US FDA reverses course on Regenxbio's rare-disease gene therapy, backs accelerated approval bid

By Siddhi Mahatole June 22 (Reuters) - Regenxbio said on Monday the U.S. FDA has indicated that existing data for its ...
FiercePharma

Government watchdog recommends tweaks to FDA accelerated approval program

An inspector general report has raised some concerns about the FDA's accelerated approval pathway while also showing that the agency's controversial approval of Biogen's Alzheimer's disease drug ...
RAPS

Study: Most nononcology accelerated approval pivotal trials use surrogate endpoints

Most pivotal clinical trials supporting the accelerated approval of nononcology products reported surrogate measures as primary endpoints, according to a recent research letter published in JAMA. Ian ...
Zacks Investment Research on MSN

QURE soars as FDA backs AMT-130 data for accelerated approval

Shares of uniQure QURE soared 78.4% on June 17, after the company announced a major regulatory milestone for AMT-130, its ...
The American Journal of Managed Care

Strategies for Evaluating NSCLC Therapies Granted FDA Accelerated Approval

Experts discuss the FDA's Accelerated Approval Program, emphasizing its role in providing earlier access to innovative cancer therapies for patients. This episode focuses on the strategies for ...
RAPS

Expert proposes changes to accelerated approval reforms in user fee bills

With the Senate Committee on Health, Education, Labor and Pensions (HELP) recently releasing the Food and Drug Administration Safety and Landmark Advancements (FDASLA) act from committee, some ...
Medscape

Accelerated Approval of New Frontline TKI Use in CML Raises Questions

US regulators’ use of a speedy clearance pathway for a new frontline indication for asciminib for chronic myeloid leukemia (CML) has raised questions due to the number of medications already available ...
Healio

‘Turning point’: FDA grants Modeyso accelerated approval for rare diffuse midline glioma

Please provide your email address to receive an email when new articles are posted on . The FDA approved Modeyso for patients with H3 K27M-mutant diffuse midline glioma. The therapy showed an overall ...
Monthly Prescribing Reference

Hyrnuo Granted Accelerated Approval for HER2-Mutant NSCLC

In patients naïve to HER2-targeted therapy, the objective response rate was 71% with a median duration of response of 9.2 months. The Food and Drug Administration (FDA) has granted accelerated ...
JD Supra

New Accelerated Approval Guidance Underscores Need for Accountability

On December 5, 2024, just in time for the holidays, the U.S. Food and Drug Administration (“FDA” or the “Agency”) released a draft guidance titled “Expedited Program for Serious Conditions: ...

Approvals News | Today's Latest Stories | Reuters

Reuters.com is your online source for the latest news stories and current events, ensuring our readers up to date with any ...
Medscape

FDA Issues Accelerated Approval Draft Guidance for Oncology

The US Food and Drug Administration (FDA) released a draft guidance this week that aims to improve the accelerated approval pathway for oncology drugs. The draft guidance, published in the Federal ...