ALS News Today

Clene plans CNM-Au8 filing for accelerated approval in ALS

Au8, an experimental ALS therapy. CNM-Au8 has been associated with NfL reductions, a biomarker finding linked to longer ...
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5, 2024, just in time for the holidays, the U.S. Food and Drug Administration (“FDA” or the “Agency”) released a draft guidance titled “Expedited Program for Serious Conditions: ...
JD Supra

FDA Issues Draft Guidance on Accelerated Approval Focused on How the Agency Will Implement New Authorities

On December 5, 2024, the U.S. Food and Drug Administration (FDA or Agency) issued a draft guidance entitled Expedited Program for Serious Conditions—Accelerated Approval of Drugs and Biologics (Draft ...
The Manila Times

Agios Submits sNDA to FDA for U.S. Accelerated Approval of Mitapivat in Sickle Cell Disease

NDA submission follows agreement with FDA on confirmatory trial, a requirement of the accelerated approval pathwayConfirmatory trial designed to demonstrate clinical benefit of mitapivat on reducing ...
RAPS

Study: Accelerated approval pathway working as intended in most cases

Over the last several decades, the US Food and Drug Administration’s (FDA) accelerated approval program has largely been working as it was designed to, with about half the products using the pathway ...
FiercePharma

Government watchdog recommends tweaks to FDA accelerated approval program

A government watchdog examining the FDA’s accelerated approval pathway has released a 32-page report (PDF) that concludes the agency’s controversial endorsement of Biogen’s failed Alzheimer’s ...
The American Journal of Managed Care

5 Key April FDA Approvals Signal Momentum Across Rare, Chronic Diseases

INSTI, Tenofovir-Free Regimen DOR/ISL Gains FDA Approval for HIV This fixed-dose, 2-drug combination is a once-daily, ...
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 ...
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 ...
AOL

US FDA's accelerated approval for drugs needs more transparency, says research firm

April 16 (Reuters) - The U.S. Food and Drug Administration should be more transparent when granting accelerated approval for certain drugs, influential drug pricing body, Institute for ‌Clinical and ...
Healio

Safety updates ‘common’ for cancer drugs granted FDA accelerated approval

Please provide your email address to receive an email when new articles are posted on . Most oncology drugs that received FDA accelerated approval between 2011 and 2020 required postmarketing safety ...
Monthly Prescribing Reference

Hernexeos Gets Accelerated Approval for HER2-Mutant Advanced NSCLC

Continued approval for this indication is contingent upon verification and description of clinical benefit in a confirmatory trial. The Food and Drug Administration (FDA) has granted accelerated ...