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
REHOVOT, Israel and MONMOUTH JUNCTION, N.J., Nov. 06, 2023 (GLOBE NEWSWIRE) -- 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 to AL102, a Gamma Secretase Inhibitor (GSI), for the treatment of desmoid tumors (DT). AL102 is an investigational small molecule GSI, currently being evaluated in the Phase 3...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
NEW YORK, Oct. 10, 2023 (GLOBE NEWSWIRE) -- SELLAS Life Sciences Group, Inc. (NASDAQ: SLS) (“SELLAS’’ or the “Company”), a late-stage clinical biopharmaceutical company focused on the development of novel therapies for a broad range of cancer indications, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) for SLS009, a novel and highly selective CDK9 inhibitor, for the treatment of...Read more
BOSTON and ATLANTA, Oct. 04, 2023 (GLOBE NEWSWIRE) -- Inhibikase Therapeutics, Inc. (Nasdaq: IKT) (Inhibikase or Company), a clinical-stage pharmaceutical company developing protein kinase inhibitor therapeutics to modify the course of Parkinson's disease ("PD"), Parkinson's-related disorders and other diseases of the Abelson Tyrosine Kinases, today announced that risvodetinib (IkT-148009) has been granted Orphan Drug Designation by the...Read more
MALVERN, Pa., Sept. 28, 2023 (GLOBE NEWSWIRE) -- Baudax Bio, Inc. (the “Company” or “Baudax Bio”) (NASDAQ: BXRX), a biotechnology company focused on developing T cell receptor (“TCR”) therapies utilizing human regulatory T cells (“Tregs”), as well as a portfolio of clinical stage Neuromuscular Blocking Agents (“NMBs”) and an associated reversal agent, today announced that U.S. Food and Drug Administration (FDA) has granted orphan drug...Read more
BDC-1001 is a Boltbody™ Immune-Stimulating Antibody Conjugate (ISAC) in Phase 2 development for HER2-positive breast, colorectal, endometrial, and gastroesophageal cancers The FDA granted BDC-1001 Orphan Drug Designation for the treatment of gastric cancer, including gastroesophageal junction cancer REDWOOD CITY, Calif., Sept. 28, 2023 (GLOBE NEWSWIRE) -- Bolt Biotherapeutics (Nasdaq: BOLT), a clinical-stage biopharmaceutical...Read more
STOCKHOLM, Sept. 27, 2023 /PRNewswire/ -- Calliditas Therapeutics AB (Nasdaq: CALT) (Nasdaq Stockholm: CALTX) ("Calliditas") today announced that the US Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to the company for the treatment of Alport syndrome with setanaxib. Based on supportive pre-clinical work, Calliditas plans to initiate a randomized, placebo-controlled phase 2 clinical study in Alport syndrome...Read more
LOS ANGELES / Sep 21, 2023 / Business Wire / Puma Biotechnology, Inc. (NASDAQ: PBYI), a biopharmaceutical company, announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation to alisertib, a selective, small-molecule, orally administered inhibitor of aurora kinase A, for the treatment of patients with small cell lung cancer (SCLC). SCLC is an aggressive form of lung cancer with a poor prognosis, and with...Read more
FDA Orphan Drug Designation (“ODD”) qualifies one-time treatment NXC-201 for: 7 years of U.S. market exclusivity after approval Tax credits for qualified clinical testing Waiver of the Prescription Drug User Fee (currently at almost $3 million for a new drug) The Amyloidosis market was $3.6 billion in 2017, expected to reach $6 billion in 2025, according to Grand View Research LOS ANGELES, Sept. 21, 2023 (GLOBE NEWSWIRE) -- Immix...Read more
On Track to Report Initial Data from the Global, Multiple Ascending Dose Phase 1/2 ACHIEVE Clinical Trial in the Second Half of 2023 WALTHAM, Mass., Sept. 20, 2023 (GLOBE NEWSWIRE) -- Dyne Therapeutics, Inc. (Nasdaq: DYN), a clinical-stage muscle disease company focused on advancing innovative life-transforming therapeutics for people living with genetically driven diseases, today announced that the U.S. Food and Drug...Read more
VELDONA® positioned as a potential treatment option for over 24,000 HIV-seropositive Americans SAN DIEGO, CA / ACCESSWIRE / September 18, 2023 / Ainos, Inc. (NASDAQ: AIMD, AIMDW) ("Ainos", or the "Company"), a diversified healthcare company focused on the development of AI-powered point-of-care testing, low-dose interferon therapeutics, and synthetic RNA-driven preventative medicine, announced today that the United States Food and...Read more
PLYMOUTH MEETING, Pa., Sept. 7, 2023 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. ("Harmony") (Nasdaq: HRMY), a pharmaceutical company dedicated to developing and commercializing innovative therapies for patients with rare neurological diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to pitolisant for the treatment of idiopathic hypersomnia (IH). The FDA's Orphan...Read more
WESTPORT, Conn., Sept. 7, 2023 /PRNewswire/ -- Intensity Therapeutics, Inc. ("Intensity" or the "Company") (Nasdaq: INTS), a clinical-stage biotechnology company focused on the discovery and development of proprietary, novel immune-based intratumoral cancer therapies designed to kill tumors and increase immune system recognition of cancers, today announced that the US Food and Drug Administration's Office of Orphan Products Development...Read more
KB408 IND filed on August 15, 2023 PITTSBURGH, Sept. 05, 2023 (GLOBE NEWSWIRE) -- Krystal Biotech, Inc. (the “Company”) (NASDAQ: KRYS), a commercial-stage biotechnology company focused on the discovery, development and commercialization of genetic medicines to treat diseases with high unmet medical needs, announced today that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation for KB408 for the treatment of...Read more
FDA Orphan Drug Designation (“ODD”) qualifies NXC-201 for: 7 years of U.S. market exclusivity after approval Tax credits for qualified clinical testing Waiver of the Prescription Drug User Fee (currently at almost $3 million for a new drug) The $13.9 billion Multiple Myeloma market in 2017 is expected to reach $28.7 billion in 2027 according to Wilcock, et al. Nature Reviews LOS ANGELES, Aug. 23, 2023 (GLOBE NEWSWIRE) -- Immix...Read more
Fort Lee, NJ, Aug. 17, 2023 (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
SAN DIEGO, Aug. 15, 2023 /PRNewswire/ -- Avidity Biosciences, Inc. (Nasdaq: RNA), a biopharmaceutical company committed to delivering a new class of RNA therapeutics called Antibody Oligonucleotide Conjugates (AOCs™), today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation to AOC 1044, the company's investigational therapy in development for the treatment of Duchenne muscular dystrophy (DMD) in...Read more
ODD is in addition to Three FDA Fast Track Designations Genprex expects to dose the first patient in the Acclaim-3 clinical trial in the fourth quarter of 2023 AUSTIN, Texas, Aug. 10, 2023 /PRNewswire/ -- Genprex, Inc. ("Genprex" or the "Company") (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced the...Read more
BASKING RIDGE, N.J., Aug. 08, 2023 (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 the U.S. Food and Drug Administration (“FDA”) has granted Orphan Drug Designation to LSTA1, the Company’s lead product candidate, for the treatment...Read more
Company also recently received Rare Pediatric Disease Designation from the FDA for SAT-3153 for the treatment of pediatric patients with Duchenne Toronto, Ontario--(Newsfile Corp. - August 2, 2023) - Satellos Bioscience Inc. (TSXV: MSCL) (OTCQB: MSCLF) ("Satellos" or the "Company"), a drug discovery company developing small molecule therapeutics to regenerate muscle as a new approach to treating muscle diseases and disorders,...Read more
CARLSBAD, Calif., Aug. 1, 2023 /PRNewswire/ -- Tyra Biosciences, Inc. (Nasdaq: TYRA), a clinical-stage biotechnology company focused on developing next-generation precision medicines that target large opportunities in Fibroblast Growth Factor Receptor (FGFR) biology, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to its lead precision medicine program, TYRA-300, for the treatment...Read more
ROCKVILLE, Md., June 27, 2023 (GLOBE NEWSWIRE) -- Theriva Biologics (NYSE American: TOVX), (“Theriva” or the “Company”), a clinical-stage company developing therapeutics designed to treat cancer and related diseases in areas of high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to lead clinical candidate VCN-01, Theriva’s systemic, selective, stroma-degrading oncolytic...Read more
ERAS-801 has now received ODD in addition to FDA Fast Track Designation Initial THUNDERBBOLT-1 Phase 1 monotherapy data in patients with recurrent GBM expected in H2 2023 SAN DIEGO, June 22, 2023 (GLOBE NEWSWIRE) -- Erasca, Inc. (Nasdaq: ERAS), a clinical-stage precision oncology company singularly focused on discovering, developing, and commercializing therapies for patients with RAS/MAPK pathway-driven cancers, today announced the...Read more
WATERTOWN, Mass., June 22, 2023 (GLOBE NEWSWIRE) -- Kymera Therapeutics, Inc. (NASDAQ: KYMR), a clinical-stage biopharmaceutical company advancing targeted protein degradation to deliver novel small molecule protein degrader medicines, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to KT-253 for the treatment of Acute Myeloid Leukemia (AML). KT-253 is a highly potent and selective...Read more
SAN DIEGO, June 22, 2023 (GLOBE NEWSWIRE) -- aTyr Pharma, Inc. (Nasdaq: LIFE), a biotherapeutics company engaged in the discovery and development of first-in-class medicines from its proprietary tRNA synthetase platform, today announced that the European Commission (EC) granted orphan drug designation for the company’s lead therapeutic candidate, efzofitimod, for the treatment of systemic sclerosis (SSc, or scleroderma) based on the...Read more
CRANBURY, N.J. / Jun 08, 2023 / Business Wire / Rocket Pharmaceuticals, Inc. (NASDAQ: RCKT), a leading late-stage biotechnology company advancing an integrated and sustainable pipeline of genetic therapies for rare disorders with high unmet need, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track and Orphan Drug designations to RP-A601, the Company’s gene therapy candidate for the treatment of...Read more
SAN DIEGO, June 07, 2023 (GLOBE NEWSWIRE) -- Capricor Therapeutics (NASDAQ: CAPR), a biotechnology company focused on the development of transformative cell and exosome-based therapeutics for the treatment and prevention of muscular and other select diseases, today announced an upcoming Type-B clinical meeting with the U.S. Food and Drug Administration (FDA), planned in early Q3 2023. During the planned meeting with the FDA, Capricor will...Read more
WASHINGTON, June 1, 2023 /PRNewswire/ -- Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA) today announced the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation for VCA-894A for the treatment of Charcot-Marie-Tooth disease, axonal, type 2S (CMT2S), caused by cryptic splice site variants within IGHMBP2. CMT2S is a rare subtype of Charcot-Marie-Tooth disease (CMT), an inherited peripheral neuropathy for which...Read more
In a groundbreaking development, Galera Therapeutics, Inc. (NASDAQ: GRTX) has achieved a major milestone in the fight against pancreatic cancer. The U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Galera's second product candidate, rucosopasem manganese (rucosopasem), for the treatment of pancreatic cancer. This significant recognition reflects the urgent need for innovative treatment options to combat this...Read more
MALVERN, Pa., May 18, 2023 (GLOBE NEWSWIRE) -- Galera Therapeutics, Inc. (Nasdaq: GRTX), a clinical-stage biopharmaceutical company focused on developing and commercializing a pipeline of novel, proprietary therapeutics that have the potential to transform radiotherapy in cancer, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation to Galera’s second product candidate, rucosopasem manganese...Read more
DUBLIN, May 9, 2023 /PRNewswire/ -- Theravance Biopharma, Inc. ("Theravance Biopharma" or the "Company") (NASDAQ: TBPH) today announced that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) status to ampreloxetine for the treatment of symptomatic neurogenic orthostatic hypotension (nOH) in patients with multiple system atrophy (MSA). MSA is a rare neurodegenerative disorder in which patients suffer...Read more
CAMBRIDGE, Mass., May 09, 2023 (GLOBE NEWSWIRE) -- Synlogic, Inc. (Nasdaq: SYBX), the leading company advancing therapeutics based on synthetic biology, today announced that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation (ODD) to SYNB1934 for the treatment of phenylketonuria (PKU). “We are very pleased that SYNB1934 has been granted another regulatory designation, further validating the need for new...Read more
CAMBRIDGE, Mass., April 27, 2023 (GLOBE NEWSWIRE) -- Editas Medicine, Inc. (Nasdaq: EDIT), a clinical stage genome editing company, today announced that the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to EDIT-301, an investigational, gene editing medicine, for the treatment of sickle cell disease. The FDA previously granted Orphan Drug Designation to EDIT-301 for the treatment of beta thalassemia and Rare...Read more
MALVERN, Pa., April 27, 2023 (GLOBE NEWSWIRE) -- Ocugen, Inc. (Ocugen or the Company) (NASDAQ: OCGN), a biotechnology company focused on discovering, developing, and commercializing novel gene and cell therapies, biologics, and vaccines, today announced that the FDA has granted Orphan Drug Designation for its OCU410ST (AAV5-hRORA), an adeno-associated virus serotype 5 capsid protein containing gene construct encoding human retinoic acid...Read more
C4 Therapeutics is pioneering a new class of small-molecule drugs that selectively destroy disease-causing proteins via degradation using the innate machinery of the cell. This targeted protein degradation approach offers advantages over traditional drugs, including the potential to treat a wider range of diseases...
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