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

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…

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

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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FDA Decision for Sarepta’s Gene Therapy for DMD Sets New Regulatory Precedent

Historic Regulatory Decision for First FDA-Approved Gene Therapy for DMD On June 22, 2023, in a-much anticipated decision, the United States Food and Drug Administration (FDA) granted accelerated approval for Elevidys (also known as SRP-9001), Sarepta’s one-time gene therapy for ambulatory children with Duchenne’s muscular dystrophy (DMD). However, the accelerated approval was limited to the…

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New Weapons Emerge in the Fight Against Respiratory Syncytial Virus (RSV)

Imagine your newborn baby has been hospitalized with a serious respiratory infection and is struggling to breathe. That’s a reality for thousands of US newborns and their parents every year during the respiratory syncytial virus (RSV) season. This common endemic virus doesn’t cause clinically significant disease in most children and adults, but it can cause…

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Communicating the Complexities of Subgroup Analyses at an AdCom

Within clinical trials, exploratory or post-hoc subgroup analyses are widely recognized as only “hypothesis generating” due to their high potential for bias and/or misleading interpretation. This is the main reason why Sponsors cannot make efficacy claims or seek regulatory approval based on evidence of efficacy in a certain subgroup unless that benefit is consistent with…

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Quick Snips: CRISPR

CRISPR is a powerful gene-editing tool that enables targeted therapeutic gene editing, with clinical applications for improving treatment of inherited and/or rare genetic diseases, viral infections, and arresting the progression of cancer. CRISPR gene-editing technology has the potential to revolutionize the treatment of rare genetic disorders, with the first product poised for FDA approval in…

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Neurology Takes a Page Out of the Oncology Playbook of FDA Accelerated Approvals

The field of neurology is experiencing a significant upswing in innovative therapeutic development, propelled by advances in genetics, neuroimaging techniques, and biomarker research. However, neurological diseases are inherently difficult to treat, and there remains an urgent need to rapidly translate these advances into more effective treatments. It is timely then, that several recent drug approvals…

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The Rise of ChatGPT in the Pharma Industry

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…

Thinking Outside the Beta-Amyloid Box

The dominant amyloid hypothesis of AD has translated into an armada of anti-amyloid biologics in the near-term pipeline—led by Eisai and Biogen’s lecanemab. Last month, lecanemab showed a slowing in the rate of cognitive decline by 27% over 18 months versus placebo. Lecanemab also possesses a relatively improved safety profile among the beta-amyloid mAbs, as…

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Multiple Data Sources Show That Decentralized Clinical Trials Pay Off

Traditional clinical trials are typically conducted at central locations or sites that patients must travel to in order to be evaluated and treated by trial investigators. These are referred to as centralized clinical trials. In contrast, a decentralized clinical trial (DCT) has no set location for patients to report to. Rather, clinical trial activities are…