March, 2024

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Amylyx ALS drug fails crucial study, putting company’s future in doubt

Bio Pharma Dive

The results have led Amylyx to pause promotion of Relyvrio and potentially pull it from the market in the coming weeks, a major blow to the company and ALS patients.

Drugs 339
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March 7, 2024: New Report Sets Out Posttrial Responsibilities in Pragmatic Clinical Trials

Rethinking Clinical Trials

Dr. Stephanie Morain In a new report from the NIH Pragmatic Trials Collaboratory, a team of bioethicists and implementation scientists argue for a “presumptive default” that the results of pragmatic clinical trials should be incorporated into healthcare delivery processes. This responsibility arises from a key rationale for conducting pragmatic trials: that they can facilitate uptake of their results by relevant decision-makers.

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Oncology emerges as dominant therapy area for CRISPR technology

Pharmaceutical Technology

In late 2023, the Medicines and Healthcare products Regulatory Agency approved Vertex’s Casgevy, which became the inaugural clustered regularly interspaced short palindromic repeats (CRISPR)-based drug for the haematological indications beta thalassemia and sickle cell disease (SCD).

Medicine 278
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Extreme Case of Man Who Had 217 COVID Vaccines Surprises Scientists

AuroBlog - Aurous Healthcare Clinical Trials blog

Two hundred and seventeen. That’s how many vaccines a man in Germany claims to have received for COVID-19 in just 29 months – and his body isn’t reacting the way some scientists thought it would.

Scientist 221
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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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With Pfizer struggling in 2023, CEO Bourla hit with 35% pay cut to $21.6M

Fierce Pharma

Pfizer knew 2023 was going to be a challenging year of transition. But even the drugmaker was surprised by the plummeting demand for its COVID-19 products, missing badly on its 2023 guidance. | After a difficult year for Pfizer, in which its share price fell by 44%, CEO Albert Bourla’s compensation fell by 35% from $33 million in 2022 to $21.6 million in 2023.

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FDA Approves Madrigal’s Rezdiffra as First MASH Therapy

BioSpace

Madrigal Pharmaceuticals’ Rezdiffra (resmetirom) is the first-ever approved therapy for metabolic dysfunction-associated steatohepatitis—a decision experts say could signal a sea change in treatment of the disease.

More Trending

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Cannabinoids show promise in acute migraine clinical trial

pharmaphorum

Inhaled cannabinoids have been shown to perform better than placebo in providing pain relief for people suffering from acute migraine in a clinical trial

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AD/PD 2024: TauRx’s HMTM promising despite lacking primary endpoint data

Pharmaceutical Technology

Participants cannot remain blind to their treatment arm due to the properties of TauRx's drug, which causes urinary discoloration.

Drugs 299
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NABL Kolkata spearheads cutting-edge awareness programme on calibration of medical devices

AuroBlog - Aurous Healthcare Clinical Trials blog

In order to set in pioneering progress in Eastern India’s laboratory standards, the National Accreditation Board for Testing and Calibration Laboratories (NABL) Kolkata office has orchestrated a ground-breaking virtual awareness program, revolutionizing perspectives on calibration within the Eastern India region’s laboratories.

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FDA approves first MASH drug: Madrigal's Rezdiffra breaks ground in notorious biopharma graveyard

Fierce Pharma

The decades-long wait for an effective treatment for metabolic dysfunction-associated steatohepatitis (MASH) has ended, as the FDA has approved the first drug for the fatty liver disease. | The decades-long wait for an effective treatment for metabolic dysfunction-associated steatohepatitis (MASH) has ended. The FDA has approved Madrigal's resmetirom under the brand name Rezdiffra as the first drug for the fatty liver disease.

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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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BMS’s Breyanzi Becomes First CAR-T Therapy for Two Types of Leukemia

BioSpace

The FDA approved Bristol Myers Squibb’s Breyanzi for chronic lymphocytic leukemia and small lymphocytic leukemia prior to Friday’s adcomm for the company’s other CAR-T therapy, Abecma.

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Madrigal, FDA approval in hand, outlines plan to sell MASH drug

Bio Pharma Dive

The company expects initial uptake of Rezdiffra, which costs $47,400 per year, to be slow as doctors and insurers establish the protocols needed to find patients eligible for treatment.

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Lilly’s Alzheimer’s drug delayed as FDA seeks adcomm

pharmaphorum

The FDA has said it will seek the advice of independent advisors before it concludes its review of Eli Lilly’s amyloid-targeting Alzheimer’s disease therapy donanemab, delaying its review of the drug. Lilly said the US regulator “wants to further understand topics related to evaluating the safety and efficacy of donanemab,” which includes the safety and efficacy results of its pivotal TRAILBLAZER-ALZ 2 study.

Drugs 124
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The growing uses of medicinal cannabis

Pharmaceutical Technology

Demand for medicinal cannabis is increasing globally to treat a range of illnesses and conditions.

Medicine 340
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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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There’s a Surprisingly Simple Way to Remove Microplastics From Your Drinking Water

AuroBlog - Aurous Healthcare Clinical Trials blog

Tiny fragments of microplastics are making their way deep inside our bodies in concerning quantities, significantly through our food and drink. Scientists have now found a simple and effective means of removing them from water.

Scientist 225
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Novo's obesity superstar Wegovy wins FDA nod to cut cardio risks in key label expansion

Fierce Pharma

Novo Nordisk’s fast-growing weight loss med Wegovy just added a new cardiovascular FDA approval to its label, likely enabling the med's superstar status to reach new levels. | The drug is the first weight loss treatment to win FDA approval to reduce the risk of severe cardiovascular outcomes, giving it an edge over rival Zepbound from Eli Lilly.

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AstraZeneca Boosts Rare Disease Portfolio with $1.05B Amolyt Acquisition

BioSpace

At the center of the deal is Amolyt Pharma’s late-stage candidate eneboparatide for the rare disease hypoparathyroidism. AstraZeneca also gains ownership of AZP-3813, which is being assessed for acromegaly in a Phase I trial.

Trials 109
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After surprise trial failure, ALS doctors brace for one less treatment option

Bio Pharma Dive

Physicians are preparing for tough conversations with patients on Amylyx Pharmaceuticals’ drug, Relyvrio, while holding out hope it still may help some with the disease.

Doctors 294
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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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Pain is reduced by almost half with cannabis-based medicines – report finds

Outsourcing Pharma

On March 7, Celadon Pharmaceuticals plc, a leading UK-based pharmaceutical company specializing in cannabis-based medicines, unveiled promising findings from an early economic analysis of its chronic pain clinical trial, dubbed CANPAIN.

Medicine 120
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EMA pins lack of long-term data as rationale for Novartis’ failed Vijoice bid

Pharmaceutical Technology

The EMA raised concerns about the lack of long-term data for Novartis Vijoice in its initial bid, as the Swiss company prepares for a new regulatory submission.

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Should You Let Your Dog Lick Your Face? Here’s The Science

AuroBlog - Aurous Healthcare Clinical Trials blog

Any dog owners reading this will know the joy of getting home and being greeted with utter delight by their pet – tail wagging wildly before enthusiastic but face-drenching licks.

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AstraZeneca pledges £650M in UK investments to boost vaccine capabilities, expand near HQ

Fierce Pharma

AstraZeneca CEO Pascal Soriot, who has been critical of the U.K.’s business environment for the life sciences industry, is directing a big chunk of money into the Big Pharma’s home country. | AstraZeneca CEO Pascal Soriot, who has been critical of the U.K.’s business environment for the life sciences industry, is directing £650 million into the Big Pharma’s home country.

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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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FDA Adcomm to Tackle Complexity of CAR-T for Multiple Myeloma

BioSpace

BMS and J&J will meet with the Oncologic Drugs Advisory Committee Friday to discuss their CAR-T therapies Abecma and Carvykti as the companies seek their approval as earlier lines of treatment.

Drugs 125
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Amylyx CEOs look for a path forward following major setback

Bio Pharma Dive

Justin Klee and Joshua Cohen spoke to BioPharma Dive about the next steps for their company, which is considering pulling its only product from market after the drug failed a confirmatory study in ALS.

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7 things to know about multiple sclerosis

Antidote

Receiving a multiple sclerosis (MS) diagnosis can feel overwhelming and isolating for many. MS is a neurodegenerative disease that often involves challenging symptoms — but knowing what to expect and what resources are available can help.

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With FDA greenlight, BMS Breyanzi brings CAR-T cell therapy to CLL and SLL

Pharmaceutical Technology

Amidst the many BTK inhibitors, Breyanzi will be the first and only CAR-T cell therapy available for the treatment of CLL and SLL.

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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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Women in life sciences: Miruna Sasu

pharmaphorum

Celebrate International Women’s Day by learning about Miruna Sasu, a prominent female figure in the life sciences field and CEO of COTA. Explore how the company uses AI in the oncology space.

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Despite early death concerns, FDA advisers back J&J and Legend's Carvykti for earlier myeloma

Fierce Pharma

Even though the BCMA CAR-T therapy Carvykti showed a potential problem of early patient deaths in a trial, experts on the FDA’s Oncologic Drugs Advisory Committee (ODAC) still believe that the drug | Even though the BCMA CAR-T therapy Carvykti showed a potential problem of early patient deaths in a trial, experts on the FDA’s Oncologic Drugs Advisory Committee still believe that the drug’s long-term benefits outweigh its risks.

Trials 123
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BIO Boots WuXi AppTec from Trade Group Amid US National Security Concerns

BioSpace

In a change of position as congressional scrutiny of WuXi AppTec grows, the Biotechnology Innovation Organization announced it is taking steps to separate from the China-based biotech.

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German biotech Tubulis cashes in on ADC ‘momentum’ with €128M financing

Bio Pharma Dive

The funding for the startup, which partnered with Bristol Myers Squibb last year, adds to a surge of recent investments in developers of antibody-drug conjugates.

Antibody 290
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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.