As reported on BioSpace, the U.S. Food and Drug Administration (FDA) has approved Partner Therapeutics’ bispecific antibody Bizengri for adults with cholangiocarcinoma harboring an NRG1 gene fusion, marking a significant…
As reported on FierceBioTech, Gilead Sciences has closed one of its midstage clinical studies evaluating an experimental HIV regimen, following ongoing safety concerns that prompted U.S. regulators to intervene last…
On February 23, 2026, FDA issued a Draft Guidance document, called “Considerations for the use of the Plausible Mechanism Framework to Develop Individualized Therapies that Target Specific Genetic Conditions with Known Biological…
As reported on PharmaBiz, the US Food and Drug Administration (FDA) has approved Novo Nordisk’s higher‑dose formulation of semaglutide, Wegovy HD (7.2 mg once weekly), for chronic weight management. The…
As reported on PharmaBiz, the US Food and Drug Administration has broadened the approved use of GSK’s respiratory syncytial virus (RSV) vaccine, Arexvy, to include adults aged 18 to 49…
As reported by FiercePharma, the U.S. Food and Drug Administration has approved the first fully independent generic version of GlaxoSmithKline’s widely used asthma inhaler, Flovent, marking a significant shift in…
As reported on FierceBioTech, the U.S. Food and Drug Administration has released its complete response letter (CRL) detailing why it declined to approve Regenxbio’s experimental gene therapy, RGX‑121, for Hunter…
The U.S. Food and Drug Administration has agreed to review Otsuka Pharmaceutical’s application for centanafadine, a new extended‑release therapy under investigation for attention‑deficit hyperactivity disorder (ADHD). The agency has granted…
The U.S. Food and Drug Administration (FDA) has approved Yartemlea (narsoplimab‑wuug), marking the first authorized therapy for hematopoietic stem cell transplant‑associated thrombotic microangiopathy (TA‑TMA) in adults and children aged 2…
As reported on PharmaBiz, Amneal Pharmaceuticals, in collaboration with mAbxience, has received U.S. Food and Drug Administration (FDA) approval for two denosumab biosimilars: Boncresa (referencing Prolia) and Oziltus (referencing Xgeva).…
In a recent statement, the U.S. Food and Drug Administration (FDA) has authorized regular approval to rucaparib (Rubraca) for adults with metastatic castration-resistant prostate cancer (mCRPC) harboring deleterious BRCA mutations—either…
As reported by Healio, the U.S. Food and Drug Administration (FDA) has officially qualified AIM-MASH AI Assist, marking a milestone as the first artificial intelligence tool cleared to support drug…
In a recent statement, The U.S. Food and Drug Administration (FDA) has introduced the Technology-Enabled Meaningful Patient Outcomes (TEMPO) Pilot, a groundbreaking initiative aimed at expanding access to digital health…
The U.S. Food and Drug Administration (FDA) has approved Koselugo (selumetinib), an oral MEK inhibitor developed by Alexion, AstraZeneca Rare Disease, for adults with neurofibromatosis type 1 (NF1) who have…
Denali Therapeutics (Nasdaq: DNLI) announced that the U.S. Food and Drug Administration (FDA) has extended the review period for its Biologics License Application (BLA) for tividenofusp alfa, an investigational therapy…
The U.S. Food and Drug Administration (FDA) has granted Fast Track designation to SAR402663, Sanofi’s investigational gene therapy for neovascular age-related macular degeneration (AMD). As seen in a report, this…
As 2025 winds down, the pharmaceutical industry is bracing for a series of pivotal FDA decisions that could redefine treatment paradigms and reshape competitive dynamics across several major therapeutic areas.…
NS Pharma, Inc., a subsidiary of Nippon Shinyaku Co., Ltd., has received a significant regulatory boost for its experimental treatment targeting Duchenne muscular dystrophy (DMD). As reported in The AI…
On September 3, 2025, the US Food and Drug Administration (FDA) introduced the Rare Disease Evidence Principles (RDEP) process, a groundbreaking step for drug development targeting ultra-rare genetic diseases. According…
After years of determined advocacy and scientific perseverance, Stealth BioTherapeutics has achieved a landmark victory with the FDA’s accelerated approval of elamipretide (now branded as Forzinity) the first-ever treatment for…
The U.S. Food and Drug Administration (FDA) has introduced a new regulatory framework aimed at improving and clarifying the approval process for drugs targeting rare diseases, also known as orphan…
August 2025 was a landmark month for rare disease innovation, as the U.S. Food and Drug Administration (FDA) granted four historic approvals, each representing a first for their respective conditions,…
Ultragenyx Pharmaceutical, a biotechnology company developing treatments for rare diseases, has received a setback from the U.S. Food and Drug Administration (FDA) regarding its gene therapy candidate, DTX401. According to…
The FDA recently informed Muscular Dystrophy News that it has granted orphan drug designation to the experimental drug ISX9-CPC, a product of the IPS Heart company located in Houston, Texas.…
The FDA is taking significant steps to update and streamline its regulatory approach to rare diseases, according to a recent article from BioCentury.com. Recognizing the unique challenges faced by patients,…
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