MPR

Otarmeni Wins FDA Approval for Genetic Sensorineural Hearing Loss

Supported by the CHORD trial, this AAV vector-based therapy delivers a working copy of the OTOF gene and restores otoferlin ...
Fierce Pharma

FDA's accelerated approval pathway needs stronger transparency, evidence standards: ICER

The FDA should strengthen the evidence required for drug approvals under its accelerated pathway and increase transparency ...

FDA gives three vouchers for faster psychedelic approvals

The US has given three drugmakers accelerated pathways to approval for psychedelic medications with clinical trials underway.
The American Journal of Managed Care

Lunsotogene Parvec Becomes First FDA-Approved Gene Therapy for OTOF-Related Hearing Loss

Formerly known as DB-OTO, lunsotogene parvec is an adeno-associated virus (AAV) vector–based gene therapy administered via a ...
Applied Clinical Trials Online

FDA Clears Lunsotogene Parvec for OTOF-Related Genetic Hearing Loss

The FDA has granted accelerated approval to lunsotogene parvec-cwha (Otarmeni), an adeno-associated virus (AAV) vector–based ...
BioSpace

Drug pricing watchdog calls for increased transparency into FDA’s accelerated approval decisions

Greater transparency in FDA decision-making was possibly the most common theme for stakeholders interviewed by the Institute ...
PharmExec

The Long Wait for Rare Disease Treatment Approval: Q&A with Dr. Patricia E. Greenstein

FDA is formalizing RWD as core evidence, enabling “single pivotal trial plus confirmatory evidence” approaches that may ...
pharmaphorum

FDA accelerated approval path needs improvement, says ICER

That's the conclusion of a just-published report (PDF) from the influential Institute for Clinical and Economic Review (ICER) ...
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 ...
RAPS

FDA’s accelerated approval program: Is change on the way?

The U.S. Food and Drug Administration (FDA) launched its accelerated approval program in 1992, offering a pathway to bring life-saving drugs and biologics to the market faster. After nearly 30 years, ...
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 ...