Sat.May 13, 2023 - Fri.May 19, 2023

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New research sheds light on symptoms of understudied spine disease

Medical Xpress

After hearing firsthand from patients about how the disease impacted their lives, a team of researchers and graduate students at University of Western Ontario were inspired to investigate the symptoms associated with an understudied spine disease called diffuse idiopathic skeletal hyperostosis (DISH).

Research 115
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Teva puts rival to Prometheus, Pfizer drugs at center of turnaround plans

Bio Pharma Dive

At an investor day, executives highlighted an experimental inflammatory disease drug targeting the protein TL1A as important to their long-term growth prospects.

Drugs 360
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US FDA approves Bausch + Lomb and Novaliq’s DED treatment Miebo

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has granted approval to Bausch + Lomb and Novaliq’s Miebo (perfluorohexyloctane ophthalmic solution) to treat the signs and symptoms of dry eye disease (DED). Formerly known as NOV03, Miebo is a first-in-class eye drop designed for preventing the evaporation of excessive tears and restoring tear balance in evaporative DED patients.

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Krystal's Vyjuvek becomes first topical gene therapy with FDA nod to treat rare skin disease

Fierce Pharma

Krystal's Vyjuvek becomes first topical gene therapy with FDA nod to treat rare skin disease zbecker Fri, 05/19/2023 - 16:25

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Unlocking Excellence: How Catalent Is Transforming Japan’s Clinical Research

Planning on running clinical trials in Japan? How can you reliably supply these studies? Discover Catalent’s clinical supply packaging facility in Shiga, Japan. Strategically located between Tokyo and Osaka, and one of largest in Japan, this 6,000 square meter facility offers comprehensive services including primary and secondary clinical packaging and labelling, comparator sourcing, cold chain storage, local and global distribution, local language support and white glove service to support stud

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AI voice coach shows promise in psychotherapy for depression and anxiety

Medical Xpress

With scarce clinical resources unable to keep pace with elevated rates of mental illness, technological solutions could help decrease waitlists and disparities in access to therapy. Recent advances in artificial intelligence have fueled interest in the use of chatbots and virtual assistants for mental health treatment.

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Siren emerges from stealth with plans to marry gene therapy to cancer immunotherapy

Bio Pharma Dive

Spun out of the lab of former UCSF researcher Nicole Paulk, the biotech is targeting brain and eye tumors with a new kind of treatment it has dubbed “immuno-gene therapy.

More Trending

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NPR and DrugPatentWatch – Can drug injector pens be swapped?

Drug Patent Watch

On NPR’s All Things Considered, Sydney Lupkin asked DrugPatentWatch if drug patents could be used to identify injector pens which could then be swapped to fix drug shortages. Referring to… The post NPR and DrugPatentWatch – Can drug injector pens be swapped? appeared first on DrugPatentWatch - Make Better Decisions.

Drugs 116
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An elusive mechanism of wasting syndrome cachexia revealed

Medical Xpress

Researchers from the Department of Molecular Biology and Genetics at Koc University in Turkey and the Pole of Endocrinology, Diabetology and Nutrition, Institute of Experimental and Clinical Research at Université Catholique de Louvain in Belgium, have collaborated to interrogate the mechanisms behind a cancer-associated wasting syndrome—cachexia.

Genetics 141
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Biotech startup Ray raises $100M for vision-restoring gene therapies

Bio Pharma Dive

Founded in early 2021, the company is using optogenetics to design treatments that could work across disease genotypes and in the later stages of vision loss.

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CHARM Therapeutics secures funding for oncology drug discovery research

Pharmaceutical Technology

CHARM Therapeutics has received investment from NVIDIA’s venture investment arm, NVentures, for deep learning-enabled drug discovery research. Representing a major milestone for the company, the investment will enable CHARM to do further work on the research and development of new medicines for oncology. It will also allow the company to continue to fund its growing organisation while utilising the accelerated computing platform of NVIDIA.

Research 259
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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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Regulatory tracker: Another China-made PD-1 starts FDA journey as Elevar, Hengrui target big cancer type

Fierce Pharma

Regulatory tracker: Another China-made PD-1 starts FDA journey as Elevar, Hengrui target big cancer type aliu Wed, 05/17/2023 - 09:41

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New treatment for human parasitic worm infections shows high efficacy

Medical Xpress

Soil-transmitted helminth infections are caused by different species of parasitic worms, including whipworms, hookworms and roundworms. Worldwide, more than 1.5 billion people are infected with at least one soil-transmitted helminth, with most of the infected population living in low- and middle-income countries.

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Moderna reports ‘encouraging’ early data for one of its rare disease medicines

Bio Pharma Dive

The biotech, best known for its vaccine research, said its clinical trial is the first to report results of a messenger RNA therapeutic designed for intracellular protein replacement.

RNA 294
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EMA issues guidance amidst European medicine shortage

Pharmaceutical Technology

The European Medicines Agency (EMA) has published recommendations to increase communication and planning efforts in a bid stop the current medicine shortages becoming even worse. Within the 14-page document , published by an EMA special task force, are 10 recommendations of best practices that “marketing authorisation holders, wholesalers, distributors, and manufacturers can consider adopting to ensure continuity of medicinal product supply and reduce the impact of shortages”.

Medicine 249
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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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Intercept's NASH dreams may be dashed after FDA panel votes against Ocaliva's approval bid

Fierce Pharma

Intercept's NASH dreams may be dashed after FDA panel votes against Ocaliva's approval bid fkansteiner Fri, 05/19/2023 - 17:30

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Dementia study reveals how toxic proteins spread through brain

Medical Xpress

Fresh insights into the spread of damaging proteins that build up in the brains of people with Alzheimer's disease could hold the key to stopping the condition from progressing, a study says.

Protein 138
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UniQure sells royalty rights to hemophilia gene therapy

Bio Pharma Dive

The Dutch biotech sold a portion of royalties owed by CSL Behring on sales of Hemgenix, adding to a cash pile it has built since out-licensing the treatment.

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Myeloid raises funds to support lead cell therapy programme

Pharmaceutical Technology

Myeloid Therapeutics has raised $73m to support the continued clinical development of its lead cell therapy programme, MT-101, in Phase I/II trials for T cell lymphoma. Led by Hatteras Investment Partners, the financing round has seen participation from existing investors, including 8VC, Alexandria Venture Investments and Newpath Partners, along with new investors Moore Strategic Ventures and ARCH Venture Partners.

In-Vivo 246
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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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Debiopharm commences pivotal glioblastoma therapy study

Pharma Times

The therapy is a brain-penetrant used in various combinations in patients with progressive glioblastoma

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New threat to privacy? Scientists sound alarm about DNA tool

Medical Xpress

The traces of genetic material that humans constantly shed wherever they go could soon be used to track individual people, or even whole ethnic groups, scientists said on Monday, warning of a looming "ethical quagmire".

Scientist 133
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Roche pushes forward with MS drug amid rivals’ setbacks

Bio Pharma Dive

The company said its “reversible” BTK inhibitor, which trails competitors from Sanofi and Merck KGaA, succeeded in Phase 2 testing without raising new safety concerns.

Drugs 258
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Cost of disease-modifying therapies for multiple sclerosis continues to rise in US

Pharmaceutical Technology

Multiple sclerosis (MS) is a primary autoimmune disease in which inflammation is a core contributor to the degeneration of the central nervous system (CNS), leading to neurological disability and affecting sensory, visual, motor, and autonomic systems. While MS is not a terminal diagnosis, the effect of the disease on the CNS can significantly impact patients’ independence and disturb their daily lives.

Marketing 246
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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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Relieving the itch - new treatment for CKD-associated pruritus in adults on haemodialysis

Outsourcing Pharma

A small molecule drug has been recommended by Englandâs National Institute for Health Care and Excellence (NICE) for the treatment of adults with moderate to severe chronic kidney disease (CKD)-associated pruritus in adult patients on haemodialysis.

Drugs 101
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Engineers design sutures that can deliver drugs or sense inflammation

Medical Xpress

Inspired by sutures developed thousands of years ago, MIT engineers have designed "smart" sutures that can not only hold tissue in place, but also detect inflammation and release drugs.

Engineer 130
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PTC’s drug for PKU succeeds in late-stage clinical trial

Bio Pharma Dive

The biotech plans to discuss the study results with regulators to determine next steps toward an approval application for the rare disease treatment.

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MIF invests in Nectin to investigate multiple myeloma drug candidate

Pharmaceutical Technology

Multiple Myeloma Research Foundation’s (MMRF) venture philanthropy subsidiary Myeloma Investment Fund (MIF) has made an undisclosed investment in Nectin Therapeutics to assess the potential of NTX1088 to treat multiple myeloma. Along with the MIF’s investment, Nectin has so far secured more than $33m from aMoon, Peregrine Ventures, Israel Biotech Fund, Cancer Focus Fund and Integra.

Drugs 246
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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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AbbVie, Genmab's lymphoma drug Epkinly snags FDA approval in heated blood cancer race

Fierce Pharma

AbbVie, Genmab's lymphoma drug Epkinly snags FDA approval in heated blood cancer race aliu Fri, 05/19/2023 - 11:21

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WHO warns against using artificial sweeteners

Medical Xpress

Artificial sweeteners, used to replace sugar in a vast range of products, do not help in losing weight and can have serious health effects, according to the World Health Organization.

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FDA advisers back maternal use of Pfizer RSV vaccine

Bio Pharma Dive

The panel voted 14-0 that Pfizer’s data showed its shot to be effective. The vaccine could be the first for protecting infants by maternal immunization.

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FDA gives go-ahead on labelling for Cumberland’s therapy for fever and pain in infants

Pharmaceutical Technology

The US Food and Drug Administration (FDA) has approved expanded labelling for Cumberland Pharmaceuticals’ Caldolor therapy to include use in infants. Caldolar is an intravenously provided formulation of ibuprofen which may now be administered to treat pain and fever in patients from three months to six months of age. The newly approved label features information about the product’s indications and usage, suitable patient groups, clinical trial results, potential side effects and patient safety

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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.