Fri.Feb 23, 2024

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BioMarin preaches patience amid slow sales for hemophilia gene therapy

Bio Pharma Dive

The company earned only $3.5 million last year from its Roctavian treatment, far below the $50 million to $150 million range it had forecast eight months ago.

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FDA generic drug approvals rose in 2023 in bid for improved access

Pharmaceutical Technology

The FDA Office of Generic Drugs reported a rise in generic drug approvals, as several first-time generics entered the market.

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J&J secures EMA backing for earlier CAR-T use in multiple myeloma

Bio Pharma Dive

European drug regulators recommended clearing J&J and Legend Biotech's Carvykti for use as early as after first relapse, potentially giving the therapy an advantage over Bristol Myers’ Abecma.

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NICE recommends Pfizer’s Ritlecitinib for alopecia areata treatment

Pharmaceutical Technology

The UK NICE has recommended Pfizer's Ritlecitinib as a new treatment option for individuals suffering from alopecia areata.

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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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The opportunities and challenges facing rare disease therapies developers

pharmaphorum

Explore the opportunities and challenges facing rare disease therapies developers, including the impact of Rare Disease Day, FDA regulations, and cutting-edge technologies like CRISPR gene therapy.

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AstraZeneca completes Gracell Biotechnologies acquisition for $1.2bn

Pharmaceutical Technology

AstraZeneca has announced the conclusion of its acquisition of Gracell Biotechnologies for approximately $1.2bn.

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FDA to decide on Sanofi/Regeneron’s Dupixent for COPD in June

Pharmaceutical Technology

Sanofi and Regeneron’s Dupixent has received priority review by the US FDA, with a target action date of 27 June.

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To BioMarin CEO Alexander Hardy, hemophilia A gene therapy Roctavian needs 3 stars to align

Fierce Pharma

Even Alexander Hardy, the former Genentech leader who managed the blockbuster hemophilia drug Hemlibra, can’t immediately bend the laggard launch trajectory of BioMarin’s gene therapy Roctavian.

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Bavarian Nordic’s chikungunya vaccine secures EMA fast-track

Pharmaceutical Technology

The EMA has granted an accelerated review of CHIKV VLP as Bavarian Nordic attempts to catch Valneva’s Ixchiq.

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Bavarian Nordic gets swift EU review of chikungunya shot

pharmaphorum

Bavarian Nordic will get an accelerated review of its chikungunya vaccine CHIKV VLP at the EMA, as it tries to chase down a lead held by rival Valneva. The Danish pharma group said it is planning to file for approval of its vaccine in the first half of this year, adding the EU regulator’s human medicine committee, the CHMP, has said it will cut the review time from 210 to 150 days under its accelerated assessment procedure.

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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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CHMP provides positive opinion for BMS’s Reblozyl

Pharmaceutical Technology

The positive opinion is based on findings from the Phase III COMMANDS trial which met its primary endpoint.

Trials 130
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Congressional lawmakers strike back at Biden admin's 'march-in' proposal to corral drug costs

Fierce Pharma

As the Biden administration’s exploration of “march-in rights" draws support from powerful entities like the Federal Trade Commission (FTC), a group of lawmakers on both sides of the aisle have war | As the Biden administration’s exploration of “march-in rights" draws support from powerful entities like the Federal Trade Commission, a small group of lawmakers on both sides of the aisle have warned the approach could “hamstring” U.S. innovation.

Drugs 100
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Moderna reports net loss of $4.7bn for full year 2023

Pharmaceutical Technology

Moderna has reported a net loss of $4.7bn for the full year 2023 as against a net income of $8.4bn in 2022.

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Joining a clinical trial: Helpful hints for discussions with loved ones

Antidote

Individuals living with an illness are likely well-versed in the art of discussing difficult topics with their family and friends. However, for those who are considering taking part in a clinical trial, there may be extra questions to consider when sharing their intent to enroll.

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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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Anticipation rises for RSV rollout as positive data emerges

Pharmaceutical Technology

Following major successes in the RSV landscape over the last few years, experts debate rollout plans.

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AI improves adenoma detection in colonoscopies

pharmaphorum

AI software developed by Israeli medtech Magentiq-Eye has been shown to improve detection rates for adenoma, a precursor to colorectal cancer, in colonoscopy procedures

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After long road to approval, Teva and Alvotech believe their Humira biosimilar Simlandi may have an edge

Fierce Pharma

Teva and Alvotech have braved a long and winding road on the quest to win approval for their biosimilar to AbbVie’s Humira. | Teva and Alvotech have braved a long and winding road on the quest to win approval for their biosimilar to AbbVie’s Humira. Now, after multiple manufacturing setbacks, the partners have finally scored their inaugural regulatory nod in the United States.

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FDA will decide in June on Dupixent for COPD

pharmaphorum

The FDA has started a priority review of Sanofi and Regeneron’s Dupixent as a treatment for chronic obstructive pulmonary disease (COPD), which could become the first new treatment for the widespread and life-threatening disease in over a decade.

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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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Oncopeptides' Pepaxto reaches end of the line in US after FDA yanks approval

Fierce Pharma

Following a contentious advisory committee meeting and an FDA request for the withdrawal of Oncopeptides’ multiple myeloma drug Pepaxto from the U.S. | Following a contentious advisory committee meeting and an FDA request for withdrawal of Oncopeptides’ multiple myeloma drug Pepaxto from the U.S. market in late 2022, the other shoe has dropped.

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After Roche’s exit, Blueprint passes Gavreto on to Rigel

pharmaphorum

After losing Roche as a partner, Blueprint Medicines has sold US commercial rights to RET inhibitor Gavreto to Rigel Pharma in a deal worth up to $117.

Medicine 105
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'The Top Line': Breaking down Eli Lilly’s ESG formula

Fierce Pharma

This week on “The Top Line,” we explore the impact of environmental, social and governance measures, commonly known as ESG, on the pharmaceutical industry. | This week on "The Top Line," we explore the impact of environmental, social, and governance measures, commonly known as ESG, on the pharmaceutical industry. Fierce Pharma's Fraiser Kansteiner engages in a conversation with Jim Greffet, Head of ESG Strategy at Eli Lilly, to gain insights and discuss Lilly's specific ESG

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CSL, Travere close on EU approval of IgAN drug sparsentan

pharmaphorum

Travere and partner CSL Vifor close on EU approval of sparsentan for rare kidney disease IgA nephropathy, after CHMP's positive opinion on the drug.

Drugs 104
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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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Rare Disease Day 2024: Highlighting Statistics and Patient Stories

XTalks

Rare Disease Day is a global observance held on the last day of February each year that is aimed at raising awareness among the general public and decision-makers about rare diseases and their impact on patients’ lives. Rare Disease Day 2024 falls on February 29 this year as this year is a leap year, making the day even more rare and special. Despite varying definitions across different countries and regions, a disease is generally deemed to be rare if it affects less than one in 2,000 people.

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Revolutionizing clinical trials with generative AI: A discussion with IQVIA's Wing Lon Ng

Outsourcing Pharma

In the fast-evolving landscape of healthcare and pharmaceuticals, the role of technology, particularly artificial intelligence (AI), is becoming increasingly prominent.

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In search of the fabled tricorder

pharmaphorum

In search of the legendary tricorder using cutting-edge AI and MedTech. Discover the future of healthcare technology today.

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Identifying Trends Surrounding the Use of In-House Pharmacies for High-Cost Drugs

Pharmaceutical Commerce

A cross-sectional study investigates how this supply chain link helps increase Medicare Part D spending on self-administered specialty drugs.

Pharmacy 104
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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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NHS offers preventative bowel cancer screening for patients with Lynch syndrome

Pharma Times

In England, around 1,100 cases of bowel cancer are caused by the inherited condition every year

Genetics 112
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Four Keys to Success for Retail and Specialty-Lite Hub Services

Drug Channels

Today’s guest post comes from Nicole Grycan, VP of Commercialization at Phil, Inc. Nicole discusses how hubs can improve access and adherence. She outlines four success factors for manufacturers that use a digital hub partner. To learn more, register for Phil's upcoming webinar: Improving Access, Coverage, and GTN with a Digital Hub. Read on for Nicole’s insights.

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AbbVie’s Skyrizi shows promise as personalised treatment for psoriasis

Pharma Times

The immunosuppressant reduced inflammation in patients with the skin condition after three days

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Meet the researcher: Jenny Hogstrom-Stakem

Drug Discovery World

Jenny Hogstrom-Stakem , Postdoctoral Research Fellow, was recently awarded the 2023 AACR-AstraZeneca Breast Cancer Research Fellowship. Here she shares her career highlights and advice with DDW’s Diana Spencer. DS: Can you tell us what you do and what you’re working on at the moment? JHS : I’m a postdoctoral research fellow at Beth Israel Deaconess Medical Center/ Harvard Medical School doing breast cancer research.

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The New Clinical Trial Supply Chain: Resilient, Flexible, and Patient-Centric

The global landscape of clinical trials is rapidly changing as studies become more complex. An increasing number of sponsors are seeking enhanced flexibility in their supply chains to address a variety of clinical supply challenges, including patient demand and reducing delays. Demand-led supply and direct-to-patient distribution are next-generation solutions that are helping to meet these growing needs, allowing for more streamlined processes and patient-centric studies.