Hematopoietics

Phase 3 Trial of Incyte’s Jakafi Met Primary and Both Secondary Endpoints in Patients with Chronic Graft-Versus-Host Disease

WILMINGTON, Del.–(BUSINESS WIRE)–Incyte (Nasdaq:INCY) today announced that the Phase 3 REACH3 study evaluating ruxolitinib (Jakafi®) in patients with moderate or severe steroid-refractory or steroid dependent chronic graft-versus-host disease (GVHD) met its primary endpoint of superior overall response rate (ORR) at Week 24 compared to best available therapy (BAT). In addition, …

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FDA Grants Orchard Therapeutics’ OTL-203 Orphan Drug and Rare Pediatric Disease Designations for Treatment of MPS-I

BOSTON and LONDON, July 20, 2020 (GLOBE NEWSWIRE) — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company has received both orphan drug designation and rare pediatric disease designation from the U.S Food and Drug Administration (FDA) for OTL-203, an ex vivo autologous hematopoietic stem cell (HSC) …

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Orchard Therapeutics Announces Global License Agreements for Stable Cell Line Technology from GSK

BOSTON and LONDON, July 15, 2020 (GLOBE NEWSWIRE) — Orchard Therapeutics (Nasdaq: ORTX), a global gene therapy leader, today announced that the company has entered into two worldwide royalty-bearing license agreements with GlaxoSmithKline plc (GSK) (LSE/NYSE: GSK) for use of their proprietary lentiviral stable cell line technology (LV-SCLT) for Orchard’s …

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FDA Approves Inqovi Tablets, Oral Hypomethylating Agent Therapy for Intermediate and High-Risk MDS and CMML

PLEASANTON, Calif. & PRINCETON, N.J. & TOKYO–(BUSINESS WIRE)–Astex Pharmaceuticals, Inc.; Taiho Oncology, Inc.; and Otsuka Pharmaceutical Co., Ltd. today announce that the U.S. Food and Drug Administration (FDA) and Health Canada have approved INQOVI® (decitabine and cedazuridine) tablets. The three companies are all part of the Otsuka group of companies. INQOVI …

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Kadmon Announces Positive Topline Results of Pivotal Trial of Belumosudil in Chronic Graft-Versus-Host Disease

NEW YORK, NY / ACCESSWIRE / May 21, 2020 / Kadmon Holdings, Inc. (NYSE:KDMN) today announced positive topline results from the primary analysis of ROCKstar (KD025-213), the pivotal trial evaluating belumosudil (KD025) in patients with chronic graft-versus-host disease (cGVHD) who have received at least two prior lines of systemic therapy. Belumosudil (KD025) achieved clinically meaningful …

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Gamida Cell Announces Positive Topline Data from Phase 3 Clinical Study of Omidubicel in Patients with High-Risk Hematologic Malignancies

BOSTON–(BUSINESS WIRE)–Gamida Cell Ltd. (Nasdaq: GMDA), an advanced cell therapy company committed to finding cures for blood cancers and serious blood diseases, today announced positive topline results from its Phase 3 clinical study evaluating the safety and efficacy of omidubicel, an investigational advanced cell therapy in development as a potential life-saving …

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New Data Demonstrates Andexxa Can Provide a Net Cost Reduction for the Treatment of Intracranial Hemorrhage Associated with Oral Factor Xa Inhibitors

SOUTH SAN FRANCISCO, Calif., March 5, 2020 /PRNewswire/ — Portola Pharmaceuticals, Inc.® (Nasdaq: PTLA) today announced the presentation of new data demonstrating that using Andexxa® [coagulation factor Xa (recombinant), inactivated-zhzo] to treat patients with intracranial hemorrhage (ICH) associated with the oral Factor Xa inhibitors apixaban or rivaroxaban is projected to …

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Astex Announces FDA Accepts for Priority Review its NDA for Oral C-DEC for the Treatment of MDS and CMML

PLEASANTON, Calif.–(BUSINESS WIRE)–Astex Pharmaceuticals, Inc., a wholly owned subsidiary of Otsuka Pharmaceutical Co. Ltd., based in Japan, today announced that the U.S. FDA has accepted for Priority Review its NDA for oral C-DEC (cedazuridine and decitabine) as a treatment for adults with previously untreated intermediate- and high-risk MDS including CMML. …

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FDA Grants Fast Track Designation to Imago’s Bomedemstat for Treatment of Essential Thrombocythemia

SAN FRANCISCO–(BUSINESS WIRE)–Imago BioSciences, Inc., a clinical-stage biotechnology company developing innovative treatments for myeloid diseases, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation for the development of bomedemstat (IMG-7289) for the treatment of essential thrombocythemia (ET), a bone marrow disease associated with high …

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Akari Therapeutics Initiates Pivotal Phase 3 Trial of Nomacopan in Pediatric Hematopoietic Stem Cell Transplant-Related Thrombotic Microangiopathy

NEW YORK and LONDON, Dec. 23, 2019 (GLOBE NEWSWIRE) — Akari Therapeutics, Plc (Nasdaq:AKTX), a biopharmaceutical company focused on innovative therapeutics to treat orphan autoimmune and inflammatory diseases where the complement and/or leukotriene systems are implicated, announces that a U.S. Food and Drug Administration (FDA) investigational new drug application (IND) …

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