An FDA official kicked off the 2023 Muscular Dystrophy Association (MDA) Clinical and Scientific Conference Monday with a keynote address that described the steps the agency is taking to advance the development of gene therapies for rare disorders.

Peter Marks, director of the FDA's Center for Biologics Evaluation and Research (CBER), summarized his speech in an interview with Neurology Live, in which he highlighted the work of the MDA, in particular.

More than 10% of drugs granted accelerated approval by the US Food and Drug Administration (FDA) have not complied with the agency’s 2019 labeling guidance, according to a new study. The study authors wrote that the lack of labeling transparency can affect prescribing habits and call on FDA to ensure regulatory compliance through meetings with manufacturers, warning letters or by levying fines.

Targeting everyday chronic conditions to rare diseases that require costly treatments, CMS Tuesday announced an intent to test 3 new models in an effort to lower drug prices and widen access across Medicare Part D, Medicare Part B, and Medicaid.

It’s going to be a long wait, but RegenXBio is already waving a flag to show that the Hunter syndrome gene therapy RGX-121 is headed for an accelerated approval request at the FDA.

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, could substantial reforms be on the way?

At RAPS Convergence 2021, David Dorsey of Janssen R&D, said Congress could choose to make changes to the accelerated approval program through the upcoming reauthorization of the Prescription Drug User Fee Act (PDUFA). Congress has already acted to codify and grant enforcement authority to the FDA through the original act and subsequent reauthorizations, he explained.

The FDA on Monday handed an accelerated approval to Seagen and Genmab’s Tivdak (tisotumab vedotin-tftv, or “TV”) in second-line patients with recurrent or metastatic cervical cancer who previously progressed after chemotherapy rather than PD-(L)1 systemic therapy, the companies said in a release.

Roche has decided to voluntarily withdraw the US accelerated approval for Tecentriq (atezolizumab) in combination with Abraxane (nab-paclitaxel) to treat patients with PD-L1-positive metastatic triple-negative breast cancer (mTNBC).