Wed.May 24, 2023

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FDA delays decision on Duchenne gene therapy and considers narrower approval

Bio Pharma Dive

The agency will extend its review of Sarepta’s treatment by roughly one month as it weighs limiting an initial OK only to children with Duchenne aged 4 to 5 years old.

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Untangling the Complexities of Cell and Gene Therapy Clinical Trials: A Supply Chain Perspective 

Pharmaceutical Technology

By Luisa Sterkel & Joana Loureiro , Tenthpin Consultants The promise and potential of cell and gene therapies (CGT) has emerged in the recent past and currently over 1.500 CGT are registered for clinical trials holding great hope for the treatment of challenging and uncurable diseases. The ability to modify or introduce genetic material in human cells in such a precise and patient-centered manner clearly constitutes a breakthrough in personalized medicine.

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FDA approves new antibiotic for hospital-acquired pneumonia

Bio Pharma Dive

The drug, called Xacduro, will be made available later this year by Innoviva Specialty Therapeutics, a new company formed from antibiotic developers Entasis and La Jolla.

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Forge Biologics joins BGTC for new AAV gene therapies development

Pharmaceutical Technology

Forge Biologics has joined the public-private collaboration, the Bespoke Gene Therapy Consortium (BGTC), to expedite the development and manufacture of new AAV [adeno-associated virus] gene therapies to treat patients with rare diseases. The BGTC is part of the accelerating medicines partnership (AMP) programme and is managed by the Foundation for the National Institutes of Health (FNIH).

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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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PTC lays off staff, fires CFO as neuromuscular drug study fails

Bio Pharma Dive

The biotech is giving up on early-stage gene therapy research as part of a restructuring meant to cut costs and redirect resources toward therapies most likely to succeed.

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New Research May Explain How This Virus Can Cause Multiple Sclerosis in Some People

AuroBlog - Aurous Healthcare Clinical Trials blog

An estimated 2.8 million people around the world have multiple sclerosis (MS), an autoimmune disease in which the immune system damages the brain and spinal cord. Symptoms include fatigue, vision disturbance, problems with mobility and balance, and cognitive dysfunction.

Research 202

More Trending

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To bring food & drugs under one controller, TN govt appoints an IAS officer R Lalvena as director of DCA

AuroBlog - Aurous Healthcare Clinical Trials blog

With an intention to bring both the administrations of the food safety department and the drug control under one controller, the Government of Tamil Nadu has given the charge of director of drugs control administration (DCA) to the commissioner of food safety, R Lalvena, an IAS officer.

Drugs 189
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Nona Biosciences and ModeX Therapeutics partner for antibodies discovery

Pharmaceutical Technology

HBM’s wholly owned subsidiary Nona Biosciences has entered an agreement with OPKO Health’s ModeX Therapeutics for the discovery of antibodies. ModeX Therapeutics will gain access to the fully human Harbour Mice platforms of Nona Bioscience for integration into its MSTAR platform to expedite the monoclonal antibodies’ discovery. This is expected to significantly shorten the preclinical antibody therapeutics development process.

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Miebo Approved as First Dry Eye Disease Treatment to Target Tear Evaporation

XTalks

Eyecare giant Bausch + Lomb received its first prescription drug approval from the US Food and Drug Administration (FDA) for its dry eye disease med Miebo (perfluorohexyloctane ophthalmic solution). The eye drops are the first and only FDA-approved treatment that directly target tear evaporation at the ocular surface to treat the signs and symptoms of dry eye disease.

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FDA approves breakthrough pneumonia therapy

Pharmaceutical Technology

Innoviva has snagged a win for its bacterial pneumonia drug Xacduro (durlobactam sodium + sulbactam) with a US Food and Drug Administration FDA approval for use in patients of ages 18 or older. The intravenous therapy has become the first pathogen-targeted therapy approved for the treatment of hospital-acquired bacterial pneumonia (HABP) and ventilator-associated bacterial pneumonia (VABP) caused by susceptible strains of bacteria called Acinetobacter baumannii-calcoaceticus complex (ABC).

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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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Innoviva subsidiary Entasis scores FDA nod for bacterial pneumonia combo drug Xacduro

Fierce Pharma

Innoviva subsidiary Entasis scores FDA nod for bacterial pneumonia combo drug Xacduro kdunleavy Wed, 05/24/2023 - 09:46

Drugs 101
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US government plans proposal to push down Medicaid drug prices

Pharmaceutical Technology

The US Department of Health and Human Services (HHS) and the Centers for Medicare & Medicaid Services (CMS) have announced steps to push down prescription drug prices under Medicaid as part of a new proposal. The potential regulation, announced on 23 May, includes steps that states can carry out to manage their drug expenditure, particularly in the context of new high-cost treatments such as cell and gene therapies.

Drugs 130
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Why Is CVS Health Clinical Trial Services Closing?

XTalks

Leading health solutions company CVS Health recently disclosed that its clinical trial business will shut down just two years after its initiation in May 2021. The company will gradually wind down its CVS Health Clinical Trial Services unit with full exit by December 31, 2024. According to the company, it is an attempt to focus on its core businesses which include retail pharmacy, health insurance, prescription drug coverage and health care and wellness. “We continually evaluate our portfo

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Y-mAbs’ Danyelza injection receives marketing approval in Brazil

Pharmaceutical Technology

Y-mAbs Therapeutics has received marketing authorisation for Danyelza (naxitamab-gqgk) 40mg/10mL injection from the Brazilian Health Regulatory Agency, Agência Nacional de Vigilância Sanitária, to treat high-risk neuroblastoma. Danyelza is a recombinant humanised monoclonal antibody that acts on the ganglioside GD2 that is highly expressed in several neuroectoderm-derived tumours and sarcomas.

Marketing 130
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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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The Manuka Honey Trademark Case, Explained

XTalks

Manuka honey, a natural product renowned for its antibacterial and potential health benefits, has been at the center of a prolonged legal dispute between Australia and New Zealand. The conflict involves the naming rights to “Manuka honey,” a product whose potential health benefits and significant price markup have made it a highly sought-after commodity in the international market.

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Inovio’s INO-3107 receives orphan drug status from EC

Pharmaceutical Technology

Inovio has received orphan drug designation for INO-3107 from the European Commission (EC) to treat recurrent respiratory papillomatosis (RRP). The investigational DNA medicine candidate INO-3107 has been designed for eliciting a targeted T cell response against HPV[human papillomavirus]-6 and HPV-11, the types of HPV that cause RRP and other related diseases.

Drugs 130
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AstraZeneca provide further Breztri evidence

Pharma Times

Vital data supports early intervention in order to treat chronic obstructive pulmonary disease

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CohBar and Morphogenesis in merger for immuno-oncology therapies

Pharmaceutical Technology

CohBar has entered into a definitive merger deal with Morphogenesis to advance a late-stage clinical immuno-oncology pipeline of therapies to overcome resistance to cancer immunotherapy. Expected to operate under the name TuHURA Biosciences, the combined company will focus on advancing two technologies of Morphogenesis that seek to overcome the major hurdles which limit existing immunotherapies’ effectiveness in treating cancer.

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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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Biopharma staffers exhausted and susceptible to burnout, report says

Fierce Pharma

Biopharma staffers exhausted and susceptible to burnout, report says kdunleavy Wed, 05/24/2023 - 16:52

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Leading innovators in recombinant viral vector-based medicinal preparations for the pharmaceutical industry

Pharmaceutical Technology

The pharmaceutical industry continues to be a hotbed of innovation, with activity driven by the evolution of new treatment paradigms, and the gravity of unmet needs, as well as the growing importance of technologies such as pharmacogenomics, digital therapeutics, and artificial intelligence. In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Recombinant viral vect

Medicine 130
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Five (or Maybe Six?) Reasons that the Largest PBMs Operate Group Purchasing Organizations

Drug Channels

In The Top Pharmacy Benefit Managers of 2022: Market Share and Trends for the Biggest Companies , I shared Drug Channels Institute's analysis showing that three pharmacy benefit managers (PBMs)—the Caremark business of CVS Health, the Express Scripts business of Cigna, and the OptumRx business of UnitedHealth Group—accounted for about 80% of total equivalent prescription claims in 2022.

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Leading innovators in tetrapeptide derivatives for the pharmaceutical industry

Pharmaceutical Technology

The pharmaceutical industry continues to be a hotbed of innovation, with activity driven by the evolution of new treatment paradigms, and the gravity of unmet needs, as well as the growing importance of technologies such as pharmacogenomics, digital therapeutics, and artificial intelligence. In the last three years alone, there have been over 633,000 patents filed and granted in the pharmaceutical industry, according to GlobalData’s report on Innovation in Pharmaceuticals: Tetrapeptide derivativ

Protein 130
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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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Celadon secures medicinal cannabis contract

Pharma Times

Celadon will sell a minimum of £3m worth of the high-THC product over the next three years

Medicine 118
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Rocket Pharmaceuticals’ gene therapy receives FDA RMAT designation

Pharmaceutical Technology

Rocket Pharmaceuticals has received Regenerative Medicine Advanced Therapy (RMAT) designation from the US Food and Drug Administration (FDA) for its gene therapy RP-L301 to treat pyruvate kinase deficiency (PKD), The investigational lentiviral-based gene therapy comprises autologous (patient-derived) hematopoietic stem cells (HSCs) genetically modified with a lentiviral vector containing a functional copy of the PKLR [pyruvate kinase-encoding] gene.

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After years of litigation, Gilead and Teva's HIV antitrust trial kicks off in California

Fierce Pharma

After years of litigation, Gilead and Teva's HIV antitrust trial kicks off in California zbecker Wed, 05/24/2023 - 17:17

Trials 101
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China accepts Junshi’s sNDA for TNBC combination therapy

Pharmaceutical Technology

China’s National Medical Products Administration (NMPA) has accepted Shanghai Junshi Biosciences ’ supplemental new drug application (sNDA) for toripalimab in combination with chemotherapy to treat advanced triple-negative breast cancer (TNBC). The anti-PD-1 monoclonal antibody toripalimab will be used along with albumin-bound paclitaxel to treat PD-L1 positive (CPS ≥ [greater than or equal to] 1) untreated metastatic or recurrent metastatic TNBC.

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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 clears Braeburn's long-acting Braxadi to treat opioid use disorder

Fierce Pharma

FDA clears Braeburn's long-acting Braxadi to treat opioid use disorder zbecker Wed, 05/24/2023 - 09:15

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Supplies of Novo Nordisk’s Wegovy starter doses to be limited

Pharmaceutical Technology

Earlier this month, Novo Nordisk revealed that they would be limiting starter doses of their obesity pharmacotherapy, Wegovy (semaglutide), in the US until September, in an effort to avoid running into supply shortages. It is an issue that the company faced in March 2022, which resulted in halted sales of Wegovy and a delay in launching in the EU. The demand for Wegovy currently outweighs supply capacity, as it is the first therapy to be approved in the obesity space that is administered on a we

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A new ABPI president; a new take on health, productivity, and partnerships – ABPI Conference part two

pharmaphorum

A new ABPI president; a new take on health, productivity, and partnerships – ABPI Conference part two Mike.

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Annexon Sets Sights on Phase III Geographic Atrophy Trial Despite Missing Mid-Stage Mark

BioSpace

Annexon Sets Sights on Phase III Geographic Atrophy Trial Despite Missing Mid-Stage Mark 5/25/2023

Trials 93
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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.