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AI’s Impact on Regulatory Affairs: From Data Management to Decision Making

ByAaron Csicseri

Artificial Intelligence (AI) is significantly transforming pharmaceutical regulatory affairs. Rather than replacing regulatory professionals, AI enhances their capabilities and allows them more time for strategic activities. This blog post explores how AI is revolutionizing the regulatory submission process, the challenges posed by the rapidly evolving AI regulatory landscape, and what regulatory affairs professionals need to…

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

ByJeff Riegel

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…

Building a Better CAR: Emerging Technology and Safety Concerns Shake Up the Field

ByJeff Riegel

When chimeric antigen receptor (CAR)‑T cells first rolled off the assembly line 7 years ago, with the approval of Kymriah, they were the newest and most technically advanced immunotherapy against cancer, and they demonstrated the potential to elicit profound and durable responses in patients with relapsed/refractory hematologic malignancies. The patient’s own (autologous) T cells are genetically modified in…

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

ByMuzamil Saleem

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

ByJackie Orabone

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’

ByKathryn Madalena

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

ByMuzamil Saleem

“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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ProEd Regulatory’s 4 Key Project Management Principles for Successful AdCom Preparation

BySamantha Procell

Preparing for an FDA advisory committee meeting (AdCom) is a daunting task that can take anywhere from 3 to 9 months of intense preparation. At ProEd Regulatory, we partner with our clients to ensure that they’re well prepared to present their data and answer questions from the committee with confidence and poise. Our clients have…

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The Not-So-Far-Out Therapeutic Promise of Psychedelics

ByAaron Csicseri

Introduction Throughout history, humans have had a complex relationship with psychedelics. For millennia, ancient indigenous cultures used them for spiritual and healing purposes. For example, in 2007, archaeologists in Spain discovered mushrooms found at a burial site dated more than 7,000 years old. The mushrooms found at the site were identified as psilocybin, a type…

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The Rise of ChatGPT in the Pharma Industry

ByAaron Csicseri

Artificial Intelligence (AI) is rapidly transforming how we work, and the pharmaceutical industry is no exception. In particular, the emergence of Generative Pre-trained Transformer (GPT) models has the potential to revolutionize several aspects of the biotech business, including drug discovery and clinical trials. According to a recent survey, 39% of healthcare professionals see AI (including…

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Landmark Approval: First CRISPR Therapy Approved by FDA

ByJackie Orabone

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…

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EMDAC meeting for Intarcia’s Diabetes Drug-Device Combo ends in rejection, but is there a silver lining?  

ByAngela Corona

On Thursday, September 21, 2023, the FDA held a meeting of the Endocrinologic and Metabolic Drugs Advisory Committee (EMDAC) to discuss Intarcia’s new drug application (NDA) for ITCA-650, an implantable device intended to provide continuous dosing of a GLP-1 agonist (exenatide) to treat type 2 diabetes. In a highly unusual move, Intarcia opted to request…