for Soft Tissue Sarcomas. Dec 30, 2020 337
Amryt Pharma treatment awarded US orphan status for fragile skin condition. Dec 24, 2020 317
Amryt Pharma treatment awarded US orphan status for fragile skin condition. Dec 24, 2020 315
HemoShear Therapeutics receives US FDA Orphan Drug Designations for HST5040. Dec 17, 2020 180
HemoShear Therapeutics receives US FDA Orphan Drug Designations for HST5040. Dec 17, 2020 176
Emmaus wins Endari's orphan drug status in Switzerland. Dec 4, 2020 218
Emmaus wins Endari's orphan drug status in Switzerland. Dec 4, 2020 222
BrainEver Receives FDA Orphan Drug Designation for BREN-02 for the Treatment of Amyotrophic Lateral Sclerosis. Nov 27, 2020 453
US FDA grants both Rare Pediatric Disease and Orphan Drug designations to Polaryx Therapeutics' PLX-300. Nov 26, 2020 185
US FDA grants both Rare Pediatric Disease and Orphan Drug designations to Polaryx Therapeutics' PLX-300. Nov 26, 2020 181
Omeros Submits its Biologics License Application to US FDA for Narsoplimab in HSCT-TMA. Nov 18, 2020 740
ExeGi Pharma Announces FDA Orphan Drug Designation for EXE-346 Live Biotherapeutic. Nov 13, 2020 338
Partnership To Manufacture MSC Platform Therapy. Nov 2, 2020 181
Bausch Health Companies receives US FDA Orphan Drug Designation for sickle cell disease drug rifaximin. Oct 30, 2020 211
Bausch Health Companies receives US FDA Orphan Drug Designation for sickle cell disease drug rifaximin. Oct 30, 2020 207
TRACON wins FDA Orphan Drug status for TRC102 in malignant glioma. Oct 27, 2020 526
Taysha gets rare FDA pediatric disease, orphan drug designations for TSHA-104. Oct 27, 2020 226
European Medicines Agency (EMA) grants Novartis's iptacopan (LNP023) orphan drug designation for IgA nephropathy (IgAN). Oct 26, 2020 205
European Medicines Agency (EMA) grants Novartis's iptacopan (LNP023) orphan drug designation for IgA nephropathy (IgAN). Oct 26, 2020 201
Tracon Pharmaceuticals announces FDA orphan drug designation for TRC102. Oct 26, 2020 182
BrainStorm, Catalent partner for manufacture of NurOwn. Oct 22, 2020 175
Novartis's Branaplam Gets US Orphan Drug Designation In Huntington's. Oct 21, 2020 195
Taysha gets FDA rare pediatric disease and orphan drug designations. Oct 14, 2020 246
US FDA grants Ascentage Pharma's APG-115 and APG-1252 two Orphan Drug Designations. Oct 9, 2020 220
US FDA grants Ascentage Pharma's APG-115 and APG-1252 two Orphan Drug Designations. Oct 9, 2020 216
Xenon on track to initiate Phase 3 trial with KCNQ2-DEE before year-end. Oct 8, 2020 348
Eyevensys Receives FDA Orphan Drug Designation for EYS611 for Treatment of Retinitis Pigmentosa. Oct 5, 2020 620
US FDA Grants Orphan Drug Designation to Ionis Treatment for Alexander Disease. Oct 1, 2020 412
US FDA grants Orphan Drug and Fast Track designations to AbbVie's elezanumab. Sep 29, 2020 204
HUYA Bioscience receives Japanese orphan drug designation for HBI-8000. Sep 29, 2020 163
US FDA grants Orphan Drug and Fast Track designations to AbbVie's elezanumab. Sep 29, 2020 200
HUYA Bioscience receives Japanese orphan drug designation for HBI-8000. Sep 29, 2020 159
AbbVie gets FDA orphan drug, fast tract designations for elezanumab. Sep 28, 2020 174
Avrobio Receives Orphan Drug Designation from the European Medicines Agency for AVR-RD-02, an Investigational Gene Therapy for Gaucher Disease. Sep 28, 2020 476
Avrobio gets orphan drug designation from the EC for AVR-RD-02. Sep 28, 2020 245
Mustang Bio announces Orphan Drug Designation for MB-207. Sep 22, 2020 370
Zynerba Pharmaceuticals cheers FDA decision to grant orphan drug designation for CBD to treat 22q deletion syndrome. Sep 18, 2020 283
Ascentage Pharma receives US FDA Orphan Drug Designation for APG-2575. Sep 8, 2020 256
Ascentage Pharma receives US FDA Orphan Drug Designation for APG-2575. Sep 8, 2020 260
Project ALS receives US FDA orphan drug designation for Prosetin. Sep 7, 2020 158
Project ALS receives US FDA orphan drug designation for Prosetin. Sep 7, 2020 154
Mustang Bio announces Orphan Drug Designation for MB-107. Sep 2, 2020 336
VelosBio Receives FDA Fast Track and Orphan Drug Designations for VLS-101 in Patients with Mantle Cell Lymphoma. Sep 2, 2020 333
Tolinapant Granted Orphan Drug Designation for the Treatment of T-cell Lymphomas by the US FDA. Sep 2, 2020 409
Kazia Therapeutics awarded US FDA Orphan Drug Designation to paxalisib treatment for malignant glioma. Aug 25, 2020 507
Kazia Therapeutics awarded US FDA Orphan Drug Designation to paxalisib treatment for malignant glioma. Aug 25, 2020 505
Kubota Vision Receives Orphan Products Clinical Trials Grants to Emixustat for Stargardt Disease. Aug 24, 2020 548
US FDA grants Kazia Therapeutics' Paxalisib orphan drug designation. Aug 24, 2020 154
US FDA grants Kazia Therapeutics' Paxalisib orphan drug designation. Aug 24, 2020 158
Krystal Biotech's KB407 granted orphan drug designation by the FDA to treat CF. Aug 17, 2020 190
Ocugen receives Orphan Drug designation for OCU400 gene therapy. Aug 10, 2020 387
Eloxx Pharmaceuticals reports Q2 EPS (20c), consensus (21c). Financial report Aug 6, 2020 164
Fortress gets 'positive opinion' on orphan drug designation from EMA. Jul 31, 2020 190
US FDA grants Regulus Therapeutics Orphan Drug Designation for RGLS4326. Jul 30, 2020 176
US FDA grants Regulus Therapeutics Orphan Drug Designation for RGLS4326. Jul 30, 2020 172
Ocugen granted Orphan Drug Designation for OCU-400 gene therapy. Jul 27, 2020 268
Centogene, Molecular Health enter collaboration for orphan drugs. Jul 27, 2020 155
Seelos Therapeutics Receives Positive EMA Opinion on Orphan Drug Designation for SLS-005 (Trehalose) in Sanfilippo Syndrome. Jul 23, 2020 191
Seelos Therapeutics Receives Positive EMA Opinion on Orphan Drug Designation for SLS-005 in Sanfilippo Syndrome. Jul 22, 2020 190
Therapeutics steps closer to Orphan Drug Designation in Europe for SLS-005 to treat Sanfilippo syndrome. Jul 22, 2020 153
Kiniksa receives Orphan Drug designation for rilonacept for pericarditis. Jul 16, 2020 223
Pharmaxis granted orphan drug designation for PXS-5505 by FDA for treatment of myelofibrosis. Jul 15, 2020 405
Clinigen Group says cancer treatment receives US orphan drug designation for ALS. Jul 14, 2020 268
Clinigen Gets US Orphan Drug Designation For ALS Treatment Aldesleukin. Jul 13, 2020 212
Amryt Pharma: Cantor Fitzgerald backs orphan drug group for NASDAQ success. Jul 10, 2020 301
Amryt Pharma: Cantor Fitzgerald backs orphan drug group for NASDAQ success. Jul 10, 2020 299
Crinetics gets orphan drug designation for paltusotine to treat acromegaly. Jul 8, 2020 222
AnaptysBio gets orphan drug designation of imsidolimab for pustular psoriasis. Jul 8, 2020 252
Revive Therapeutics returns with repurposed gout drug to fight COVID-19 and push into psychedelic medicine. Jul 3, 2020 939
US FDA grants Orphan Drug Designation to ISA Pharmaceuticals' ISA101b. Jul 2, 2020 159
US FDA grants Orphan Drug Designation to ISA Pharmaceuticals' ISA101b. Jul 2, 2020 155
ALGOTHERAPEUTIX ORPHAN DRUG DESIGNATION GRANTED. Jul 1, 2020 309
AstraZeneca Drug Selumetinib Gets Orphan Drug Designation In Japan. Jun 30, 2020 379
Oncternal Therapeutics announces orphan drug designations of cirmtuzumab by FDA. Jun 30, 2020 178
US FDA Office of Orphan Products Development grants Orphan Drug Designation to Reneo Pharmaceuticals' REN001. Jun 24, 2020 154
Protagonist Therapeutics receives Orphan Drug Designation for PTG-300. Jun 18, 2020 162
Aldeyra receives Orphan Medicinal Product Designation from EC for ADX-2191. Jun 15, 2020 248
CNS awarded FDA's orphan drug designation for berubicin for brain cancer. Jun 12, 2020 224
CNS awarded FDA's orphan drug designation for berubicin for brain cancer. Jun 12, 2020 228
US FDA grants orphan drug designation to Leap Therapeutics' DKN-01. Jun 12, 2020 154
Agios gets FDA orphan drug designation for mitapivat for thalassemia treatment. Jun 8, 2020 202
AstraZeneca Says Enhertu Granted Orphan Status In US. May 22, 2020 253
GLOBAL TOP NEWS SUMMARY: Stocks Sink As China Exerts Control In HK. May 22, 2020 2326
Alpha Cognition awarded EU patents and US FDA orphan drug designation for progranulin platform for treating neurodegenerative diseases. May 13, 2020 205
Alpha Cognition awarded EU patents and US FDA orphan drug designation for progranulin platform for treating neurodegenerative diseases. May 13, 2020 201
Medivir AB announces FDA orphan drug designation for MIV-818 to treat hepatocellular carcinoma. May 6, 2020 178
Medivir AB announces FDA orphan drug designation for MIV-818 to treat hepatocellular carcinoma. May 6, 2020 182
Medivir announces receipt of positive opinion on orphan designation in EU for drug candidate MIV-818. May 1, 2020 171
Medivir announces receipt of positive opinion on orphan designation in EU for drug candidate MIV-818. May 1, 2020 166
Medivir announces receipt of positive opinion on orphan designation in EU for drug candidate MIV-818. May 1, 2020 170
Seelos Therapeutics shares gain as it receives Orphan Drug designation for SLS-005. May 1, 2020 319
US FDA grants Orphan Drug designation to Marker Therapeutics' MT-401 leukaemia treatment. Apr 30, 2020 312
US FDA grants Orphan Drug designation to Marker Therapeutics' MT-401 leukaemia treatment. Apr 30, 2020 308
Adaptimmune announces positive COMP opinion for ADP-A2M4 in EU. Apr 28, 2020 206
CNS Pharmaceuticals Files for FDA Orphan Drug Designation for Brain Cancer Drug Berubicin. Apr 27, 2020 444
US FDA Grants Orphan Drug Designation to AmideBio's Glucagon Analog for the Treatment of Congenital Hyperinsulinism. Apr 27, 2020 278
US FDA grants Orphan Drug Designation to Clarity Pharmaceuticals' 67Cu-SARTATE. Apr 22, 2020 259
US FDA grants Orphan Drug Designation to Clarity Pharmaceuticals' 67Cu-SARTATE. Apr 22, 2020 255
Eton Pharmaceuticals Acquires US Marketing Rights to Pediatric Orphan Drug Alkindi Sprinkle from Diurnal to Enhance Pediatric Portfolio. Mar 31, 2020 370
Eton Pharmaceuticals Acquires US Marketing Rights to Pediatric Orphan Drug Alkindi Sprinkle from Diurnal to Enhance Pediatric Portfolio. Mar 31, 2020 333
Ryvu Therapeutics wins FDA orphan drug designation for SEL120 to treat acute myeloid leukemia. Mar 30, 2020 224
Ryvu Therapeutics wins FDA orphan drug designation for SEL120 to treat acute myeloid leukemia. Mar 30, 2020 220
US FDA grants orphan drug designation to Ryvu Therapeutics' SEL120. Mar 30, 2020 236
US FDA grants orphan drug designation to Ryvu Therapeutics' SEL120. Mar 30, 2020 232
AlloVir's Viralym-M Granted Orphan Drug Designation by European Medicines. Mar 30, 2020 797
APR Applied Pharma Research granted FDA orphan drug designation for APR-OD031 for treating PKU. Mar 27, 2020 223
US FDA grants Orphan Drug Designation to PR Applied Pharma Research's APR-OD031. Mar 27, 2020 264
APR Applied Pharma Research granted FDA orphan drug designation for APR-OD031 for treating PKU. Mar 27, 2020 227
US FDA grants Orphan Drug Designation to PR Applied Pharma Research's APR-OD031. Mar 27, 2020 268
Eton acquires marketing rights to pediatric orphan drug Alkindi Sprinkle. Mar 27, 2020 217
Pharma giant under fire for drug exclusivity. EDWARD THICKNESSE @edthicknesse Mar 25, 2020 173
Gilead submits request to FDA to rescind remdesivir orphan drug designation. Mar 25, 2020 232
Celsion's GEN-1 immunotherapy receives orphan drug designation from EMA. Mar 23, 2020 185
Spero Therapeutics receives FDA orphan drug designation for SPR720. Mar 11, 2020 199
Harbour BioMed Receives US FDA IND Approval for Phase 2 Clinical Trial and Orphan Drug. Mar 4, 2020 421
Diurnal Group boosts revenues from Alkindi by 516%, as losses drop dramatically. Feb 12, 2020 359
AstraZeneca's Imfinzi granted orphan status as liver cancer treatment. Jan 21, 2020 384
AstraZeneca announces grant of Orphan Drug Designation in the US to Imfinzi and tremelimumab for liver cancer treatment. Jan 21, 2020 420
AstraZeneca announces grant of Orphan Drug Designation in the US to Imfinzi and tremelimumab for liver cancer treatment. Jan 21, 2020 416
Imfinzi and tremelimumab granted Orphan Drug Designation in the US for liver cancer. Jan 20, 2020 1635
US FDA grants Orphan Drug Designation to OBI Pharma's OBI-999. Dec 27, 2019 210
US FDA grants Orphan Drug Designation to OBI Pharma's OBI-999. Dec 27, 2019 206
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
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
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
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
Akari Therapeutics' nomacopan granted U.S. orphan drug designation for BP. Sep 13, 2019 208
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
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
Mike Page, Alexion Pharmaceuticals Invites You to SMi's Orphan Drugs & Rare Diseases Conference. Conference news Sep 1, 2019 444
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 279
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
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
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

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