Orphan drugs Press Release

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Fri, 03/28/2025 - 17:57 FDA Approves Dual-Action Drug for Patients with Previously Treated Advanced Neuroendocrine Tumors, Backed by Dana-Farber Research
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Dana Farber
Fri, 03/28/2025 - 17:35 FDA Approves Qfitlia (fitusiran), the First siRNA (RNAi Therapeutic) for the Treatment of Hemophilia A or B
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Alnylam
Thu, 03/27/2025 - 18:04 Regulus Therapeutics Announces Successful Completion of its Phase 1b Multiple-Ascending Dose (MAD) Clinical Trial of Farabursen (RGLS8429) for the Treatment of Autosomal Dominant Polycystic Kidney Disease (ADPKD)
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Regulus Therapeutics
Thu, 03/27/2025 - 04:51 Chugai Obtains Regulatory Approval for First Tablet for SMA, Evrysdi
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Chugai
Thu, 03/20/2025 - 19:04 Caris Life Sciences Publishes Study Featuring the Largest Real-World Cohort of Tissue-Agnostic Indications Revealing Over 20 Percent of Patients Are Eligible for Pan-Cancer Therapies
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Caris Life Sciences
Tue, 03/18/2025 - 21:51 Chugai Launches LUNSUMIO for Intravenous Infusion, a Bispecific Antibody for Relapsed or Refractory Follicular Lymphoma in Japan
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Chugai
Wed, 03/05/2025 - 07:02 Alnylam Announces Retirement of Longtime Board Member, Dr. Phillip A. Sharp, Company Co-Founder and Industry Luminary
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Alnylam
Tue, 03/04/2025 - 01:45 Certain Genetic Alterations May Contribute to the Primary Resistance of Colorectal and Pancreatic Cancers to KRAS G12C Inhibitors
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American Associ...
Tue, 02/25/2025 - 17:51 Trevi Therapeutics Completes Enrollment for Phase 2b CORAL Trial of Haduvio in Idiopathic Pulmonary Fibrosis Patients with Chronic Cough
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Trevi Therapeutics
Thu, 02/20/2025 - 05:57 Chugai Obtains Regulatory Approval for Tecentriq for the Additional Indication of Alveolar Soft Part Sarcoma, an Ultra-rare Disease
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Chugai
Mon, 02/17/2025 - 18:15 Radiation Plus Combination Immunotherapy May Help Preserve Bladder in Some Patients With Muscle-invasive Bladder Cancer
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American Associ...
Mon, 02/17/2025 - 15:58 Some Kidney Cancer Patients Can Stop Combination Treatment After Two Years, Fox Chase Cancer Center Study Finds
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Fox Chase Cance...
Tue, 02/04/2025 - 15:41 Preoperative immunotherapy could enhance breast cancer cure rates
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UTSouthwestern
Mon, 01/27/2025 - 11:45 Ferring Highlights Commitment to C. Diff Infection Community on the Second Anniversary of the Launch of REBYOTA (fecal microbiota, live jslm)
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Ferring Pharmac...
Fri, 01/24/2025 - 17:09 Fox Chase Cancer Center Researchers Present Data on Use of Checkpoint Inhibitors and Test Measuring Their Benefit to Patients
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Fox Chase Cance...
Sun, 01/19/2025 - 23:21 Phase 2 LITESPARK-003 results suggest continued study of novel first-line combo for advanced kidney cancer
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Dana Farber
Fri, 01/17/2025 - 19:17 U.S. Food and Drug Administration Approves FoundationOneCDx as a Companion Diagnostic for OJEMDA (tovorafenib) to Treat the Most Common Form of Childhood Brain Tumor in Pediatric Patients
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Foundation Medicine
Fri, 01/17/2025 - 05:27 Chugai Wins the MHLW Ministers Award at the Seventh Japan Medical Research and Development Grand Prize for the Discovery of an ALK-Positive Lung Cancer Treatment Alecensa
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Chugai
Sat, 12/14/2024 - 01:55 Roswell Park Team Identifies Factors That Boost Effectiveness of Immunotherapy in Recurrent Ovarian Cancer
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Roswell Park Ca...
Thu, 12/12/2024 - 01:11 Could Venetoclax Improve Outcomes of CAR T for Patients with Resistant B-Cell Lymphomas? New Insights at ASH
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Roswell Park Ca...
Wed, 11/20/2024 - 15:15 Roswell Park Clinical Trial Points Toward Promising New Therapy for Most Aggressive Type of Breast Cancer
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Roswell Park Ca...
Wed, 08/28/2024 - 05:10 Alecensa Approved in Japan for the Additional Indication of Adjuvant Treatment for People with ALK-Positive Early-Stage Non-Small Cell Lung Cancer
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Chugai
Wed, 08/07/2024 - 13:27 Alnylam to Present Detailed Results from the HELIOS-B Phase 3 Study of Vutrisiran in Patients with ATTR Amyloidosis with Cardiomyopathy at the European Society of Cardiology Congress 2024
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Alnylam
Mon, 08/05/2024 - 21:27 India, USA, Europe, Russia, August 05, 2024: Global Pharma major Lupin Limited (Lupin) today announced the successful completion of a global Phase 3 clinical study of LUBT010, its biosimilar candidate to Lucentis. The study has achieved its primary endpoi
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Lupin
Sat, 08/03/2024 - 23:07 FDA Approves First Gene Therapy to Treat Adults with Metastatic Synovial Sarcoma
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FDA