All articles tagged with #fda approvals
Kalshi will start offering prediction-market bets on clinical trial outcomes and FDA regulatory decisions, selecting an initial set of markets with partner AppliedXL, signaling a move to apply market-based forecasting to biopharma and potentially provide new data-driven insights while aiming to manage risks in the field.
A Louisiana man became the state’s first functionally cured sickle cell patient via 2023 FDA-approved gene therapies Casgevy and Lyfgenia; while these treatments hold promise for tens of thousands who are Black or African American, their high cost (around $2–$3 million) and limited treatment centers create access barriers, highlighting the need for broader insurance coverage, expanded care in underserved communities, and thorough discussion of risks like infertility and long‑term cancer risk.
The piece chronicles how the once-dominant amyloid-β hypothesis has been undermined by multiple retractions and high-profile drug failures, fueling debate over data integrity and regulatory controversy. While FDA-approved antibodies targeting amyloid-β have had limited or questionable benefits and come with serious risks, researchers are increasingly exploring alternatives—such as neuroinflammation, infections, the gut microbiome, and APOE4-related pathways—suggesting Alzheimer's may result from multiple interacting factors rather than a single cause. The article highlights the persistence of amyloid research despite misconduct findings and casts doubt on a simple cure tied to amyloid-β clearance.
The FDA will default to approving drugs based on a single well-controlled pivotal trial, while allowing supporting confirmatory evidence such as mechanistic data, related indications, animal models, or real-world data. The move, framed by FDA leaders as a shift from an outdated ‘two-trial dogma,’ aims to speed development and cut costs, but has drawn criticism from some policymakers and former FDA officials who warn it could weaken patient protections. The policy preserves discretion to require additional studies in certain cases, and reactions are mixed among analysts and industry figures, with Pazdur expressing concerns before his retirement.
In 2025, the FDA approved 46 new therapeutic agents, including a diverse range of modalities such as biologics, kinase inhibitors, and gene therapies, with cancer remaining the most common therapeutic area. The year was marked by policy changes, staffing challenges, and the launch of new approval pathways, reflecting a tumultuous but innovative period for drug development.
The FDA approved 55 new drugs in 2023, marking a significant increase from the previous year and setting a 10-year rolling average high. Oncology led with 24% of the approvals, followed by neurology. Notable approvals included the first CRISPR–Cas9-based gene editing product, two RSV vaccines, and a burst of gene therapies. AstraZeneca's capivasertib became the first AKT inhibitor approved for breast cancer, and several first-in-class cancer therapies were greenlit. In neurology, Biogen and Ionis's tofersen received accelerated approval for SOD1-mutated ALS, and Eisai and Biogen's lecanemab was approved for Alzheimer's disease. The approvals spanned various modalities, including nucleotide-based therapeutics and T-cell engaging bispecific antibodies.