Sat.Dec 03, 2022 - Fri.Dec 09, 2022

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What FDA’s Newest Gene Therapy Approval Tells Us About Durability: How Long is Long Enough?

FDA Law Blog

By Mark A. Tobolowsky & James E. Valentine — On November 22, 2022, FDA approved CSL Behring’s BLA for Hemgenix (etranacogene dezaparvovec), an AAV-based gene therapy for the treatment of adults with Hemophilia B who currently use Factor IX prophylaxis therapy, have current or historical life-threatening hemorrhage, or have repeated, serious spontaneous bleeding episodes.

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Biotech veteran Jeff Jonas on leaving Sage, guiding new biotechs and his ‘personal odyssey’

Bio Pharma Dive

In an interview with BioPharma Dive, the longtime industry executive discussed becoming a biotech investor and his plans for Abio-X, a new incubator with $150 million to spend on startups.

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Kite and Daiichi Sankyo update cell therapy licensing agreement

Pharmaceutical Technology

Kite Pharma and Daiichi Sankyo have updated a partnership agreement signed in 2017 for the former’s CAR T-cell therapy, Yescarta (axicabtagene ciloleucel). Under the prior deal, Daiichi Sankyo acquired exclusive rights for the development, manufacturing and commercialisation of Yescarta in Japan. Subsequently, in the same year, Gilead Sciences acquired Kite.

Licensing 305
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Opinion: Artificial intelligence: crossing the border between health care and tech

STAT News

There’s been significant investment in companies creating artificial intelligence (AI) applications for health and health care over the last decade. But while there have been successes, notably in the area of medical imaging, the industry is known more for not yet living up to its potential — think IBM Watson. The slow pace of AI adoption in health care stems from the fact that health AI sits on the border between two large industries, health care and tech.

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The New Age of Decentralized Clinical Trials

White paper that delves into the complex topic of Decentralized Clinical Trials and how to master them within the confines of FDA Regulations

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Is lecanemab the Alzheimer's drug that will finally make a difference?

NPR Health - Shots

The experimental drug lecanemab was able to slow down Alzheimer's in a large study. Many researchers think the drug will become the first to help large numbers of patients.

Drugs 145
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Dantari emerges with a new way to make cancer drug conjugates

Bio Pharma Dive

As the success of drugs like Enhertu catalyzes new investment in antibody-drug conjugates, the California startup is launching with plans to develop more potent versions of the targeted cancer medicines.

Drugs 328

More Trending

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STAT+: ARPA-H’s new director wants to go on a hiring spree

STAT News

President Biden’s new health agency with a sweeping mandate to cure some of the system’s biggest problems has one big message: Please apply. The Biden administration finally established the Advanced Research Projects Agency for Health in March with an initial $1 billion allotment from Congress after multiple budget delays. The funds, meant to last for three years, are a far cry from the administration’s initial ask of $6.5 billion — which it’s requesting again

Research 143
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China lends billions to poor countries. Is that a burden. or a blessing?

NPR Health - Shots

China doles out much cash for infrastructure. It must be repaid. Is that frustrating for recipients? A new survey has a surprising answer. But skeptics wonder: How honest were the participants?

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Relmada sinks further as depression drug fails second large trial

Bio Pharma Dive

The company claimed an “unplausible” placebo response caused both studies to fail and is making substantive changes to its other remaining late-stage trial as a result.

Trials 315
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US FDA grants EUA for Pfizer-BioNTech’s Omicron-adapted vaccine in children

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted an amendment to the Emergency Use Authorization (EUA) for Pfi z er and BioNTech’s Omicron BA.4/BA.5-adapted bivalent Covid-19 vaccine for usage in children aged six months to four years. The vaccine is indicated for use as a third 3µg dose in the three-dose initial regimen for children of this age group.

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Roles and Responsibilities of Specialized Clinical Supply Experts

When selecting a clinical supply provider, consideration often focuses upon the manufacturing, packaging, storage and distribution capabilities available that will, at face-value, be sufficient to meet the needs of the sponsor and their trial. However, there are human-based and knowledge-driven factors that are often overlooked that go beyond these basic physical capabilities and are integral to the development and delivery of high performing clinical supply chains.

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Opinion: Victims of domestic abuse should get the same top-notch concussion care as athletes

STAT News

Two vastly different experiences — serving as a “guest coach” on the sidelines for a Division I football team and volunteering in a busy emergency department — showed me just how unequal and damaging the lack of care provided for women who are victims of domestic violence can be. In the first decade of the 2000s, one of the benefits of being a professor at Wake Forest University was the opportunity I received nearly every year to serve as a guest coach.

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Thanks to the 'tripledemic,' it can be hard to find kids' fever-reducing medicines

NPR Health - Shots

Makers of products like Children's Tylenol say they're trying to keep up with big demand as RSV, flu, and COVID spread. But medical experts note that kids' fevers don't always call for medicine.

Medicine 143
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Biotech layoffs claim more jobs at Sensei, Instil, TherapeuticsMD

Bio Pharma Dive

Cell therapy Instil Bio will cut 60% of its staff and Sensei Bio will reduce its headcount by 40%. TherapeuticsMD, meanwhile, is laying off all its employees days after announcing a deal to sell off its drug portfolio.

Drugs 311
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Sanofi and Queensland collaborate to build $190m research facility

Pharmaceutical Technology

Sanofi has signed an agreement with the Queensland Government in Australia to establish a $190m (A$280m) research facility in Brisbane. With the establishment of this Translational Science Hub, Queensland is set to become an international messenger ribonucleic acid (mRNA) vaccine hub. According to the latest deal, the University of Queensland (UQ) and Griffith University will serve as foundational partners.

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How Machine Learning Drives Clinical Trial Efficiency

Clinical trial data management is increasingly challenging as studies grow in complexity. Quickly accessing and analyzing study data is vital for assessing trial progress and patient safety. In this paper, we explore real-time data access and analysis for proactive study management. We investigate using adverse event (AE) data to monitor safety and discuss a clinical analytics platform that supports collaboration and data review workflows.

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Opinion: If you think health care is dysfunctional now, just wait until after January 1

STAT News

Doctors across the country, especially those in primary care, have been up in arms about Medicare’s proposed cuts in reimbursement that are scheduled to go into effect on January 1. They are concerned — rightfully so — that these cuts will be ruinous to their practices and compromise the care they can provide to their patients. As an emergency physician, I worry about the cuts for a different reason: emergency departments might soon be filled with more and more people who ca

Doctors 143
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In the hunt for a male contraceptive, scientists look to stop sperm in their tracks

NPR Health - Shots

For decades birth control research focused on women. Now there's a new push to develop gels, pills or other products that could keep men from getting their partners pregnant.

Scientist 141
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TherapeuticsMD to offload drugs to Australia’s Mayne after failed buyout

Bio Pharma Dive

After a private equity takeover unraveled this summer, the women’s health company is selling its drug portfolio to Mayne Pharma in a $153 million deal.

Drugs 325
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Cannabinoids receptors: popular preclinical target but banned in 137 countries

Pharmaceutical Technology

Cannabinoid receptors are a popular therapeutic target for cannabinoid-based drugs in the treatment of pain, neurological disorders and inflammation, according to GlobalData’s Pharma Intelligence Centre Drugs database. In 2022, a surge in the pipeline has led to cannabinoid receptors becoming the most popular target in preclinical development. Despite this, the medical use of cannabinoid drugs is heavily restricted, including being banned in 137 countries, according to the United Nations.

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What the FDA's New Dosage Guidance Means for the Future of Clinical Research

Speaker: Dr. Ben Locwin - Biopharmaceutical Executive & Healthcare Futurist

What will the future hold for clinical research? A recent draft from the FDA provides valuable insight. In "Optimizing the Dosage of Human Prescription Drugs and Biological Products for the Treatment of Oncologic Diseases," the FDA notes that "targeted therapies demonstrate different dose-response relationships compared to cytotoxic chemotherapy, such that doses below the Maximum Tolerated Dose (MTD) may have similar efficacy to the MTD but with fewer toxicities.

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New research links common sweetener with anxiety

Medical Xpress

Florida State University College of Medicine researchers have linked aspartame, an artificial sweetener found in nearly 5,000 diet foods and drinks, to anxiety-like behavior in mice.

Research 137
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Cancer patients endure an overlooked financial burden: hospital parking fees

STAT News

For many patients, one of the most antagonizing parts of a hospital visit is paying for parking. Those parking fees aren’t just an annoyance for the sick and injured, according to a new paper in the Journal of Medical Imaging and Radiation Sciences. The charges are actually eating into their financial well-being, particularly for people who have cancer and have to make frequent visits to the hospital for treatments like radiation and chemotherapy.

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Star Therapeutics launches Vega, growing its galaxy of drug startups

Bio Pharma Dive

The second startup to emerge from Star’s “hub-and-spoke” model aims to develop a Hemlibra-like antibody drug for the blood clotting disorder von Willebrand disease.

Drugs 297
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Why vaccine hesitancy persists in China — and what they're doing about it

NPR Health - Shots

The reluctance of many citizens — especially the elderly — to get vaccinated is a problem for a government facing intense pressure to roll back strict COVID policies.

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The New Age of Decentralized Clinical Trials

This new white paper defines and details the impact of Decentralized Clinical Trials on the Pharmaceutical industry and how the impact can be measured along with steps companies can take to ensure adoption.

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Expanding the arsenal of drugs for use against COVID-19

Medical Xpress

The ongoing COVID-19 pandemic, caused by the SARS-CoV-2 virus, has been devastating the entire world. While the vaccination program is advancing, drug treatments for COVID-19 are still highly important for those who become infected.

Drugs 131
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Eli Lilly tightens access to diabetes drug, frustrating some people with obesity

STAT News

Touted by celebrities, raved about by TikTok users and advertised by med spas, a new class of drugs for treating diabetes and obesity has exploded in popularity for its weight-loss effects, leading to rippling shortages across several of the medications. Amid the surge in demand, Eli Lilly and pharmacies have started to tighten access to the latest of this type of drug, tirzepatide, focusing on giving it to people with type 2 diabetes, the only population it’s authorized for so far.

Drugs 124
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Novartis prostate cancer drug succeeds in study key to company’s sales hopes

Bio Pharma Dive

The positive results could help Novartis secure approval for earlier use of the radiopharmaceutical drug Pluvicto, potentially broadening the number of eligible patients by several times the current market.

Sales 294
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Because of Wisconsin's abortion ban, one mother gave up trying for another child

NPR Health - Shots

Kristen Petranek has a history of miscarriages – and she has diabetes, which makes pregnancy risky. She fears that if something goes wrong, her state's law may inhibit doctors from helping her.

Doctors 136
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Accelerating Clinical Supply Through Integrated Drug Development

As the development pipeline for new drugs continues to grow, biopharmaceutical companies are re-evaluating how to best manage and balance resources across an increasing number of development projects and complex clinical trials. There are two approaches that can be used to speed a drug from development to clinic faster: timeline compression and parallel processing, but only one that considers the benefits of integrating clinical supply into the overall drug development process.

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How a viral toxin may exacerbate severe COVID-19

Medical Xpress

A new study in the journal Nature Communications reveals how a viral toxin produced by the SARS-CoV-2 virus may contribute to severe COVID-19 infections.

Research 135
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Opinion: Deferral of primary care signals a troubled future for Americans’ health

STAT News

Americans may be returning to their pre-pandemic habits , but most have not returned to their primary care doctors. Primary care visits are down 10.3% on average across U.S. cities relative to pre-pandemic levels. That, combined with more people with chronic diseases like diabetes, obesity, and cancer, and accelerating health care costs as inflation soars, signal a troubled future for the health of Americans and the U.S.

Doctors 124
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Esperion, without data, says cholesterol pill lowered heart risk in study

Bio Pharma Dive

The large cardiovascular outcomes trial is a major test for Esperion, which has struggled to sell its drug Nexletol since winning U.S. approval in 2020.

Trials 300
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When COVID closed India, these women opened their hearts — and wallets

NPR Health - Shots

Sangeeta Siwan lost her job and wasn't able to pay rent or feed her family. Her neighbors helped her survive. A study of giving in India during the pandemic shows she wasn't alone.

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Deliver Fast, Flexible Clinical Trial Insights with Spotfire

Clinical research has entered a new era, one that requires real-time analytics and visualization to allow trial leaders to work collaboratively and to develop, at the click of a mouse, deep insights that enable proactive study management. Learn how Revvity Signals helps drug developers deliver clinical trial data insights in real-time using a fast and flexible data and analytics platform to empower data-driven decision-making.