WASHINGTON, Aug. 28, 2025 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VGT-1849B, a selective peptide nucleic acid-based JAK2 inhibitor for the treatment of polycythemia vera (PV). PV is a chronic myeloproliferative disorder characterized by aberrant hematopoiesis of myeloid lineage with exuberant red cell production...Read more
SAN DIEGO, Aug. 21, 2025 (GLOBE NEWSWIRE) -- Crinetics Pharmaceuticals, Inc. (Nasdaq: CRNX) today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) for atumelnant, a novel, once-daily oral adrenocorticotropic hormone (ACTH) receptor antagonist candidate for the proposed treatment of classic congenital adrenal hyperplasia (CAH). Atumelnant is the first and only small molecule ACTH receptor...Read more
LEXINGTON, Mass., Aug. 20, 2025 (GLOBE NEWSWIRE) -- Keros Therapeutics, Inc. (“Keros”) (Nasdaq: KROS), a clinical-stage biopharmaceutical company focused on developing and commercializing novel therapeutics to treat a wide range of patients with disorders that are linked to dysfunctional signaling of the transforming growth factor-beta (“TGF-ß”) family of proteins, today announced the U.S. Food and Drug Administration (“FDA”) granted...Read more
Provides SGX945 Seven Years of U.S. Market Exclusivity Upon FDA Approval PRINCETON, N.J., Aug. 18, 2025 /PRNewswire/ -- Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that the Office of Orphan Products Development of the United States (U.S.) Food and...Read more
DURHAM, N.C. / Jul 23, 2025 / Business Wire / Precision BioSciences, Inc. (Nasdaq: DTIL), a clinical stage gene editing company utilizing its novel proprietary ARCUS® platform to develop in vivo gene editing therapies for diseases with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for PBGENE-DMD for the treatment of Duchenne muscular dystrophy (DMD). “Receipt of Orphan...Read more
PRISM-1, the ongoing registrational, Phase 3 study evaluating quemliclustat plus chemotherapy as a first-line treatment for metastatic pancreatic cancer, is expected to be fully enrolled by the end of this year. HAYWARD, Calif. / Jul 10, 2025 / Business Wire / Arcus Biosciences, Inc. (NYSE:RCUS), a clinical-stage, global biopharmaceutical company focused on developing differentiated molecules and combination therapies for patients with...Read more
In an ongoing Phase 1 trial published in Nature Medicine, MB-101 was well-tolerated and 50% of patients achieved stable disease or better with two partial responses and two complete responses lasting 7.5 and 66+ months, respectively Preclinical data support a novel combination of MB-101 (IL13Rα2‐targeted CAR-T cell therapy) and MB-108 (HSV-1 oncolytic virus) to optimize clinical results FDA previously granted Orphan Drug Designation...Read more
Ongoing progress in Phase 1 clinical trial of SENTI-202 for the treatment of Acute Myeloid Leukemia (AML) 20,800 newly diagnosed AML patients in the U.S. every year1 with 60% of patients experiencing relapse or death within 12 months2 Management releases Virtual Investor “What This Means” segment; Access here SOUTH SAN FRANCISCO, Calif., June 18, 2025 (GLOBE NEWSWIRE) -- Senti Biosciences, Inc. (Nasdaq: SNTI) (“Senti Bio” or the...Read more
U.S. FDA grants Orphan Drug Designation to Deramiocel for the treatment of Becker Muscular Dystrophy, broadening Capricor’s focus in neuromuscular diseases Capricor remains on track for the August 31, 2025, PDUFA date for Deramiocel in Duchenne Muscular Dystrophy following successful FDA Pre-License Inspection SAN DIEGO, June 17, 2025 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company developing...Read more
DUBLIN, June 05, 2025 (GLOBE NEWSWIRE) -- Avadel Pharmaceuticals plc (Nasdaq: AVDL), a biopharmaceutical company focused on transforming medicines to transform lives, announced today that LUMRYZTM has been granted Orphan Drug Designation (ODD) from the U.S. Food & Drug Administration (FDA) for the treatment of Idiopathic Hypersomnia (IH). Specifically, ODD was granted based on the plausible hypothesis that LUMRYZ may be...Read more
CAMBRIDGE, Mass., June 03, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted orphan drug designation to BEAM-101, an investigational genetically modified cell therapy for the treatment of sickle cell disease (SCD). “Sickle cell disease is a...Read more
CAMBRIDGE, Mass., May 29, 2025 (GLOBE NEWSWIRE) -- Beam Therapeutics Inc. (Nasdaq: BEAM), a biotechnology company developing precision genetic medicines through base editing, today announced that the United States (U.S.) Food and Drug Administration (FDA) has granted orphan drug designation to BEAM-302, a liver-targeting lipid-nanoparticle (LNP) formulation of a guide RNA and an mRNA encoding a base editor designed to correct the...Read more
Company advancing plans to develop TPI 287, a novel, late stage abeotaxane that appears to cross the blood-brain barrier and has published clinical efficacy data in glioblastoma multiforme (GBM) Management releases "What This Means" segment discussing the receipt of Orphan Drug Designations for TPI 287; Access here HOUSTON, TX / ACCESS Newswire / May 13, 2025 / CNS Pharmaceuticals, Inc. (NASDAQ:CNSP) ("CNS" or the "Company"), a...Read more
The FDA designation marks a significant milestone in the development of a novel treatment for a rare neurodevelopmental disorder Plan to initiate first-in-human U.S. clinical trials of lead drug candidate for treating ASD in 2026 BOSTON, April 21, 2025 (GLOBE NEWSWIRE) -- NeuroNOS, a biopharmaceutical company focused on developing treatments for autism and Alzheimer disorders, and a subsidiary of Beyond Air (NASDAQ: XAIR), today...Read more
BRISBANE, Calif., April 21, 2025 (GLOBE NEWSWIRE) -- Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-class1 targeted and immune-mediated therapeutics to fight cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to TPST-1495, the company’s novel dual receptor inhibitor of prostaglandin (PGE2) signaling, for the treatment of...Read more
Gloperba® is the first and only liquid oral version of the anti-gout medicine colchicine indicated for the prophylaxis of painful gout flares in adults. Pericarditis is an inflammation of the pericardium, the sac-like membrane that surrounds and protects the heart. It occurs when the pericardium becomes irritated or infected, leading to swelling and pain. PALO ALTO, Calif., April 16, 2025 (GLOBE NEWSWIRE) -- Scilex Holding Company...Read more
Initiated IND-enabling toxicology program IND submission planned for 4Q25; Clinical data expected in 1H26 CRANBURY, N.J., March 25, 2025 /PRNewswire/ -- Palatin Technologies, Inc. (NYSE American: PTN), a biopharmaceutical company developing first-in-class medicines based on molecules that modulate the activity of the melanocortin receptor system, today announced the US Food & Drug Administration (FDA) has granted "orphan...Read more
Orphan Drug Designation follows positive Phase 1 data presented at the 12th International Workshop on Waldenström Macroglobulinemia First-in-class Bruton’s tyrosine kinase (BTK) degrader NX-5948 assigned nonproprietary name “bexobrutideg” in newly named degrader class of drugs SAN FRANCISCO, March 17, 2025 (GLOBE NEWSWIRE) -- Nurix Therapeutics, Inc. (Nasdaq: NRIX), a clinical-stage biopharmaceutical company focused on the...Read more
CAMBRIDGE, Mass., March 14, 2025 (GLOBE NEWSWIRE) -- Korro Bio, Inc. (Korro) (Nasdaq: KRRO), a clinical-stage biopharmaceutical company focused on developing a new class of genetic medicines based on editing RNA for both rare and highly prevalent diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the investigational medicine KRRO-110 for the treatment of Alpha-1 Antitrypsin...Read more
HOUSTON, March 06, 2025 (GLOBE NEWSWIRE) -- Plus Therapeutics, Inc. (Nasdaq: PSTV) (the “Company” or “Plus Therapeutics”), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system cancers, today announces that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Rhenium (186Re) Obisbemeda for the treatment of...Read more
KING OF PRUSSIA, Pa., Jan. 28, 2025 (GLOBE NEWSWIRE) -- SEED Therapeutics Inc. (“SEED”), a biotechnology company pioneering the discovery of molecular glues for targeted protein degradation (TPD) using its proprietary RITE3™ platform, today announced that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease and Orphan Drug designations to SEED’s ST-01156 molecular glue. This novel agent degrades RBM39, an RNA...Read more
SHANGHAI & CAMBRIDGE, Mass. / Jan 22, 2025 / Business Wire / Zai Lab Limited (NASDAQ: ZLAB; HKEX: 9688) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ZL-1310, a potential highly active first-in-class DLL3 antibody-drug conjugate (ADC), for the treatment of small cell lung cancer (SCLC). “Receiving an Orphan Drug Designation for ZL-1310 recognizes its potential to treat patients...Read more
Orphan drug designation paves the way for potential of sebetralstat to be first oral on-demand treatment for HAE in Japan Submission advances Company’s strategic plan to address unmet needs in HAE on global scale CAMBRIDGE, Mass. & SALISBURY, England / Jan 21, 2025 / Business Wire / KalVista Pharmaceuticals, Inc. (NASDAQ: KALV), today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW) has granted sebetralstat...Read more
SOUTH SAN FRANCISCO, Calif., Jan. 9, 2025 /PRNewswire/ -- Rigel Pharmaceuticals, Inc. (Nasdaq: RIGL), a commercial stage biotechnology company focused on hematologic disorders and cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to R289 for the treatment of myelodysplastic syndromes (MDS). R2891, Rigel's potent and selective dual inhibitor of IRAK1 and IRAK4, is being...Read more
BRISBANE, Calif., Jan. 06, 2025 (GLOBE NEWSWIRE) -- Tempest Therapeutics, Inc. (Nasdaq: TPST), a clinical-stage biotechnology company developing first-in-class1 targeted and immune-mediated therapeutics to fight cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to amezalpat (TPST-1120), an oral, small molecule, selective PPAR⍺ antagonist for the treatment of patients with...Read more
WASHINGTON, Dec. 20, 2024 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VGT-1849A, a selective antisense oligonucleotide (ASO)-based JAK2 inhibitor for the treatment of polycythemia vera (PV), a form of a rare hematologic malignancy that is estimated to affect 1 in 2000 Americans.1 PV is a chronic myeloproliferative...Read more
WARREN, N.J., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Aquestive Therapeutics, Inc. (NASDAQ: AQST) (“Aquestive” or the “Company”), a pharmaceutical company advancing medicines to bring meaningful improvement to patients' lives through innovative science and delivery technologies, today announced the U.S. Food and Drug Administration (FDA) has granted seven years of orphan drug exclusivity (ODE) to Libervant® (diazepam) Buccal Film for the acute...Read more
World Health Organization (WHO) has classified the global resurgence of cholera at the highest internal level for emergencies; 1.3 to 4 million cholera cases and 21,000 to 143,000 cholera-related deaths occur each year worldwide Cholera is an acute diarrheal illness caused by infection of the intestine with the bacterium Vibrio cholerae Crofelemer previously granted orphan-drug designation by the FDA and the European Medicines Agency...Read more
WESTON, Fla., Dec. 9, 2024 /PRNewswire/ -- Cantex Pharmaceuticals, Inc., a clinical-stage pharmaceutical company focused on developing transformative therapies for cancer and other life-threatening medical conditions, announced today that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to Cantex's azeliragon for the treatment of brain metastasis from breast cancer. This new azeliragon designation adds to...Read more
REDWOOD CITY, Calif., Dec. 03, 2024 (GLOBE NEWSWIRE) -- Rezolute, Inc. (Nasdaq: RZLT) (“Rezolute” or the “Company”), a late-stage biopharmaceutical company dedicated to developing transformative therapies for rare diseases with serious unmet needs, today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to ersodetug for the treatment of hypoglycemia due to tumor HI. "FDA's granting of Orphan...Read more
Designation underscores significant unmet need in frontotemporal dementia and the potential role of neflamapimod in multiple neurologic disorders On track to report topline data from the RewinD-LB Phase 2b clinical trial in early-stage dementia with Lewy bodies (DLB) in December 2024 BOSTON, Nov. 27, 2024 (GLOBE NEWSWIRE) -- CervoMed Inc. (NASDAQ: CRVO), a clinical-stage company focused on developing treatments for age-related...Read more
MB-108 (HSV-1 oncolytic virus) is active and well tolerated in patients with recurrent glioblastoma in ongoing Phase 1 clinical trial Preclinical data support a novel combination of MB-108 (HSV-1 oncolytic virus) and MB-101 (IL13Rα2‐targeted CAR-T cell therapy) to optimize clinical results WORCESTER, Mass., Nov. 07, 2024 (GLOBE NEWSWIRE) -- Mustang Bio, Inc. (“Mustang” or the “Company”) (Nasdaq: MBIO), a clinical-stage...Read more
NASHVILLE, Tenn., Nov. 6, 2024 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (NASDAQ: CPIX), a specialty pharmaceutical company, announced today that the United States (U.S.) Food and Drug Administration (FDA) granted Orphan Drug Designation and Rare Pediatric Disease Designation to Ifetroban for the treatment of cardiomyopathy associated with Duchenne muscular dystrophy (DMD). Cumberland is completing the FIGHT DMD™ trial, a...Read more
Immuneering recently announced positive initial Phase 2a data, including complete and partial responses, with IMM-1-104 in combination with chemotherapy in first-line pancreatic cancer patients Initial data from at least one additional arm of the Phase 2a portion of the Company’s Phase 1/2a trial is expected by year-end CAMBRIDGE, Mass., Oct. 15, 2024 (GLOBE NEWSWIRE) -- Immuneering Corporation (Nasdaq: IMRX), a...Read more
RAMAT GAN, Israel, Oct. 09, 2024 (GLOBE NEWSWIRE) -- Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address oncological and inflammatory diseases, today announced the Company’s oncology drug candidate, Namodenoson, has been granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the indication of pancreatic cancer,...Read more
BOSTON / Sep 30, 2024 / Business Wire / Astria Therapeutics, Inc. (Nasdaq:ATXS), a biopharmaceutical company focused on developing life-changing therapies for allergic and immunologic diseases, today announced that navenibart (STAR-0215) has been granted Orphan Drug Designation for the treatment of hereditary angioedema (HAE) by the U.S. Food and Drug Administration (FDA). Navenibart is a monoclonal antibody inhibitor of plasma kallikrein...Read more
LEXINGTON, Mass. and AMSTERDAM, Sept. 23, 2024 (GLOBE NEWSWIRE) -- uniQure N.V. (NASDAQ: QURE), a leading gene therapy company advancing transformative therapies for patients with severe medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to AMT-191, uniQure’s investigational gene therapy for the treatment of Fabry disease, a rare, inherited genetic disease. In August 2024,...Read more
U.S. FDA Orphan Drug exclusivity provides seven years of market exclusivity for YORVIPATH in the United States for the treatment of hypoparathyroidism in adults COPENHAGEN, Denmark, Sept. 11, 2024 (GLOBE NEWSWIRE) -- Ascendis Pharma A/S (Nasdaq: ASND) today announced that the United States Food & Drug Administration (FDA) has granted Orphan Drug exclusivity to YORVIPATH® (palopegteriparatide, developed as TransCon PTH),...Read more
Elraglusib is a Class-Leading GSK-3β Inhibitor with a Novel, Multimodal Mechanism of Action in Multiple Refractory Cancer Trials Orphan Drug Designation in Soft Tissue Sarcomas Expands Company’s Potential to Address Cancers with High Unmet Medical Need CHICAGO and FORT WORTH, Texas, Sept. 11, 2024 (GLOBE NEWSWIRE) -- Actuate Therapeutics, Inc. (NASDAQ: ACTU) (“Actuate” or the “Company”), a clinical-stage biopharmaceutical company,...Read more
CAMBRIDGE, Mass., Sept. 11, 2024 (GLOBE NEWSWIRE) -- Agios Pharmaceuticals, Inc. (Nasdaq: AGIO), a leader in cellular metabolism and PK activation pioneering therapies for rare diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to the company’s novel pyruvate kinase (PK) activator tebapivat (AG-946) for the treatment of myelodysplastic syndromes (MDS). “Receiving orphan drug...Read more
BASKING RIDGE, N.J., Sept. 05, 2024 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced that the U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug Designation to certepetide for the treatment of...Read more
FORT LEE, N.J., Aug. 29, 2024 (GLOBE NEWSWIRE) -- Nuvectis Pharma, Inc. (NASDAQ: NVCT) (“Nuvectis” or the “Company”), a clinical-stage biopharmaceutical company focused on the development of innovative precision medicines for the treatment of serious conditions of unmet medical need in oncology, today announced that NXP800 was granted Orphan Drug Designation by the United States Food and Drug Administration (“FDA”) for the treatment of...Read more
Neuroblastoma is rare but is the most common infancy malignancy with a median age of diagnosis of 17 months. In the U.S., it accounts for up to around 10% of all childhood cancer cases and 15% of pediatric cancer-related deaths[1],[2] Orphan Drug designation provides for seven-years' marketing exclusivity should opaganib be approved in neuroblastoma and may confer additional benefits such as accelerated development and review times,...Read more
NEW YORK, Aug. 01, 2024 (GLOBE NEWSWIRE) -- Cellectis (the “Company”) (Euronext Growth: ALCLS - NASDAQ: CLLS), a clinical-stage biotechnology company using its pioneering gene-editing platform to develop life-saving cell and gene therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to Cellectis’ CLLS52 (alemtuzumab), an Investigational Medicinal Product (IMP) used as...Read more
Expect to report updated data from the ongoing RAMP 205 trial in first-line metastatic pancreatic cancer in Q1 2025 BOSTON / Jul 29, 2024 / Business Wire / Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to avutometinib, a RAF/MEK clamp, in combination with...Read more
PETACH TIKVA, Israel, July 11, 2024 (GLOBE NEWSWIRE) -- Can-Fite BioPharma Ltd. (NYSE American: CANF) (TASE: CANF), a biotechnology company advancing a pipeline of proprietary small molecule drugs that address oncological and inflammatory diseases, today announced that it has submitted an application to the U.S. Food and Drug Administration (FDA) for Orphan Drug Designation for its drug candidate Namodenoson in the treatment...Read more
With the tafenoquine for acute babesiosis orphan drug designation, 60 Degrees Pharmaceuticals now qualifies for certain incentives, including market exclusivity, tax credits, and exemption from certain FDA filing fees. 60 Degrees Pharmaceuticals recently announced it has entered into an agreement with Tufts Medical Center in Boston to conduct the world’s first clinical trial evaluating the efficacy and...Read more
AUSTIN, TX and DURHAM, NC, June 10, 2024 (GLOBE NEWSWIRE) -- Shattuck Labs, Inc. (Shattuck) (Nasdaq: STTK), a clinical-stage biotechnology company pioneering the development of bifunctional fusion proteins as a new class of biologic medicine for the treatment of patients with cancer and autoimmune disease, today announced that the U.S. FDA has granted orphan drug designation (ODD) to lead clinical candidate SL-172154 for the treatment of...Read more
ChromaDex plans to conduct additional studies on NRC in anticipation of filing for an Investigational New Drug Application (IND) for the treatment of AT LOS ANGELES / Jun 07, 2024 / Business Wire / ChromaDex Corp. (NASDAQ:CDXC), the global authority on nicotinamide adenine dinucleotide (NAD+) and healthy aging research, today announced that the U.S. Food & Drug Administration (FDA) granted Orphan Drug Designation (ODD) and Rare...Read more
FDA Orphan Designation provides CAN-3110 certain developmental financial incentives, with potential for up to 7 years of marketing exclusivity in the United States, if approved CAN-3110 phase 1b data on the feasibility and safety of multiple doses of CAN-3110 will be featured in poster presentation at 2024 ASCO Annual Meeting NEEDHAM, Mass., May 30, 2024 (GLOBE NEWSWIRE) -- Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq:...Read more
NEW YORK, May 13, 2024 (GLOBE NEWSWIRE) -- Tiziana Life Sciences, Ltd. (Nasdaq: TLSA) (“Tiziana” or the “Company”), a biotechnology company developing breakthrough immunomodulation therapies via novel routes of drug delivery, today announced it has submitted an FDA request to obtain Orphan Drug Designation for intranasal foralumab for the treatment of non-active secondary progressive Multiple Sclerosis (na-SPMS). This request would make...Read more
Provides MarVax™ Heat Stable Vaccine Seven Years of U.S. Market Exclusivity Upon FDA Approval PRINCETON, N.J., April 15, 2024 /PRNewswire/ -- Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that the Office of Orphan Products Development of the United...Read more
NEEDHAM, Mass., April 11, 2024 (GLOBE NEWSWIRE) -- Candel Therapeutics, Inc. (Candel or the Company) (Nasdaq: CADL), a clinical stage biopharmaceutical company focused on developing multimodal biological immunotherapies to help patients fight cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to CAN-2409, Candel’s most advanced multimodal biological immunotherapy candidate, for the...Read more
Provides SuVax™ Heat Stable Vaccine Seven Years of U.S. Market Exclusivity Upon FDA Approval PRINCETON, N.J., April 11, 2024 /PRNewswire/ -- Soligenix, Inc. (Nasdaq: SNGX) (Soligenix or the Company), a late-stage biopharmaceutical company focused on developing and commercializing products to treat rare diseases where there is an unmet medical need, announced today that the Office of Orphan Products Development of the United...Read more
FDA designation provides potentially seven years of market exclusivity after approval and expanded partnering opportunities for tecarfarin PONTE VEDRA, Fla., April 9, 2024 /PRNewswire/ -- Cadrenal Therapeutics, Inc. (Nasdaq: CVKD), a biopharmaceutical company developing tecarfarin, a late-stage novel oral and reversible anticoagulant (blood thinner) designed to prevent heart attacks, strokes, and deaths due to blood clots...Read more
BASKING RIDGE, N.J., April 09, 2024 (GLOBE NEWSWIRE) -- Lisata Therapeutics, Inc. (Nasdaq: LSTA) (“Lisata” or the “Company”), a clinical-stage pharmaceutical company developing innovative therapies for the treatment of advanced solid tumors and other serious diseases, today announced that the U.S. Food and Drug Administration (the “FDA”) has granted Orphan Drug Designation (“ODD”) to LSTA1, the Company’s lead product candidate,...Read more
FLORHAM PARK, N.J., March 20, 2024 (GLOBE NEWSWIRE) -- Celularity Inc. (NASDAQ: CELU) (“Celularity”), a regenerative medicine company developing placental-derived allogeneic cell therapies and advanced biomaterial products, announced today that it has submitted a request to the U.S. Food and Drug Administration (FDA) for orphan drug designation for its off-the-shelf, placental-derived cell therapy, PDA-002, for treating...Read more
PHILADELPHIA, March 20, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, for the...Read more
Data Describing Activity of P-BCMA-ALLO1 in BCMA-Experienced Patients to be Presented at Upcoming AACR Meeting SAN DIEGO, March 13, 2024 /PRNewswire/ -- Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene therapy company advancing a new class of treatments for patients with cancer and rare diseases, today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for the...Read more
LYT-200 is being advanced in hematological malignancies such as acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (MDS), and locally advanced/metastatic solid tumors, including head and neck cancers Phase 1b clinical trial evaluating LYT-200 in relapsed/refractory AML and MDS patients is ongoing BOSTON / Mar 13, 2024 / Business Wire / PureTech Health plc (Nasdaq: PRTC, LSE: PRTC) ("PureTech" or the "Company"), a...Read more
BOSTON, March 13, 2024 (GLOBE NEWSWIRE) -- PepGen Inc. (Nasdaq: PEPG), a clinical-stage biotechnology company advancing the next generation of oligonucleotide therapies with the goal of transforming the treatment of severe neuromuscular and neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) granted both orphan drug and rare pediatric disease designations for PGN-EDO51, an investigational therapeutic...Read more
FOSTER CITY, Calif., March 11, 2024 (GLOBE NEWSWIRE) -- Terns Pharmaceuticals, Inc. (“Terns” or the “Company”) (Nasdaq: TERN), a clinical-stage biopharmaceutical company developing a portfolio of small-molecule product candidates to address serious diseases, including oncology and obesity, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for TERN-701 for the treatment of chronic myeloid...Read more
PHOENIX / Mar 06, 2024 / Business Wire / Creative Medical Technology Holdings, Inc. (NASDAQ: CELZ), a leader in the field of regenerative medicine with a focus on developing novel therapies in immunotherapy, endocrinology, urology, gynecology, and orthopedics, is pleased to announce the successful receipt of Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for CELZ-101, ImmCelzTM. This cutting-edge therapy is aimed...Read more
STOCKHOLM, March 6, 2024 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ('Calliditas') today announced that the FDA has granted an orphan drug exclusivity period of seven years for TARPEYO®, expiring in December 2030 based on when the company obtained full approval with a new indication for this drug product. Following full approval in December 2023, TARPEYO® (budesonide) is indicated 'to...Read more
Recurrent low-grade serous ovarian cancer is a rare cancer with no FDA-approved treatments Ongoing Phase 3 RAMP 301 trial is evaluating avutometinib and defactinib in recurrent low-grade serous ovarian cancer On track to submit rolling NDA for Accelerated Approval in H1 2024 BOSTON / Mar 05, 2024 / Business Wire / Verastem Oncology (Nasdaq: VSTM), a biopharmaceutical company committed to advancing new medicines for patients, today...Read more
LOS ALTOS, Calif., March 04, 2024 (GLOBE NEWSWIRE) -- Unicycive Therapeutics, Inc. (Nasdaq: UNCY), a clinical-stage biotechnology company developing therapies for patients with kidney disease (the “Company or “Unicycive”), today announced that the U.S. Food and Drug Administration has granted orphan drug designation (ODD) to UNI-494 for the prevention of Delayed Graft Function (DGF) in kidney transplant patients. UNI-494 is a...Read more
PLYMOUTH MEETING, Pa., Feb. 20, 2024 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to pitolisant for the treatment of Prader-Willi syndrome (PWS). "The decision to grant Orphan Drug designation to pitolisant indicates that it could be a promising treatment option for people living with Prader-Willi syndrome," said...Read more
Designation Based on Pre-Clinical Data and Initial Clinical Data from the Company's MAvERIC-Pilot Phase II Study Toronto, Ontario--(Newsfile Corp. - February 15, 2024) - Cardiol Therapeutics Inc. (NASDAQ: CRDL) (TSX: CRDL) ("Cardiol" or the "Company"), a clinical-stage life sciences company focused on the research and clinical development of anti-inflammatory and anti-fibrotic therapies for the treatment of heart disease, announces...Read more
CARLSBAD, Calif., Feb. 15, 2024 /PRNewswire/ -- Ionis Pharmaceuticals, Inc. (Nasdaq: IONS) announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to the investigational medicine olezarsen for the treatment of familial chylomicronemia syndrome (FCS), a rare, genetic disease characterized by extremely elevated triglyceride levels and recurrent acute pancreatitis (AP). The FDA...Read more
NEW YORK, Feb. 14, 2024 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that the United States Food and Drug Administration (FDA) has granted its allogeneic cell therapy Revascor® (rexlemestrocel-L) an Orphan-Drug Designation (ODD) following submission of results from the randomized controlled trial in children with hypoplastic left...Read more
Cholera is an acute diarrheal illness caused by infection of the intestine with the bacterium Vibrio cholerae Crofelemer previously granted Orphan Drug Designation by the FDA and the EMA for both short bowel syndrome and microvillus inclusion disease SAN FRANCISCO, CA / ACCESSWIRE / February 12, 2024 / Jaguar Health (NASDAQ:JAGX) today announced that Jaguar family company Napo Pharmaceuticals (Napo) has submitted an Orphan Drug...Read more
WATERTOWN, Mass., Feb. 09, 2024 (GLOBE NEWSWIRE) -- Disc Medicine, Inc. (NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the discovery, development, and commercialization of novel treatments for patients suffering from serious hematologic diseases, today announced that the United States Food and Drug Administration (FDA) has granted Orphan Drug Designation to DISC-3405 for the treatment of patients with polycythemia...Read more
BURLINGAME, Calif., Feb. 08, 2024 (GLOBE NEWSWIRE) -- Corvus Pharmaceuticals, Inc. (NASDAQ: CRVS), a clinical-stage biopharmaceutical company, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for soquelitinib for the treatment of T cell lymphoma. Soquelitinib, the Company’s lead ITK inhibitor candidate, is expected to advance into a Phase 3 registrational clinical trial in patients with...Read more
PHILADELPHIA, Feb. 01, 2024 (GLOBE NEWSWIRE) -- Cabaletta Bio, Inc. (Nasdaq: CABA), a clinical-stage biotechnology company focused on developing and launching the first curative targeted cell therapies for patients with autoimmune diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to CABA-201, a 4-1BB-containing fully human CD19-CAR T cell investigational therapy, for the...Read more
Patient dosing expected to begin mid-to-late Q1 2024 CHARLESTOWN, Mass., Jan. 16, 2024 (GLOBE NEWSWIRE) -- Solid Biosciences Inc. (Nasdaq: SLDB), a life sciences company developing precision genetic medicines for neuromuscular and cardiac diseases, today announced that it has been granted orphan drug designation (ODD) from the U.S. Food and Drug Administration (FDA) for SGT-003, the company’s next-generation Duchenne muscular...Read more
CAMBRIDGE, Mass. and NESS ZIONA, Israel, Jan. 04, 2024 (GLOBE NEWSWIRE) -- BiomX Inc. (NYSE American: PHGE) (“BiomX”), a clinical-stage company advancing novel natural and engineered phage therapies that target specific pathogenic bacteria, today announced that its phage cocktail, BX004, has been granted Orphan Drug Designation (“ODD”) by the United States Food and Drug Administration (“FDA”), for the treatment of chronic...Read more
SLS009 Demonstrated Promising Efficacy in Phase 1 Study with 36.4% Clinical Response (ORR) in r/r Peripheral T-cell Lymphomas (PTCL); ORR in r/r PTCL Patients with Standard of Care is 25.8% One Patient with Complete Metabolic Response Continuing Treatment for over 62 weeks and another patient with Complete Response by CT Continuing Treatment for over 24 weeks - Phase 1b/2 Study in PTCL Ongoing with top line data expected in 1H...Read more
First orphan drug designation for Nana-val granted in EBV+ solid tumors Seventh orphan drug designation for Nana-val globally; fifth granted by the FDA SAN DIEGO, Dec. 12, 2023 (GLOBE NEWSWIRE) -- Viracta Therapeutics, Inc. (Nasdaq: VIRX), a clinical-stage precision oncology company focused on the treatment and prevention of virus-associated cancers that impact patients worldwide, today announced that the U.S. Food and Drug...Read more
BOULDER, Colo. / Nov 30, 2023 / Business Wire / Edgewise Therapeutics, Inc., (Nasdaq: EWTX), a leading muscle disease biopharmaceutical company, today announced that the U.S. Food & Drug Administration (FDA) has granted EDG-5506 Orphan Drug Designation (ODD) for the treatment of Duchenne muscular dystrophy (Duchenne) and Becker muscular dystrophy (Becker) and Rare Pediatric Disease Designation (RPDD) for the treatment of Duchenne....Read more
The Orphan Drug Designation strengthens LP-284’s clinical development path and can provide for additional market exclusivity and commercial protection. Lantern has initiated a first-in-human Phase 1 trial (NCT06132503) for LP-284 in B-cell non-Hodgkin’s lymphomas (NHL), including high-grade B-cell lymphoma (HGBL) and mantle cell lymphoma (MCL). This Orphan Drug Designation marks the second such designation for drug-candidate LP-284 this...Read more
First cystic fibrosis patient in Phase 1b study successfully completed two administrations of ARCT-032 On track to share interim Phase 1b data in H1 2024 SAN DIEGO / Nov 27, 2023 / Business Wire / Arcturus Therapeutics Holdings Inc. (the “Company”, “Arcturus”, Nasdaq: ARCT), a global late-stage clinical messenger RNA medicines company focused on the development of infectious disease vaccines and opportunities within liver and...Read more
LONDON, Ontario and WINDHAM COUNTY, Conn., Nov. 27, 2023 (GLOBE NEWSWIRE) -- Sernova Corp. (TSX:SVA) (OTCQB:SEOVF) (FSE/XETRA:PSH), a clinical-stage company and leader in cell therapeutics, today announced the U.S. Food and Drug Administration (FDA) has granted both Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) for the company’s Hemophilia A program. The FDA grants orphan designation, also referred to as...Read more
NEW YORK, Nov. 26, 2023 (GLOBE NEWSWIRE) -- Mesoblast Limited (Nasdaq:MESO; ASX:MSB), global leader in allogeneic cellular medicines for inflammatory diseases, today announced that it has filed for orphan drug designation (ODD) and rare pediatric disease designation (RPDD) with the United States Food and Drug Administration (FDA) for its allogeneic cell therapy Revascor® (rexlemestrocel-L) in the treatment of the congenital heart disease...Read more
WALTHAM, Mass., Nov. 15, 2023 (GLOBE NEWSWIRE) -- Ardelyx, Inc. (Nasdaq: ARDX), a biopharmaceutical company founded with a mission to discover, develop and commercialize innovative, first-in-class medicines that meet significant unmet medical needs, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to XPHOZAH® (tenapanor) for the treatment of pediatric hyperphosphatemia. XPHOZAH is a...Read more
Third orphan drug designation (ODD) granted to THIO by the FDA; drug also holds ODDs for hepatocellular carcinoma and small cell lung cancer Benefits include 7 years of U.S. market exclusivity after drug approval and tax credits for qualified clinical testing Expected glioblastoma market growth from $2.2 billion to $3.2 billion globally in the next three years CHICAGO / Nov 10, 2023 / Business Wire / MAIA Biotechnology, Inc., (NYSE...Read more
Lipella Pharmaceuticals' LP-310 clinical stage asset is formulated for delivering active agents to the oral cavity for diseases of the mouth including oral lichen planus and oral graft-versus-host disease PITTSBURGH, Nov. 10, 2023 /PRNewswire/ -- Lipella Pharmaceuticals Inc. (Nasdaq: LIPO), a clinical stage pharmaceutical company addressing serious diseases with significant unmet need based in Pittsburgh, PA, announced...Read more
SOUTH SAN FRANCISCO, Calif., Nov. 09, 2023 (GLOBE NEWSWIRE) -- Lyell Immunopharma, Inc. (Nasdaq: LYEL), a clinical‑stage T-cell reprogramming company advancing a diverse pipeline of cell therapies for patients with solid tumors, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to LYL845, an investigational tumor infiltrating lymphocyte (TIL) product candidate for the treatment of patients...Read more
AUSTIN, Texas, Nov. 03, 2023 (GLOBE NEWSWIRE) -- Plus Therapeutics, Inc. (Nasdaq: PSTV) (the “Company”), a clinical-stage pharmaceutical company developing targeted radiotherapeutics with advanced platform technologies for central nervous system cancers, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to rhenium (186Re) obisbemeda for the treatment of breast cancer with...Read more
TORONTO and HAIFA, Israel, Oct. 30, 2023 (GLOBE NEWSWIRE) -- NurExone Biologic Inc. (TSXV: NRX) (FSE: J90) (NRX.V) (the “Company” or “NurExone”), a pioneering biotechnology company, is thrilled to announce that the U.S. Food and Drug Administration (FDA) has granted Orphan-Drug Designation (ODD) for its ExoPTEN therapy, recognizing the potential of this groundbreaking regenerative therapy for acute spinal cord injury, a condition where...Read more
CAMBRIDGE, Mass. / Oct 18, 2023 / Business Wire / Sage Therapeutics, Inc. (Nasdaq: SAGE), a biopharmaceutical company leading the way to create a world with better brain health, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to SAGE-718 for the treatment of Huntington’s disease (HD). SAGE-718 is in development as a potential oral therapy for cognitive disorders associated with NMDA receptor...Read more
Terns Pharmaceuticals is a clinical-stage biopharmaceutical company developing a portfolio of small-molecule product candidates to address serious diseases, including oncology and obesity. Terns’ pipeline contains three clinical stage development programs including GLP-1 receptor...
CLICK TO LEARN MORE