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
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...
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