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https://www.businesswire.com/news/home/20250624122250/en/Notice-of-Orphan-Drug-Designation-for-JR-446-for-Mucopolysaccharidosis-Type-IIIB-by-European-Commission-EC Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by European... JCR and MEDIPAL announced that the EC has granted ODD to JR-446, an investigational drug for the treatment of mucopolysaccharidosis type IIIB. orphan drug designation https://www.prweb.com/releases/andarix_pharmaceuticals_receives_pancreatic_cancer_orphan_drug_designation_from_the_fda/prweb12012733.htm Andarix Pharmaceuticals Receives Pancreatic Cancer Orphan Drug Designation from the FDA Somerville, Massachusetts (PRWEB) July 15, 2014 -- ANDARIX Pharmaceuticals, a leader in the discovery and development of targeted peptide therapy for cancer... orphan drug designation pancreatic cancer from the andarix pharmaceuticals https://investingnews.com/cardiol-therapeutics-granted-orphan-drug-designation-for-its-lead-drug-candidate-for-the-treatment-of-pericarditis/ Cardiol Therapeutics Granted Orphan Drug Designation for its Lead Drug Candidate for the Treatment... Feb 15, 2024 - Designation Based on Pre-Clinical Data and Initial Clinical Data from the Company's MAvERIC-Pilot Phase II StudyThis is a Designated News Release.Cardiol... orphan drug designation https://www.biospace.com/fda-grants-orphan-drug-designation-to-9mw2821 FDA Grants Orphan Drug Designation to 9MW2821 - BioSpace May 7, 2024 - Mabwell has been granted Orphan Drug Designation (ODD) by the U.S. Food and Drug Administration (FDA), for the treatment of esophageal cancer. orphan drug designation fda grants https://www.biospace.com/press-releases/neuronos-granted-fda-orphan-drug-designation-for-phelan-mcdermid-syndrome-a-neurodevelopmental-disorder-linked-to-autism NeuroNOS Granted FDA Orphan Drug Designation for Phelan-McDermid Syndrome, a Neurodevelopmental... orphan drug designation phelan mcdermid syndrome https://www.biospace.com/press-releases/cure-rare-disease-receives-orphan-drug-designation-from-fda-for-investigational-therapy-targeting-spinocerebellar-ataxia-type-3-sca3 Cure Rare Disease Receives Orphan Drug Designation from FDA for Investigational Therapy Targeting... cure rare disease orphan drug designation https://www.biospace.com/press-releases/quoin-pharmaceuticals-announces-european-medicines-agency-ema-grants-orphan-drug-designation-for-qrx003-for-the-treatment-of-netherton-syndrome Quoin Pharmaceuticals Announces European Medicines Agency (EMA) Grants Orphan Drug Designation for... european medicines agency orphan drug designation https://www.biospace.com/exegi-pharma-announces-fda-orphan-drug-designation-for-exe-346-live-biotherapeutic ExeGi Pharma Announces FDA Orphan Drug Designation for EXE-346 Live Biotherapeutic - BioSpace Nov 13, 2020 - ExeGi Pharma LLC, a company focused on developing live biotherapeutic ("LBP") drugs and probiotics, announced today that its biologic drug candidate, EXE-346,... orphan drug designation https://www.digitaljournal.com/pr/news/fda-grants-orphan-drug-designation-to-jaguar-health-for-crofelemer-for-microvillus-inclusion-disease-mvid-a-second-rare-disease-indication-in-the-us FDA Grants Orphan Drug Designation to Jaguar Health for Crofelemer for Microvillus Inclusion... orphan drug designation https://www.globenewswire.com/news-release/2015/03/24/718335/10126184/en/Agios-Pharmaceuticals-Announces-Orphan-Drug-Designation-of-AG-348-for-Treatment-of-Pyruvate-Kinase-Deficiency.html Agios Pharmaceuticals Announces Orphan Drug Designation of - PK deficiency is a severe and rare genetic disease with currently no approved or disease modifying treatments - CAMBRIDGE, Mass., March 24, 2015... orphan drug designation agios pharmaceuticals announces https://www.biospace.com/b-treeway-b-announces-orphan-drug-designation-for-tw001 Treeway Announces Orphan Drug Designation For TW001 - BioSpace orphan drug designation announces https://menafn.com/1109862340/Vivace-Therapeutics-Announces-Receipt-Of-Orphan-Drug-Designation-For-VT3989-For-Treatment-Of-Mesothelioma Vivace Therapeutics Announces Receipt Of Orphan Drug Designation For VT3989 For Treatment Of... Vivace Therapeutics Announces Receipt Of Orphan Drug Designation For VT3989 For Treatment Of Mesothelioma. Potential Best-in-Class TEAD Autopalmitoylation... orphan drug designation vivace therapeutics announces receipt https://www.biospace.com/marinus-pharmaceuticals-ganaxolone-receives-fda-orphan-drug-designation-in-pcdh19-female-epilepsy Marinus Pharmaceuticals' Ganaxolone Receives FDA Orphan Drug Designation In PCDH19 Female Epilepsy... orphan drug designation https://www.biospace.com/neurop-receives-fda-orphan-drug-designation-for-np10679-for-treatment-of-subarachnoid-hemorrhage NeurOp Receives FDA Orphan Drug Designation for NP10679 for Treatment of Subarachnoid Hemorrhage -... Dec 8, 2021 - NeurOp, Inc., a clinical-stage biotechnology company focused on neurological and psychiatric disorders, today announced that the U.S. Food and Drug... orphan drug designation https://www.prnewswire.com/news-releases/nova-mentis-earns-psilocybin-orphan-drug-designation-in-europe-for-fragile-x-syndrome-301408462.html Nova Mentis Earns Psilocybin Orphan Drug Designation in Europe for Fragile X Syndrome /PRNewswire/ - Nova Mentis Life Science Corp. (CSE: NOVA) (FSE: HN3Q) (OTCQB: NMLSF) ("NOVA" or the "Company"), a biotechnology company and global leader in... orphan drug designation https://www.prnewswire.co.uk/news-releases/phaserx-receives-positive-opinion-for-orphan-drug-designation-for-prx-otc-from-european-medicines-agency-620346943.html PhaseRx Receives Positive Opinion for Orphan Drug Designation for PRX-OTC from European Medicines... /PRNewswire/ -- PhaseRx, Inc. (NASDAQ: PZRX), a biopharmaceutical company developing mRNA treatments for life-threatening inherited liver diseases in... orphan drug designation https://www.prnewswire.com/news-releases/infinant-health-announces-submission-of-orphan-drug-designation-application-302105147.html Infinant Health Announces Submission of Orphan Drug Designation Application /PRNewswire/ -- Infinant Health, a privately-held company focused on changing the trajectory of human health, one baby at a time, announced it has filed an... orphan drug designation health announces submission application https://www.prnewswire.co.uk/news-releases/amo-pharma-receives-fda-orphan-drug-designation-for-amo-02-for-treatment-of-congenital-myotonic-636895603.html AMO Pharma Receives FDA Orphan Drug Designation for AMO-02 for Treatment of Congenital Myotonic... /PRNewswire/ -- AMO Pharma Limited ("AMO Pharma"), a privately held biopharmaceutical company focusing on debilitating diseases with limited or no treatment... orphan drug designation https://www.prnewswire.co.uk/news-releases/top-companies-receiving-orphan-drug-designation-in-2017-637165873.html Top Companies Receiving Orphan Drug Designation in 2017 /PRNewswire/ -- Under the Orphan Drug Act (ODA), drugs, vaccines, and diagnostic agents qualify for orphan status if they are intended to treat a disease... orphan drug designation top companies receiving 2017 https://www.finanznachrichten.de/nachrichten-2018-03/43259184-tetra-bio-pharma-inc-tetra-bio-pharma-announces-fda-orphan-drug-designation-for-ppp001-in-treatment-of-complex-regional-pain-syndrome-256.htm Tetra Bio-Pharma Inc.: Tetra Bio-Pharma Announces FDA Orphan Drug Designation for PPP001 in... OTTAWA, ONTARIO -- (Marketwired) -- 03/14/18 -- Tetra Bio-Pharma Inc. ("Tetra" or the "Corporation") (TSX VENTURE: TBP)(OTCQB: TBPMF), today announced that the... orphan drug designation bio pharma https://www.news-medical.net/news/20090831/Doxorubicin-granted-orphan-drug-designation-by-the-FDA.aspx Doxorubicin granted orphan drug designation by the FDA Jun 24, 2019 - Delcath Systems, Inc., a medical technology company testing its proprietary treatment method for primary and metastatic cancers to the liver, announced that... orphan drug designation by the doxorubicin granted fda https://www.newsfilecorp.com/release/117271 Kiora Pharmaceuticals Granted Orphan Drug Designation for KIO-301, an Investigational Drug for the... Salt Lake City, Utah--(Newsfile Corp. - March 18, 2022) - Kiora Pharmaceuticals, Inc. (NASDAQ: KPRX), ("Kiora" or the "Company") has... orphan drug designation https://www.fiercebiotech.com/biotech/cytokinetics-announces-orphan-drug-designation-granted-to-ck-2017357-for-treatment-of Cytokinetics Announces Orphan Drug Designation Granted to CK-2017357 for the Treatment of... Mar 10, 2010 - Cytokinetics Announces Orphan Drug Designation Granted to CK-2017357 for the Treatment of Amyotrophic Lateral Sclerosis South San Francisco, CA - March 10,... orphan drug designation https://www.biospace.com/pharmadrug-files-for-fda-orphan-drug-designation-for-dmt-in-stroke PharmaDrug Files for FDA Orphan Drug Designation for DMT in Stroke - BioSpace Feb 11, 2021 - Acute ischemic stroke is characterized by the sudden loss of blood circulation to an area of the brain, resulting in a corresponding loss of neurologic... orphan drug designation files fda https://www.biospace.com/praxis-precision-medicines-receives-orphan-drug-designation-for-prax-562-for-the-treatment-of-scn2a-deeprax-562-phase-2-clinical-trial-for-treatment-of-dees-expected-to-initiate-in-first-half-of-2022 Praxis Precision Medicines Receives Orphan Drug Designation for PRAX-562 for the Treatment of... Apr 30, 2021 - Praxis Precision Medicines, Inc. (NASDAQ: PRAX), a clinical-stage biopharmaceutical company translating genetic insights into the development of therapies for... orphan drug designation https://www.biospace.com/fda/eydisbio-receives-fda-orphan-drug-designation-for-tak1-inhibitor-for-the-treatment-of-systemic-sclerosis EydisBio Receives FDA Orphan Drug Designation for TAK1 Inhibitor for the Treatment of Systemic... orphan drug designation https://www.businesswire.com/news/home/20250107878739/en/AliveGen-Receives-FDA-Orphan-Drug-Designation-for-ALG-801-for-Pulmonary-Arterial-Hypertension AliveGen Receives FDA Orphan Drug Designation for ALG-801 for Pulmonary Arterial Hypertension AliveGen USA Inc. (AliveGen), a clinical stage biopharmaceutical company, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan... orphan drug designation https://www.biospace.com/ignyta-receives-orphan-drug-designation-from-fda-for-entrectinib-for-the-treatment-of-molecularly-defined-subsets-of-colorectal-cancer Ignyta Receives Orphan Drug Designation From FDA For Entrectinib For The Treatment Of Molecularly... orphan drug designation https://www.biospace.com/press-releases/avidity-biosciences-receives-orphan-drug-designation-in-japan-for-delpacibart-etedesiran-del-desiran-for-treatment-of-myotonic-dystrophy-type-1 Avidity Biosciences Receives Orphan Drug Designation in Japan for Delpacibart Etedesiran... orphan drug designation in japan avidity biosciences receives https://www.barchart.com/story/news/36621241/roxadustat-granted-orphan-drug-designation-for-the-treatment-of-myelodysplastic-syndromes-by-the-u-s-food-and-drug-administration Roxadustat Granted Orphan Drug Designation for the Treatment of Myelodysplastic Syndromes by the... Company is on track to file the Phase 3 protocol in the fourth quarter of 2025 SAN FRANCISCO, Dec. 15, 2025 (GLOBE NEWSWIRE) -- FibroGen, Inc. (NASDAQ: FGEN)... orphan drug designation for the https://investingnews.com/daily/life-science-investing/biotech-investing/matinas-biopharma-receives-orphan-drug-designation-for-mat2203/ Matinas BioPharma Receives Orphan Drug Designation for MAT2203 | INN Jan 2, 2026 - Matinas BioPharma Holdings (NYSE American:MTNB) has announced it has received orphan drug designation from the US Food and Drug administration for MAT2203, a... orphan drug designation biopharma receives inn https://www.newswire.com/news/repair-biotechnologies-receives-orphan-drug-designation-from-the-fda-22575375 Repair Biotechnologies Receives Orphan Drug Designation From the FDA for the Treatment of... orphan drug designation https://www.businesswire.com/news/home/20250225004448/en/FDA-Grants-Orphan-Drug-Designation-to-Revolutionary-Cystic-Fibrosis-Therapy FDA Grants Orphan Drug Designation to Revolutionary Cystic Fibrosis Therapy Porosome Therapeutics, Inc. announced the Orphan Drug Designation of its cystic fibrosis therapy by the Food and Drug Administration (FDA). orphan drug designation cystic fibrosis fda grants revolutionary https://www.sri.com/press/story/sri-earns-fda-orphan-drug-designation-for-pancreatic-cancer/ SRI earns FDA Orphan Drug Designation for pancreatic cancer - SRI Feb 19, 2025 - SRI's recent designation allow clinical development of immunotherapy treatment and brings more affordable therapies to market faster. orphan drug designation sri earns fda pancreatic https://www.prnewswire.co.uk/news-releases/asceneurons-tau-modifier-asn120290-receives-orphan-drug-designation-for-progressive-supranuclear-palsy-688472891.html Asceneuron's Tau Modifier ASN120290 Receives Orphan Drug Designation for Progressive Supranuclear... /PRNewswire/ -- Advancement of novel inhibitor targeting accumulation of toxic neurofibrillary tau tangles Asceneuron SA, an emerging leader in the... orphan drug designation https://www.finanznachrichten.de/nachrichten-2025-06/65742029-jcr-pharmaceuticals-co-ltd-notice-of-orphan-drug-designation-for-jr-446-for-mucopolysaccharidosis-type-iiib-by-european-commission-ec-004.htm JCR Pharmaceuticals Co., Ltd.: Notice of Orphan Drug Designation for JR-446 for... MEDIPAL HOLDINGS CORPORATION(TSE 7459, MEDIPAL) and JCR Pharmaceuticals Co., Ltd.(TSE 4552, JCR) today announced that the European Commission (EC) has granted... orphan drug designation co ltd https://www.biospace.com/ayala-pharmaceuticals-announces-al102-receives-orphan-drug-designation-for-desmoid-tumors Ayala Pharmaceuticals Announces AL102 Receives Orphan Drug Designation for Desmoid Tumors - BioSpace Nov 6, 2023 - Ayala Pharmaceuticals, Inc. (OTCQX: ADXS), a clinical-stage oncology company, today announced that the U.S. Food and Drug Administration (FDA) has granted... orphan drug designation https://www.biospace.com/ignyta-receives-orphan-drug-designation-from-fda-for-entrectinib-for-the-treatment-of-molecularly-defined-subsets-of-non-small-cell-lung-cancer Ignyta Receives Orphan Drug Designation From FDA For Entrectinib For The Treatment Of Molecularly... orphan drug designation https://www.biospace.com/ultimovacs-granted-orphan-drug-designation-by-fda-for-uv1-cancer-vaccine-in-metastatic-melanoma Ultimovacs Granted Orphan Drug Designation by FDA for UV1 Cancer Vaccine in Metastatic Melanoma -... Dec 2, 2021 - Ultimovacs ASA ("Ultimovacs") (OSE ULTI), a clinical stage leader in immune stimulatory vaccines for cancer, today announces that the U.S. Food and Drug... https://www.newswire.com/news/oncolyze-announces-fda-orphan-drug-designation-for-om-301-for-the-22007391 Oncolyze Announces FDA Orphan Drug Designation for OM-301 for the Treatment of Multiple Myeloma |... https://www.biospace.com/press-releases/u-s-fda-grants-orphan-drug-designation-to-adcentrx-therapeutics-adrx-0405-steap1-adc-for-gastric-cancer U.S. FDA Grants Orphan Drug Designation to Adcentrx Therapeutics' ADRX-0405 STEAP1 ADC for Gastric... https://www.biospace.com/ascentage-pharma-s-mdm2-p53-inhibitor-apg-115-alrizomadlin-granted-an-orphan-drug-designation-by-the-fda-for-the-treatment-of-stage-iib-iv-melanoma-marking-the-twelfth-obtained-by-the-company Ascentage Pharma's MDM2-p53 Inhibitor APG-115 (Alrizomadlin) Granted an Orphan Drug Designation by... Jul 21, 2021 - Ascentage Pharma, a globally focused biotechnology company engaged in developing novel therapies for cancers, chronic hepatitis B, and age-related diseases,... https://www.businesswire.com/news/home/20250507299102/en/Notice-of-Orphan-Drug-Designation-for-JR-446-for-Mucopolysaccharidosis-Type-IIIB-by-the-U.S.-FDA Notice of Orphan Drug Designation for JR-446 for Mucopolysaccharidosis Type IIIB by the U.S. FDA MEDIPAL and JCR announced that the U.S. FDA granted orphan drug designation (ODD) to JR-446, an investigational drug for the treatment of MPS IIIB. https://www.biospace.com/viracta-therapeutics-announces-orphan-drug-designation-granted-by-the-u-s-fda-to-nana-val-for-the-treatment-of-nasopharyngeal-carcinoma Viracta Therapeutics Announces Orphan Drug Designation Granted by the U.S. FDA to Nana-val for the... Dec 12, 2023 - Viracta Therapeutics, Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted an orphan drug designation to Nana-val (nanatinostat in... https://www.biospace.com/press-releases/tempest-receives-orphan-drug-designation-from-the-fda-for-tpst-1495-to-treat-patients-with-fap Tempest Receives Orphan Drug Designation from the FDA for TPST-1495 to Treat Patients with FAP -... https://www.biospace.com/kymera-therapeutics-receives-fda-orphan-drug-designation-for-kt-333-a-first-in-class-investigational-stat3-degrader-for-the-treatment-of-cutaneous-t-cell-lymphoma Kymera Therapeutics Receives FDA Orphan Drug Designation for KT-333, a First-in-Class,... Sep 15, 2022 - Kymera Therapeutics, Inc. today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to KT-333 for the treatment of... https://www.biospace.com/european-medicines-agency-ema-grants-orphan-drug-designation-for-moleculin-s-treatment-of-acute-myeloid-leukemia-aml European Medicines Agency (EMA) Grants Orphan Drug Designation for Moleculin's Treatment of Acute... Apr 18, 2024 - Moleculin Biotech, Inc., (Nasdaq: MBRX) (Moleculin or the Company), a clinical stage pharmaceutical company with a broad portfolio of drug candidates targeting... european medicines agency https://www.sanofi.com/en/media-room/press-releases/2025/2025-04-03-05-00-00-3054815 Press Release: Rilzabrutinib granted orphan drug designation in the US for two rare diseases with... Rilzabrutinib granted orphan drug designation in the US for two rare diseases with no approved medicines Designation granted for warm autoimmune hemolytic... https://www.legislation.gov.au/C2012G00255/asmade/details Designation of Adalimumab (HUMIRA) as an Orphan Drug - AbbVie Pty Ltd - Federal Register of... https://www.gsk.com/en-gb/media/press-releases/benlysta-granted-orphan-drug-designation-by-us-fda-for-the-potential-treatment-of-systemic-sclerosis/ Benlysta granted Orphan Drug Designation by US FDA for the potential treatment of systemic... GSK plans to initiate a phase II/III trial for systemic sclerosis associated interstitial lung disease (SSc-ILD) in the first half of 2023. https://www.takeda.com/newsroom/newsreleases/2016/japanese-mhlw-grants-orphan-drug-designation-in-japan-to-takedas-oral-proteasome-inhibitor-ixazomib-for-patients-with-relapsedrefractory-multiple-myeloma/ Japanese MHLW Grants Orphan Drug Designation in Japan to Takeda's Oral Proteasome Inhibitor... Japanese MHLW Grants Orphan Drug Designation in Japan to Takeda's Oral Proteasome Inhibitor Ixazomib for Patients with Relapsed/Refractory Multiple Myeloma https://www.fujifilm.com/us/en/news/ff-10832-granted-fda-orphan-drug-designation-for-the-treatment-of-biliary-tract-cancer FF-10832 Granted FDA Orphan Drug Designation for the Treatment of Biliary Tract Cancer | Fujifilm... https://www.biospace.com/press-releases/cero-therapeutics-holdings-inc-announces-fda-orphan-drug-designation-granted-to-cer-1236-for-the-treatment-of-acute-myeloid-leukemia-aml CERo Therapeutics Holdings, Inc. Announces FDA Orphan Drug Designation Granted to CER-1236 for the...