Trending Articles

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Pharma benefited from basing business overseas. An international tax effort could spur a rethink.

Bio Pharma Dive

U.S. tax law changes enacted six years ago slashed large pharma companies' rates and saved them billions. Now, a push for an international floor could disrupt their R&D accounting.

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Aro Biotherapeutics raises funds for Pompe disease treatment

Pharmaceutical Technology

Aro Biotherapeutics has raised $41.5m in a Series B financing round to advance the development of ABX1100 for Pompe disease.

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Health Canada approves Jazz’s cannabis derived seizure therapy

Pharmaceutical Technology

Epidiolex has been approved as an adjunct therapy for seizures associated with three rare forms of epilepsy in patients aged two and older.

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The Significance of the MHRA Approval and Upcoming FDA Review of the First Gene Editing Treatment

Worldwide Clinical Trials

By: Amy Raymond, PMP, PhD, Senior Director, Therapeutic Strategy Lead, Cellular & Genetic Medicines On November 16, the British regulatory body, Medicines and Healthcare products Regulatory Agency (MHRA) approved exagamglogene autotemcel (exa-cel) to treat severe sickle cell disease for patients ages 12 and up, becoming the first gene editing treatment to receive regulatory approval.

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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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November 30, 2023: HCSRN Calls for Abstracts and Panel Presentations for 2024 Annual Conference

Rethinking Clinical Trials

The Health Care Systems Research Network (HCSRN) is accepting abstract submissions and panel presentation submissions for its 2024 Annual Conference until December 11, 2023. This year’s meeting will be held in Milwaukee, Wisconsin, from April 9 to 11, 2024. The HCSRN is a 20-member research network focused on supporting research institutes aligned with healthcare delivery systems.

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The Role of Process and Technology Innovation in Advanced Biopharmaceutical Manufacturing

Pharma Mirror

By Alexei Voloshin, Global Head of Bioprocess Science, 3M Biopharmaceutical Purification Article Synopsis: Modern biotechnology has brought a revolution in improving the human condition around the world both in terms of quantity and quality. The rapid pace of innovation in biopharmaceutical treatments has enabled the treatment of life-threatening and life-changing conditions such as cancer, inflammation, and infectious disease.

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Acelyrin claims errors by CRO Fortrea led to late-stage trial downfall

Pharmaceutical Technology

A Phase IIb/III trial studying the company’s immunotherapy izokibep, failed to achieve statistical significance for its primary endpoint

Trials 243
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FDA investigates 'serious risk' of secondary cancer following CAR-T treatment

Fierce Pharma

In a blow to CAR-T therapies, the FDA is investigating a “serious risk” of patients developing new cancers after treatment with these highly efficacious oncology drugs. | In a blow to CAR-T therapies, the FDA is investigating a “serious risk” of patients developing new cancers after treatment with these highly efficacious oncology drugs.

Drugs 143
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November 28, 2023: Workshop Summary Now Available From ‘Getting the Right Evidence to Decision-Makers Faster’

Rethinking Clinical Trials

The workshop summary is now available from the NIH Pragmatic Trials Collaboratory’s recent workshop, “Getting the Right Evidence to Decision-Makers Faster.” The 2-day workshop explored the critical cycle of evidence generation by researchers to decision-making by healthcare system leaders to implement the findings of pragmatic clinical trials conducted within healthcare systems.

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BMS Expands Cardio Partnership with Avidity in Potential $2.3B Deal

BioSpace

Building on a previous 2021 collaboration, Bristol Myers Squibb is paying $100 million upfront for the development of five cardiovascular targets utilizing Avidity Biosciences’ antibody oligonucleotide conjugates.

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Commercial Drug Sourcing for Clinical Trials

The sourcing of commercial drugs for use in clinical trials either as a comparator, rescue or co-medication can be a complex process. Sponsors can face a number of challenges when trying to obtain these critical supplies for their studies, including addressing product lead times, availability, expiry limitations, safety and cost, among others. Choosing the right partner with global sourcing, regulatory, quality and clinical packaging expertise can provide the necessary guidance to help sponsors

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FDA names chief scientist Bumpus as Woodcock’s successor

Bio Pharma Dive

Bumpus, a former Johns Hopkins professor, named “creating a new model” for the FDA’s Office of Regulatory Affairs as one of her priorities when she steps into the role.

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Pfizer takes Poland to court over alleged missed COVID-19 vaccine payments

Fierce Pharma

As the pandemic eased earlier this year, officials in Poland became vocal critics of the financial obligations laid down by Pfizer’s large vaccine supply pact with the European Union. | As the pandemic eased earlier this year, officials in Poland became vocal critics of the financial obligations laid down by Pfizer’s large vaccine supply pact with the European Union.

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November 29, 2023: Special Biostatistics Series Continues With Guidelines for Stepped-Wedge Trials

Rethinking Clinical Trials

In this Friday’s PCT Grand Rounds, Jim Hughes of the University of Washington will continue our special series, Advances in the Design and Analysis of Pragmatic Clinical Trials, with his presentation, “Guidelines for Design and Analysis of Stepped-Wedge Trials.” The session will be held on Friday, December 1, at 1:00 pm eastern. Hughes is a professor emeritus of biostatistics at the University of Washington.

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Karuna Secures FDA Decision Date for Schizophrenia Candidate KarXT

BioSpace

The regulator accepted Karuna Therapeutics' NDA for KarXT and set a PDUFA date of September 26, 2024. If approved, it would be the first new mechanism of action to treat schizophrenia in decades, the company contends.

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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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BioMarin secures hemophilia gene therapy coverage in Germany

Bio Pharma Dive

Drawn-out negotiations led to a lower price than initially expected, but analysts called the agreement a step forward for the biotech company.

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VPAS is dead, long live VPAG—could the UK’s new clawback revision save the day?    

Pharmaceutical Technology

After months of negotiations, the UK's DHSC and the Association of the British Pharmaceutical Industry emerged with a plan on to replace VPAS.

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Amgen expands pact with Amazon to usher drug manufacturing into the AI era

Fierce Pharma

Seeking to identify ways to improve the discovery and production of medicines, Amgen is expanding its partnership with tech giant Amazon. | Amgen is growing its decade-old collaboration with Amazon Web Services—a widely used cloud platform—to create generative artificial intelligence that the companies aim to use to increase the manufacturing throughput of pharmaceuticals.

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November 27, 2023: NIH Announces Funding Opportunity for Implementation Studies of Substance Use Treatment and Prevention in Populations That Experience Health Disparities

Rethinking Clinical Trials

The National Institutes of Health’s Office of Disease Prevention (ODP) issued a new funding opportunity to support implementation studies in treatment and prevention for alcohol, tobacco, and other drugs use and misuse in adult populations that experience health disparities. ODP and participating NIH Institutes and Centers are inviting applications for research projects that test innovative approaches to implementing screening, brief intervention, and referral to treatment or prevention fo

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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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Legalized medical cannabis five years on - Releaf discusses how far the industry hasn't come

BioPharma Reporter

It is five years this month since a ground-breaking change in UK healthcare history happened â with doctors being able to prescribe cannabis-based medicines for the first time.

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Roivant’s Matt Gline on raising capital and biotech’s outlook in 2024

Bio Pharma Dive

Fresh off a $7 billion deal with Roche, the chief executive spoke with BioPharma Dive about this year’s “discordant combination” of major biotech achievements alongside market headwinds.

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FDA accepts Orexo’s NDA for opioid overdose medication

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has accepted to review Orexo’s new drug application (NDA) for opioid overdose medication, OX124.

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Biden admin urges Supreme Court to nix mifepristone restrictions

Fierce Pharma

Following similar campaigns mounted by industry players and biopharma trade groups, the United States' Solicitor General has joined a chorus of voices calling on the Supreme Court to overturn an ap | Following similar campaigns mounted by industry players and biopharma trade groups, the United States' Solicitor General has joined a chorus of voices calling on the Supreme Court to overturn an appeals court’s August ruling on the abortion pill mifepristone.

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2022 Research: The Rapid Rise of Ocean Freight Visibility

A research study conducted by The Journal of Commerce and FourKites surveyed hundreds of international shippers, exploring how their usage of global supply chain visibility technology has evolved since the onset of global disruptions caused by COVID-19. For international shippers, ocean freight visibility has evolved from optional to essential and satisfaction with visibility varies greatly depending on how it is obtained and delivered.

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SpringWorks Wins First FDA Approval for Non-Cancerous Desmoid Tumors

BioSpace

The Ogsiveo tablets are the first approval therapy for the rare subtype of soft tissue sarcomas, which can lead to severe pain and disability and previously were treated primarily through surgery.

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What’s a corneal epithelial defect?

Antidote

The cornea is the outermost layer of the human eye, serving both as a protective covering and allowing light into the retina. To perform this function, the cornea is made up of several layers, the outermost of which is known as the epithelium.

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FDA investigating cancer risk linked to CAR-T cell therapy

Bio Pharma Dive

The agency said the benefit of approved treatments like Gilead’s Yescarta still outweighs any such risk, but the alert could slow drugmaker efforts to develop the treatments for wider use.

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Bayer expands German manufacturing facilities to fulfill supply demand

Pharmaceutical Technology

Bayer announced the launch of a Berlin, Germany-based manufacturing facility to meet future demands from the US, China and Europe.

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An Innovative & Creative Problem Solver Approach to Selling in the Medical Device Space

Speaker: Steve Goldstein, Sales Leader

Are you currently in sales, or involved in a business that depends on strong sales results? What about the extremely competitive world of medical device sales? What are some of the top challenges your customers face and how do you approach understanding what’s most important to them? Join Steve Goldstein, Sales Success Coach, Motivational Speaker and Medical Device Sales Leader from Gold Selling LLC., to discover critical strategies and approaches you can take to engage your customers, achieve g

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Scottish stem cell pro RoslinCT inks deal to produce Vertex and CRISPR's Casgevy

Fierce Pharma

Following the historic approval of the world’s first CRISPR-based gene-editing therapy in the United Kingdom this month, Vertex Pharmaceuticals has tapped its long-time manufacturing partner Roslin | RoslinCT, the CDMO arm of the Roslin Institute, is set to manufacture Vertex and CRISPR Therapeutics’ exa-cel “across the world” as part of Vertex’s global production network.

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Boehringer Ingelheim Targets Stroma-Rich Cancers in Potential $509M AI Deal

BioSpace

The pharma giant is paying $9 million upfront to Phenomic AI to develop targets for stroma-rich cancers, some of the hardest cancers to treat, utilizing its single-cell RNA computing platform.

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Who Monitors the Monitors? CRA Evaluations Essential to Better Trials and Relationships

ACRP blog

In the best of all possible worlds, clinical trial teams at study sites get along professionally and productively with each and every clinical research associate (CRA) who visits in person or remotely to monitor the progress and compliance of studies on behalf of their sponsors. In the real world, however, the relationship may be more of a “can’t live with them, can’t live without them” scenario as far as the site team members’ feelings about certain CRAs are concerned.

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AbbVie joins ADC dealmaking with $10B ImmunoGen buyout

Bio Pharma Dive

The acquisition of ImmunoGen gives AbbVie an approved medicine for ovarian cancer, and continues a pharma spending spree on antibody-drug conjugates.

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Making the Case for Case Management for Cell and Gene Therapies

As demand for advanced therapies increases, so does the need for more specialized supply chain support, as these products have strict transportation and handling requirements. Deviations from the intended supply chain can delay timelines and affect these high value products, which can be detrimental to budgets, but more importantly can have dire effects on the patients whose lives depend on receiving these critical doses safely and on time.