BioReview

Biotechnology

Larimar Therapeutics Achieves FDA Breakthrough: First Protein Replacement Therapy for Friedreich's Ataxia Receives Accelerated Development Status

Larimar Therapeutics announced FDA Breakthrough Therapy Designation for nomlabofusp, the first protein replacement therapy for Friedreich's ataxia, opening accelerated pathways for this devastating rare disease.

Pharmaceuticals

Takeda and Protagonist Achieve Major FDA Milestone: First-in-Class Polycythemia Vera Therapy Rusfertide Receives Priority Review

In a significant regulatory achievement that could transform treatment for one of hematology's most challenging conditions, Takeda Pharmaceutical and Protagonist Therapeutics announced on March 2, 2026, that the FDA has accepted their New Drug Application (NDA) and granted Priority Review for rusfertide, a first-in-class hepcidin mimetic peptide for

Biotechnology

Neurogene Achieves Historic FDA Breakthrough: First Gene Therapy for Rett Syndrome Receives Accelerated Development Status

In a landmark regulatory achievement that could transform treatment for one of pediatric neurology's most devastating conditions, Neurogene announced on February 27, 2026, that the FDA has granted Breakthrough Therapy Designation to NGN-401, positioning the company's investigational gene therapy as the first potential cure for Rett

Biotechnology

Pentixapharm Breaks New Ground: FDA Clears Revolutionary CXCR4-Targeted Theranostic Program for Blood Cancer Treatment

Berlin-based Pentixapharm announces FDA clearance of two Investigational New Drug applications for its groundbreaking CXCR4-targeted theranostic program combining precision imaging with targeted radiotherapy for blood cancer treatment.

Biotechnology

Gilead's $7.8 Billion Gamble: Why the Arcellx Acquisition Could Reshape Multiple Myeloma Treatment

Gilead Sciences' $7.8 billion acquisition of Arcellx marks a strategic bet on anito-cel, a promising CAR-T therapy that could transform multiple myeloma treatment and revitalize the company's cell therapy business.

Policy

FDA Drops Two-Trial Dogma: Historic Policy Shift Could Accelerate Drug Approvals and Transform Biotech Development

In what may be the most significant regulatory development for biotech in a decade, the FDA announced on February 18, 2026, that it is abandoning its long-standing expectation of two pivotal clinical trials for new drug approvals. Moving forward, the agency will require only one adequate and well-controlled clinical trial

Biotechnology

Genentech Achieves Major Regulatory Milestone as FDA Accepts Giredestrant Application for Advanced Breast Cancer

Genentech announces FDA acceptance of New Drug Application for giredestrant, an oral selective estrogen receptor degrader combination therapy for ESR1-mutated advanced breast cancer, with priority review and December 2026 decision target.

Pharmaceuticals

Johnson & Johnson Achieves FDA Breakthrough: Rybrevant Faspro Expands Beyond Lung Cancer Into Head and Neck Malignancies

In a significant regulatory milestone that could transform treatment options for one of oncology's most challenging malignancies, Johnson & Johnson announced on February 18, 2026, that the FDA has granted Breakthrough Therapy Designation to Rybrevant Faspro (amivantamab and hyaluronidase-lpuj) for treating advanced head and neck squamous cell carcinoma.

Pharmaceuticals

Novartis Achieves Historic Breakthrough: Remibrutinib Becomes First Targeted Therapy to Succeed in Chronic Inducible Urticaria Phase III Trial

Novartis announces positive Phase III results for remibrutinib in chronic inducible urticaria, positioning the oral BTK inhibitor as the first targeted therapy for this debilitating condition affecting millions worldwide.

Biotechnology

Krystal Biotech Achieves Historic FDA Breakthrough: First Inhalable Gene Therapy for Lung Cancer Receives RMAT Designation

Krystal Biotech announces FDA RMAT designation for KB707, the world's first inhalable gene therapy for advanced lung cancer, marking a revolutionary breakthrough in cancer treatment.

Pharmaceuticals

FDA's Unprecedented Rejection of Moderna's mRNA Flu Vaccine Sends Shockwaves Through Biotech Industry

The FDA's unprecedented rejection of Moderna's mRNA flu vaccine application marks a dramatic departure from traditional regulatory practices and raises profound questions about the future of vaccine innovation in America.

Biotechnology

HanchorBio Achieves FDA Breakthrough: First SIRPα-IgG4 Fc Fusion Protein Granted Orphan Status for Gastric Cancer

Taiwan-based HanchorBio announced FDA Orphan Drug Designation for HCB101, marking the first recognition of a SIRPα-IgG4 Fc fusion protein in gastric cancer treatment.