FDA

The Measurement Problem Behind Two Decades of Neurology Trial Failures

The FDA now treats continuous, real-time data sharing as a regulatory direction of travel rather than a theoretical future. The neurology field should pay close attention.

GSK Eyes FDA Filing After Immunotherapy Succeeds in Key Rectal Cancer Study

GSK drug Jemperli met the main goal of a pivotal Phase 2 test in patients with a rare type of rectal cancer, paving the way for regulatory submissions. This antibody drug has already received a voucher to speed up its review under a new FDA pilot program.

Brain Interfaces Don’t Need Surgery to Be Transformative – and the Distinction Is Now a Strategic One

Non-surgical implantable BCI is increasingly capable of matching surgical performance while transforming the risk-benefit equation, and it is now the most credible path along all three dimensions simultaneously.

Vera Therapeutics’ First-in-Class Kidney Disease Drug Lands Speedy FDA Approval

FDA approval of Vera Therapeutics’ Trutakna makes it first in a new class of medicines designed to block two targets to treat immunoglobulin A nephropathy, an autoimmune disease that can progress to end-stage renal failure. Competition looms from Vertex Pharmaceuticals, on track to receive a November regulatory decision for a drug designed to block the same two proteins.

Organ-on-a-chip, Organoids, but Drug Testing on Whole Organs? This Startup is Betting On It

Many startups and established companies focused on drug testing on human data in preclinical studies have been boosted by FDA's move away from reliance on animal testing towards organoid, organ-on-a-chip, and other in silico technologies. A Connecticut startup believes whole-organ testing should be an essential part of this mix.

Orca Bio Cell Therapy Gets Landmark FDA Nod for New Kind of Living Medicine

FDA approval of Orca Bio’s Tregzi makes it the first cell therapy based on regulatory T cells, or Tregs. Clinical trial results showed reduced risk of graft-versus-host disease, a common complication of the allogeneic stem cell transplants that are a standard treatment for certain blood cancers.

FDA Approves Viridian’s Thyroid Eye Disease Drug, Competitor to a Blockbuster Amgen Med

Viridian Therapeutics’ Lumvoa is approved for treating both the chronic and active phases of thyroid eye disease. This drug will offer patients an alternative to Tepezza, an Amgen drug that had been the only approved therapy for this inflammatory eye disorder.

A Metabolic Disease Posing Severe Pancreatic Risks Gets Its First FDA-Approved Therapy

Ionis Pharmaceuticals’ Tryngolza is now the first approved therapy for treating severe hypertriglyceridemia, a prevalent chronic metabolic disorder that can lead to acute pancreatitis. This drug is a key piece of Ionis’s pivot away from partnering its assets, and instead developing and commercializing them on its own.

Real-Time Trials Are Here — Now We Need Real-Time Trust

For years the healthcare system has relied on the slow machinery of research — and that system is failing the people it was built for. It was designed for a world that operated with an abundance of caution and lacked the modern tools of today. That world no longer exists.

New FDA Nod Keeps Gilead Drug Competitive With AstraZeneca & Daiichi in Breast Cancer

Gilead Sciences antibody drug conjugate Trodelvy is now approved as a first-line treatment for triple-negative breast cancer. This expanded approval for Trodelvy comes with a slightly broader label that gives it an edge over the AstraZeneca and Daiichi Sankyo ADC Datroway.

FDA Reversal on uniQure Gene Therapy Is Another Sign of Renewed Regulatory Flexibility

UniQure is preparing to seek accelerated FDA approval for its Huntington’s disease gene therapy, a move that follows the FDA’s reversal of its prior position calling for another clinical trial. This gene therapy is the latest in a growing list of rare disease treatments to receive reconsideration from an FDA that appears to be swinging back to regulatory flexibility.

The FDA’s Bayesian Guidance Could Quietly Reshape Clinical Trial Design

New FDA guidance on the use of Bayesian statistics signals a broader shift in accommodating more flexible clinical trial designs and the complexities of diseases such as certain cancers and rare disorders, which may lead to more efficient trials, lower development costs, and faster innovation.

The FDA Opened the Door For Rare Disease Patients — Here’s What It Takes to Walk Through It

The Plausible Mechanism Framework is a breakthrough. Turning it into treatments that reach families will take five things the framework doesn't provide.

Amgen Defends Rare Disease Drug in FDA Crosshairs

FDA concerns about Amgen’s Tavneos were sparked by securities fraud litigation against ChemoCentryx, the company that originally developed the rare disease drug. The FDA wants to withdraw Tavneos’s approval, citing safety risks and alleged manipulation of the drug’s clinical trial data.

Debunked Episode 27: The Impact of an FDA Commissioner’s Departure, Insider Trading, Microdosing GLP-1s and a Win for Lower Drug Prices

MedCity News Editor-in-Chief Arundhati Parmar and Samir Batra, Managing Partner of Health Innovation Pitch, co-hosts of the Debunked Podcast, discussed the impact of Marty Makary’s resignation from the FDA, among other topics.