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Vertex, CRISPR score landmark FDA approval for sickle cell disease gene therapy Casgevy

Fierce Pharma

Groundbreaking? Game-changing? Transformational? Historic? | Vertex Pharmaceuticals and CRISPR Therapeutics have won an historic FDA approval for their sickle cell disease gene therapy Casgevy. The transformational treatment is a potential cure for the debilitating and life-threatening disease which affects more than 100,000 in the United States, most of them Black.

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Some of Your Doctors’ Best Qualities are the Same Ones Stifling Your Healthcare Experience

MedCity News

Although problems in US healthcare abound, the most insidious challenges are often tightly coupled with the industry’s most appealing and instilled virtues — a highly selective and independent workforce, (b) an extremely low tolerance for risk, and (c) a high degree of professional oversight.

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As Biden administration eyes 'march-in' rights to lower drug prices, pharma gears up for battle

Fierce Pharma

While so-called “march-in” rights have yet to materialize in the United States, the controversial measure to cut drug prices appears to be gaining traction with the government. | While so-called “march-in” rights have yet to materialize in the United States, the controversial measure to cut drug prices appears to be gaining traction with the government.

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AI’s Role in Transforming Healthcare

MedCity News

Embracing the possibilities that AI unlocks, our commitment to seamlessly integrating AI into healthcare workflows reflects our belief that technology should melt into the background, empowering human caregivers to focus on delivering compassionate patient care.

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Clinical Supply Strategies for CROs

When a CRO is bidding on a project where clinical supplies will be one of the aspects to manage on behalf of the client via a partner, leveraging the expertise of a chosen clinical supply partner can be a valuable resource in demonstrating the CRO’s understanding of and ability to deliver upon critical drug-supply related aspects of the project, and ability to hit key milestones such as FPI for their client.

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FDA Approves Two Landmark Cell-Based Gene Therapies for Sickle Cell Disease

PharmExec

FDA approval of bluebird bio’s Lyfgenia and Vertex Pharmaceuticals' and CRISPR Therapeutics’ Casgevy marks significant milestone in the treatment of sickle cell disease.

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Seattle Children’s Hospital launches BrainChild Bio for CNS therapies

Pharmaceutical Technology

Seattle Children's launched BrainChild Bio to focus on expediting the development of CAR T-cell therapies in central nervous system tumours.

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Moving Beyond Medicine To Treat Chronic Pain

MedCity News

I look at non-pharmacological approaches like digital therapeutics as having the potential to give someone like me living with chronic pain everything I need to make a healthy recovery: access to digital treatments and clinicians, to community, coaching, and navigation services.

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FDA Grants Breakthrough Designation to Johnson & Johnson’s Novel Treatment for HR-NMIBC

PharmExec

TAR-200 has a novel targeted releasing system for the treatment of patients with Bacillus Calmette-Guérin-unresponsive high-risk non-muscle-invasive bladder cancer who are ineligible for bladder removal surgery.

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Drug Solutions Podcast: Staying on Track with Formulation of Cell Culture Media

PharmaTech

Tom Fletcher, director of Research and Development at FUJIFILM Irvine Scientific; and Bob Newman, PhD, chief scientific director at FUJIFILM Irvine Scientific, discuss formulation, specifically with how to prepare culture media for use, single use technologies, and automations to create closed systems in cell/gene therapies with former Pharmaceutical Technology editor Jill Murphy.

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Roche makes obesity play with $2.7bn deal to buy Carmot

pharmaphorum

Roche has agreed a deal to acquire US biotech Carmot Therapeutics that if consummated will thrust it into the increasingly competitive market for incretin-based therapies for diabetes and obesity. The Swiss pharma group has agreed to pay $2.7 billion upfront in cash when the deal closes, with shareholders in the US company also in line for up to $400 million if the drug programmes covered by the agreement meet various milestones.

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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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FDA approves bluebird's sickle cell disease gene therapy. Can Lyfgenia overcome CRISPR’s halo?

Fierce Pharma

Alongside a historic approval for the first therapy utilizing the Nobel Prize-winning CRISPR/Cas9 gene-editing technology, the FDA has cleared a rival gene replacement therapy, also for sickle cell | Alongside a historic approval for the first therapy utilizing the Nobel Prize-winning CRISPR/Cas9 gene-editing technology, the FDA has cleared a rival gene replacement therapy, also for sickle cell disease (SCD).

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Are Registered Dietitians the Key To Optimizing Weight Loss With GLP-1s?

MedCity News

Prescriptions for GLP-1s are soaring, but fewer than a third of people prescribed the costly drugs will continue taking them one year later without personalized support. Without the expert guidance of a registered dietitian, you risk not achieving the significant clinical benefits these drugs offer and forfeiting the potential for sustainable, transformative health outcomes.

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Artificial Intelligence Changing the Landscape for Healthcare Social Media Marketing

PharmExec

Experts from Hootsuite noted that the primary social media ROI concern for healthcare companies is the time and money it takes to maintain a multi-platform presence.

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Japan approves BMS’ Abecma for multiple myeloma treatment

Pharmaceutical Technology

Japan has granted approval for the sBLA of Bristol-Myers Squibb (BMS) for Abecma to treat relapsed or refractory multiple myeloma (RRMM).

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How to Stay Competitive in the Evolving State of Martech

Marketing technology is essential for B2B marketers to stay competitive in a rapidly changing digital landscape — and with 53% of marketers experiencing legacy technology issues and limitations, they’re researching innovations to expand and refine their technology stacks. To help practitioners keep up with the rapidly evolving martech landscape, this special report will discuss: How practitioners are integrating technologies and systems to encourage information-sharing between departments and pr

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Biden may order compulsory licensing in drug pricing move

pharmaphorum

The White House said today that it may take enforced ownership of patents on medicines developed with public funding if their manufacturers set their prices too high. The proposed framework for so-called ‘march-in rights’ on taxpayer-funded drugs and other inventions is the latest entry in the US federal government’s playbook to limit price increases for drugs, and will no doubt throw it once again into open contention with the pharma industry.

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FDA endorses Novartis' 'pipeline in a pill,' granting first nod for potential blockbuster Fabhalta

Fierce Pharma

With an FDA approval in PNH, Novartis has gained its first nod for iptacopan, dubbed two months ago “a pipeline in pill," by analysts at ODDO BHF.

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Which Digital Health Startups Shined the Brightest in 2023?

MedCity News

CB Insights released a list of what it deems to be the 50 most promising digital health companies of 2023. The market research firm said that the startups on the list are working to improve four main aspects of healthcare: personalization, efficiency, equity and accessibility.

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Prevalence of Eight Chronic Health Conditions Reach Unprecedented Levels in US

PharmExec

Arthritis, asthma, cancer, cardiovascular diseases, chronic kidney disease, COPD, depression, and diabetes reach the highest recorded levels since the America’s Health Rankings Annual Report began tracking them in 1990.

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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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Common drug could facilitate “huge step-change” in managing type 1 diabetes

European Pharmaceutical Review

Results from a world-first human trial, published in the New England Journal of Medicine , has shown that a drug called baricitinib has promise as the first disease-modifying treatment of its kind for type 1 diabetes, that can be administered as a tablet.  baricitinib… [demonstrated an ability to] preserve the body’s own insulin production and suppress the progression of type 1 diabetes in people who initiated treatment within 100 days of diagnosis” The researchers noted that baricit

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Trial finds Lilly’s Olumiant could treat type 1 diabetes

pharmaphorum

Treatment with Eli Lilly’s JAK inhibitor Olumiant has been shown to preserve the function of insulin-producing pancreatic beta cells in patients with type 1 diabetes, potentially opening up a whole new avenue of therapy.

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Bristol Myers Squibb bids farewell to dealmaking czar Elizabeth Mily

Fierce Pharma

Three years on, Bristol Myers Squibb is bidding adieu to its top dealmaker. | Three years on, Bristol Myers Squibb is bidding adieu to its top dealmaker. Elizabeth Mily, who replaced BMS’ former M&A czar Paul Biondi back in 2020, is headed for the exit, the company confirmed Friday.

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‘Increasingly Concerned’ Senators Launch Investigation Into Private Equity’s Effect on Healthcare

MedCity News

This week, two bipartisan members of the Senate Budget Committee launched an investigation into private equity and its impact on healthcare. They initiated the probe to get answers about “questionable financial transactions” that could be hurting care quality for patients at hospitals owned by private equity firms.

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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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Reviewing the FDA’s Regulatory Approach to Dietary Supplements

PharmExec

National Library of Medicine study emphasizes inadequacies in ensuring the safety and efficacy of over-the-counter dietary supplements.

Medicine 105
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Antibody-immunotherapy combination shows promise in lung cancer

European Pharmaceutical Review

In an early human study , US researchers have found that combining immunotherapy with dupilumab, an Interleukin-4 (IL-4) receptor-blocking antibody, boosted the immune system of lung cancer patients. Dupilumab is used widely as an allergy and asthma treatment, stated the authors of the corresponding research paper, which has been published in Nature.

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Pfizer’s multiple myeloma treatment secures conditional approval in EU

Pharmaceutical Technology

Pfizer has added the EU to its growing number of markets for its targeted immunotherapy intended for relapsed/refractory multiple myeloma.

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Novartis hit with FDA letter detailing 'significant' Kymriah manufacturing shortfalls

Fierce Pharma

When it comes to manufacturing cell therapies, it's often said that the "process is the product." But churning out these complex, individualized drugs has routinely tripped up even the industry's l | In recent months, Novartis has faced scrutiny from the FDA over Kymriah production shortfalls, a newly posted letter shows. The company says it's working to address the situation.

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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.

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Ketryx Rakes In $14M to Speed Up Medical Device Development & Quality Assurance

MedCity News

Ketryx raised $14 million in Series A funds. The startups sells a platform — designed around FDA regulations and quality standards — that automates much of the work required from medical device companies’ development and quality teams.

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Medtronic drops $738m takeover of wearable insulin pump firm

pharmaphorum

Medtech giant Medtronic has decided not to go ahead with a planned acquisition of EOFlow, a developer of patch-based insulin pumps, saying that there have been “multiple breaches” of their takeover agreement.

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Phase III data for novel telomerase inhibitor released

European Pharmaceutical Review

Results from Geron’s Phase III IMerge trial evaluating its first-in-class investigational telomerase inhibitor imetelstat, have shown long-term and durable response in participants with a rare haematologic malignancy. Efficacy of the treatment was compared to placebo in patients with lower risk myelodysplastic syndromes (MDS) relapsed/refractory or ineligible for erythropoiesis stimulating agents (ESAs).

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The weight loss market looks unstoppable. How high could it go?

PharmaVoice

Market expectations for obesity drugs are soaring and competition is heating up. But hitting the heights will require overcoming payer and other issues.

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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.