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Regulatory Reforms on the Horizon

In February 2024, we reported on anticipated changes to the format of FDA advisory committee meetings (AdComs) based on public comments from FDA Commissioner Robert Califf and other FDA officials earlier this year. Some of those changes have, indeed, come to pass—most notably, a full‑throated return to in-person AdComs at the White Oak facility in…

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The Power of Inclusion: Navigating the FDA’s Diversity Action Plan

In December 2022, Congress passed the Food and Drug Omnibus Reform Act (FDORA), which requires, among other things, that drug and device sponsors submit diversity action plans for clinical trials that commence enrollment 180 days after publication of the final guidance. FDORA codifies a long-standing push by the US Food and Drug Administration (FDA) to…

The First FDA-Approved NASH Treatment Arrives After Almost Half a Century in the Making

The FDA has approved Madrigal Pharmaceuticals’ Rezdiffra™ (resmetirom) for nonalcoholic steatohepatitis (NASH). The approval is a watershed event, marking a historic turning point in the long-standing quest to find an effective treatment for the millions of Americans afflicted by this devastating condition.  A targeted treatment for NASH has eluded pharmaceutical scientists for nearly a half century……

Rare Disease Day 2024

This year, Rare Disease Day is on February 29, 2024. Rare Disease Day is observed on the last day of February, which is the “rarest of calendar dates1 to underscore the nature of these diseases” and serves to raise awareness of rare diseases and those individuals affected by them. FDA is holding a virtual meeting2…

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AdComs: They Are a-Changin’

Changes Are Coming to Advisory Committee Meetings Advisory Committee meetings (AdComs) are a long-standing feature of the FDA drug approval process. How and where those meetings are conducted has been evolving over the past few years, since the introduction of virtual meetings, and even more considerable reforms may be coming sooner rather than later. According…

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FDA Convenes Experts to Address Knowledge Gaps in Noninvasive Testing for NASH

“Silent killer” is an apt description for a potentially fatal disease that progresses slowly, without any obvious symptoms in the early stages. When you couple this insidious type of pathology with a rapidly increasing prevalence, the disease is often described in terms akin to an epidemic. Nonalcoholic steatohepatitis (NASH)—a disease characterized by fat accumulation in the…

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Landmark Approval: First CRISPR Therapy Approved by FDA

Today, the US Food and Drug Administration (FDA) approved exagamglogene autotemcel (exa‑cel), the first CRISPR-based gene therapy for the treatment of sickle cell disease (SCD). Exa‑cel was co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics. This is a landmark approval that paves the way not only for better outcomes for patients with SCD, but for many…