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DAWNZERA’s EU Approval Strengthens the Hereditary Angioedema Treatment Landscape

Summary The European Commission granted marketing authorization for DAWNZERA (donidalorsen) on January 21, 2026. Developed by Ionis Pharmaceuticals and Otsuka Pharmaceutical, it is the first and only RNA-targeted therapy approved in the EU for the prevention of hereditary angioedema (HAE). Approval applies to patients aged 12 years and older across the European Union. The European Commission has granted marketing authorization for DAWNZERA (donidalorsen), marking a significant milestone in rare disease therapeutics. Developed by Ionis Pharmaceuticals in partnership with Otsuka Pharmaceutical, DAWNZERA is the first and only RNA-targeted therapy for ...

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FDA Approves Omeros’ YARTEMLEA: A Breakthrough for Stem Cell Transplant Patients Facing TA-TMA

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WASKYRA Wins FDA Approval, Marking a Historic Leap for Rare Disease Gene Therapies

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Novo Nordisk Seeks FDA Approval for Higher-Dose 7.2 mg WEGOVY Injection; Ascendis Pharma Granted FDA Review Extension for TransCon CNP in Children with Achondroplasia; VOYXACT Receives FDA Fast-Track Approval for Treatment of High-Risk Primary IgAN; Sarepta Begins Next ENDEAVOR Cohort Focused on Immunosuppressive Optimization for ELEVIDYS in Advanced DMD; IMFINZI Gains U.S. Approval as the Sole Perioperative Immunotherapy for Early-Stage Gastric and GEJ Cancer

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Arrowhead Hits the Mark in FCS Treatment with REDEMPLO Approval—Now Comes the Ionis Challenge

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Novartis Secures FDA Approval for ITVISMA; Kelun-Biotech’s Phase III Study Shows Sac-TMT + Keytruda Achieves Primary Endpoint in First-Line PD-L1-Positive NSCLC; Evoke Phase 3 Trials Failed to Show a Statistically Significant Impact on Alzheimer’s Disease Progression; Sangamo Therapeutics Gets FDA Nod for Rolling BLA Submission of ST-920 in Fabry Disease; Bayer’s HYRNUO Receives FDA Accelerated Approval for Previously Treated HER2-mutant NSCLC

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Huntington’s Disease Treatment Meets Its Match: Gene Therapy in Action

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Achondroplasia Treatment: Emerging Therapies, Pathway Strategies, and Market Outlook

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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

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UCB’s KYGEVVI Approval: A Breakthrough for Thymidine Kinase 2 Deficiency

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Decoding the $60 Billion Future: Top Trends Driving the DNA Sequencing Market to 2032

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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

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Merck Wins FDA Approval for KEYTRUDA QLEX for Subcutaneous Use in Adults With Solid Tumors; Incyte Gains FDA Nod for OPZELURA Cream in Children Aged 2–11 With Atopic Dermatitis; Minovia Therapeutics Receives FDA Fast Track Designation for MNV-201 in Myelodysplastic Syndrome; MavriX Bio Secures FDA Fast Track for MVX-220 Gene Therapy in Angelman Syndrome; Biocon Biologics Gets FDA Approval for Denosumab Biosimilars BOSAYA and AUKELSO

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The Rise of AI-Powered Point-of-Care Diagnostics: Transforming Real-Time Patient Care

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NeuroNOS’ BA-101 Granted FDA Orphan Drug Status for Glioblastoma; Takeda’s VONVENDI Approval Expanded for Von Willebrand Disease in Adults and Children; Telix Gains FDA Nod on Resubmission Pathway for TLX101-CDx NDA; Saol Therapeutics Receives FDA CRL for SL1009 in Pyruvate Dehydrogenase Complex Deficiency; FDA Grants Breakthrough Therapy Designation to Boehringer’s HERNEXEOS for HER2-Mutant Advanced NSCLC

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Precigen’s PAPZIMEOS Wins Full FDA Approval for Recurrent Respiratory Papillomatosis; Tonix’s Tonmya Becomes First FDA-Approved Fibromyalgia Treatment in Over 15 Years; FDA Extends BLA Review for REGENXBIO’s RGX-121 in MPS II Patients; SystImmune’s Izalontamab Brengitecan (EGFRxHER3 ADC) Gets FDA Breakthrough Designation for EGFR-Mutated NSCLC; Soligenix Granted FDA Orphan Drug Status to SGX945 for Behçet’s Disease Therapy Following Phase II Success

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Anbogen’s ABT-301 Cleared by FDA for Phase I/II Colorectal Cancer Trial; Dyne’s DYNE-251 Gets FDA Breakthrough Tag for Duchenne Muscular Dystrophy; BMS’s BREYANZI Accepted for FDA Priority Review in MZL; Clarametyx’s CMTX-101 Earns FDA Fast Track and QIDP for Infections; Anixa Begins FDA-Approved IND Transfer for Phase II Breast Cancer Vaccine Trial

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