Acute Myeloid Leukemia
Nov 18, 2025
Sarepta Secures FDA Approval of Revised Prescribing Information for ELEVIDYS; Merck to Acquire Cidara Therapeutics; BMS & J&J Halt Milvexian Phase III in ACS After Interim Review; Kura Oncology & Kyowa Kirin Win FDA Green Light for KOMZIFTI in NPM1-Mutated AML; Novartis’ Phase III KLU156 (GanLum) Trial Hits Primary Endpoint
FDA Approves Major Safety and Indication Updates for Sarepta's ELEVIDYS Gene Therapy Sarepta Therapeutics announced that the U.S. Food and Drug Administration (FDA) has approved significant updates to the prescribing information for its Duchenne muscular dystrophy (DMD) gene therapy, ELEVIDYS (delandistrogene mo...
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Oct 28, 2025
Syndax Pharmaceuticals Receives FDA Approval for REVUFORJ; FDA Approves BLENREP for Adults with Relapsed/Refractory Multiple Myeloma; Novartis to Acquire Avidity for $12 Billion; BridgeBio Reports Positive Phase 3 Results for Small Molecule BBP-418 in LGMD2I/R9 FORTIFY Study; Eli Lilly to Acquire Adverum Biotechnologies
Syndax Pharmaceuticals Receives Approval for REVUFORJ from the FDA Syndax Pharmaceuticals announced that the FDA has approved REVUFORJ (revumenib) for the treatment of relapsed or refractory acute myeloid leukemia with a susceptible nucleophosmin 1 (NPM1) mutation in adult and pediatric patients one year and old...
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Oct 07, 2025
Aminex Therapeutics Secures FDA Orphan Drug Designation for AMXT 1501 + DFMO in Neuroblastoma; Eccogene Gets FDA Nod to Begin Phase IIa MOSAIC Trial of ECC4703 + ECC0509 in MASH; Orca Bio’s Orca-T Earns FDA Priority Review for Hematologic Malignancies; Alto Neuroscience Gains FDA Fast Track for ALTO-101 in Cognitive Impairment Linked to Schizophrenia; Taysha Gene Therapies Wins FDA Breakthrough Therapy Status for TSHA-102 in Rett Syndrome
Aminex Therapeutics Secures FDA Orphan Drug Designation for AMXT 1501 + DFMO in Neuroblastoma Aminex Therapeutics, Inc., a clinical-stage biotechnology company developing novel cancer therapies, announced that the FDA has granted Orphan Drug Designation (ODD) to AMXT 1501 in combination with difluoromethylornith...
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Sep 16, 2025
Lion TCR Secures Triple FDA Milestones with IND Clearance for Chronic Hepatitis B; Corstasis Therapeutics Wins FDA Approval for ENBUMYST Nasal Spray to Treat Edema in CHF, Liver, and Kidney Disease; Amneal Gains FDA Approval for Sodium Oxybate Oral Solution in Narcolepsy Patients with Cataplexy and Excessive Daytime Sleepiness; Greenwich LifeSciences’ GLSI-100 Granted FDA Fast Track Designation; Akeso’s Ligufalimab Granted FDA Orphan Drug Designation for Acute Myeloid Leukemia
Lion TCR Secures Triple FDA Milestones for Chronic Hepatitis B with IND Clearance of TCR-T Therapy Lion TCR announced that the FDA has cleared its Investigational New Drug (IND) application for LioCyx-M004, authorizing the initiation of phase Ib/II clinical trials in patients with chronic hepatitis B (CHB). The ...
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Jul 22, 2025
Sarepta Therapeutics Pauses ELEVIDYS Shipments in U.S. After FDA Intervention Over Patient Deaths; ImCheck’s ICT01 Granted FDA Orphan Drug Designation for Acute Myeloid Leukemia; RedHill Biopharma Gets Positive FDA Feedback on RHB-204 for Crohn’s Disease Approval Pathway; FDA Grants Fast Track Designation to ProMIS Neurosciences’ PMN310 for Alzheimer’s Disease; Oruka Therapeutics Receives IND Clearance for Phase IIa Trial of ORKA-001 in Psoriasis
Sarepta Therapeutics Pauses ELEVIDYS Shipments in U.S. After FDA Intervention Over Patient Deaths Sarepta Therapeutics, Inc. has voluntarily and temporarily paused all shipments of ELEVIDYS (delandistrogene moxeparvovec) in the U.S., effective July 22, 2025, following engagement with the FDA on safety labeling u...
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Jun 03, 2025
Beam’s BEAM-302 Earns FDA Orphan Drug Tag for AATD; Incannex’s IHL-42X Moves to Phase III After FDA Protocol OK; Kura and Kyowa Kirin’s Ziftomenib Gets FDA Priority Review in NPM1-Mutant AML; GSK’s Linerixibat NDA Accepted by FDA for PBC-Linked Pruritus; Ascendis’ TransCon CNP Granted FDA Priority Review for Achondroplasia
Beam Therapeutics Secures FDA Orphan Drug Designation for BEAM-302 in AATD Beam Therapeutics Inc. announced that the FDA has granted Orphan Drug Designation to its investigational therapy, BEAM-302, for the treatment of alpha-1 antitrypsin deficiency (AATD). BEAM-302 is a liver-targeting lipid nanoparticle (LNP)...
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Feb 10, 2025
Acute Myeloid Leukemia: The Battle, The Breakthroughs, The Future
Acute Myeloid Leukemia (AML) is a rapidly progressing cancer that originates in the bone marrow—the soft, inner part of bones where blood cells are produced—and swiftly moves into the blood. Globally, the burden of AML has been increasing over the past few decades. The exact cause of AML remains elusive, but severa...
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Feb 07, 2025
10 Game-Changing Acute Myeloid Leukemia Drugs Revolutionizing Treatment
Acute Myeloid Leukemia (AML) is a fast-moving and aggressive cancer that demands timely and effective treatment. With the current acute myeloid leukemia therapies showing limited success for many patients, the focus has shifted to breakthrough AML drugs that are redefining the treatment landscape. From innovative s...
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Dec 30, 2024
6 Emerging Treg Cell-based Therapies Shaping the Future of Immunotherapy
Treg (regulatory T-cell) therapies utilize the immunosuppressive abilities of regulatory T-cells to address various conditions, such as autoimmune diseases, inflammatory disorders, and transplant rejection. These treatments take advantage of Tregs’ natural role in maintaining immune balance by suppressing overactiv...
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Dec 03, 2024
FDA Grants Fast Track for Lin BioScience’s LBS-007; Alnylam’s AMVUTTRA sNDA Under Review; FDA Approves RAPIBLYK for Atrial Fibrillation; Applied Therapeutics Receives CRL for Govorestat in Galactosemia; R289 Gets Fast Track for Lower-Risk MDS.
Lin BioScience Receives FDA Fast Track Designation for LBS-007 Lin BioScience, a clinical-stage biopharmaceutical company focused on developing innovative therapies for acute leukemia, announced that its lead pipeline candidate, LBS-007, has been granted Fast Track Designation by the FDA for the treatment of acu...
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