PharmaFocus Today – Page 241 – media

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

Pharmaceutical Executive Editorial Staff FDA approval of bluebird bio’s Lyfgenia and Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Casgevy marks significant milestone in the treatment of sickle cell disease. The FDA has approved a pair of landmark treatments for sickle cell disease (SCD) in patients 12 years of age and older, marking the first cell-based gene therapies for SCD. As part of today’s regulatory action, both bluebird bio’s Lyfgenia (lovotibeglogene autotemcel [lovo-cel]) and Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel [exa-cel]) gained approval for the inherited blood disorders, which affect approximately 100,000 people in the United States. “Sickle cell disease is a rare, debilitating and life-threatening blood disorder with significant unmet need, and we are excited to advance the field especially for individuals whose lives have been severely disrupted by the disease by approving two cell-based gene therapies today,” Nicole Verdun, MD, director of the Office of Therapeutic Products within the ...
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- December 12, 2023
On Heels of ASH Data, Pfizer Eyes FDA Submission for New Hemophilia Drug

Pfizer already reported its experimental drug for hemophilia A and B met a pivotal study’s goals of reducing bleeding episodes. During the annual meeting of the American Society of Hematology, the company presented a full look at the results, which are expected to support regulatory submissions soon. By FRANK VINLUAN Patients with hemophilia A or B can manage the inherited bleeding disorders with regular infusions of the clotting proteins they lack. This treatment helps—until it doesn’t. One problem with these infused factor replacement therapies is patients can develop inhibitors, which are antibodies that render the clotting proteins ineffective. Experimental Pfizer drug marstacimab takes a different approach to both forms of hemophilia. Results from a pivotal study show the subcutaneously injected drug met the main goals of reducing bleeding episodes. The company also has some additional long-term data showing continuing improvement beyond the initial clinical trial evaluation period. The full Phase ...
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- December 12, 2023
BD receives 510(k) clearance for fingerstick blood test sample collection device

Dive Brief Becton Dickinson has received 510(k) clearance for a device that collects blood from a fingerstick instead of from the vein. The MiniDraw Capillary Blood Collection System container is slightly larger than its predicate device, the BD Microtainer MAP Microtube, but is cleared for sample collection in “ancillary healthcare facilities,” positioning BD and its partner Babson Diagnostics to support blood collection from community sites such as pharmacies. When Babson rolls out its BetterWay blood testing service next year, the device could support the collection of samples for lipid panel, selected chemistry tests, and hemoglobin and hematocrit analyses. Dive Insight BD began working with Babson to develop a capillary blood collection and testing system in 2019. The partners expanded their collaboration last year to support work on self-collection, mobile services and at-home collection. Devices that enable the collection of capillary, rather than venous, blood for tests of hemoglobin and hematocrit ...
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- December 12, 2023
Discovery of Rogue Protein Paves Way for Diagnostic Test to Detect Early-Onset Dementia

Neurodegenerative disorders are often marked by the buildup of amyloid filament inclusions of specific proteins in the brain. These proteins are critical in diagnosing and treating the associated diseases. Frontotemporal lobar degeneration (FTLD) is one such disorder, leading to frontotemporal dementia, which ranks just behind Alzheimer’s disease in prevalence. In about 10% of FTLD cases, the identity of the filament-forming protein was previously unknown. The protein FUS was the suspected culprit, given its presence in brain inclusions and its known genetic role in some instances of amyotrophic lateral sclerosis, another neurodegenerative condition. Now, researchers using electron cryo-microscopy have unexpectedly found that the filaments are instead formed by the protein TAF15. The research team from MRC Laboratory of Molecular Biology (Cambridge, UK) extracted amyloid filaments from the brains of four individuals affected by this type of FTLD. They carried out neuropathological examinations on the brain tissues obtained. Employing electron cryo-microscopy, they ...
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- December 12, 2023
Edwards plans to spin off critical care business next year

BY SEAN WHOOLEY Edwards Lifesciences (NYSE: EW)+ announced today that it intends to spin off its critical care business by the end of 2024. The company shared a number of key plans going forward as it outlined its plans for future growth. Among its plans, Edwards expects to spin off the critical care unit and its full range of smart monitoring technologies next year. “During 2024, Edwards plans to support the growth and leadership of innovations in advanced patient monitoring, with the goal of improving the quality of care for millions of patients annually,” Edwards wrote in a news release. Analysts expect shares of Edwards to move up on the news today. Shares of EW rose 1% at $70.17 apiece in early-morning trading today. MassDevice’s MedTech 100 Index — which includes stocks of the world’s largest medical device companies — remained even. Edwards intends to complete the spin-off tax-free. The ...
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- December 11, 2023
How to Choose the Right Distribution Partner

By understanding the interconnectedness of the healthcare industry, aligning goals and maintaining an open line of communication, distributors and healthcare organizations can cultivate lasting, dependable partnerships, while working together to develop innovative ideas and solutions. By MARC DELORENZO From manufacturing pharmaceutical and medical products to distributing them to the patients who need them, the healthcare supply chain is extensive, complex and constantly evolving. From regulatory shifts to changing provider needs, it’s more important than ever that providers forge strong partnerships with their distributors. When choosing the right distribution partner for your organization, it’s important to consider the foundation of what that relationship should look like. The distributor’s mission and values should align with your institution’s to create a solid basis for a lasting partnership. Particularly important for the healthcare field, the right distribution partner should be collaborative, transparent and adaptable, with the ability to align their services to meet the ...
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- December 11, 2023
Vanda expands commercial presence with $100M deal for US rights to J&J multiple sclerosis drug

Vanda Pharmaceuticals has a new commercial asset under its belt after striking a deal to buy certain rights to Johnson & Johnson’s multiple sclerosis med Ponvory for $100 million. Vanda secured U.S. and Canadian rights to the daily oral selective sphingosine-1-phosphate receptor 1 (S1P1R) modulator from J&J’s Actelion subsidiary. Ponvory was approved in 2021 to treat relapsing forms of multiple sclerosis and could be a potential treatment for a group of inflammatory and autoimmune disorders, including psoriasis and ulcerative colitis, Vanda said in its Thursday press release. The buy is a “significant milestone” for Vanda, CEO and chairman Mihael H. Polymeropoulos, M.D., said in a statement. The deal “expands our commercial portfolio and gives us access to a versatile immune response modifier that can potentially have broad application in treating a number of autoimmune-based disorders,” the chief added. J&J’s sale of certain Ponvory rights comes after Fierce Pharma in February ...
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- December 11, 2023
FDA fast tracks Solid Biosciences’s Duchenne gene therapy

The FDA has granted fast track designation to Solid Biosciences’s gene therapy SGT-003 for the treatment of DMD. The US-based company received clearance from the FDA to start Phase I/II trial for the gene therapy on 14 November. SGT-003 uses a novel capsid AAV-SLB101 to deliver the DNA sequence encoding the shortened form of the dystrophin protein (microdystrophin). It also has R16 and R17 nNOS binding protein domains. DMD is a rare genetic condition that causes progressive muscle weakness. It affects approximately six in 100,000 individuals across Europe and North America, as per the US Muscular Dystrophy Association. The first gene therapy for treating DMD was approved earlier this year. In June, Sarepta Therapeutics’s gene therapy Elevidys (delandistrogene moxeparvovec-rokl) received accelerated approval by the FDA for treating ambulatory patients aged 4-5 years with a confirmed mutation in the DMD gene. There has been an increased interest in developing gene therapies ...
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- December 11, 2023
Roche shares positive late-stage results for inavolisib combination in advanced breast cancer

Roche has shared positive results from a late-stage study of its investigational therapy, inavolisib, as part of a combination treatment for advanced breast cancer. The phase 3 INAVO120 trial has been evaluating the targeted drug alongside Pfizer’s CDK4/6 inhibitor Ibrance (palbociclib) and fulvestrant as a potential first-line treatment option for patients with PIK3CA-mutated, hormone receptor (HR)-positive, HER2-negative, endocrine-resistant, locally advanced or metastatic breast cancer. The study met its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement in progression-free survival compared to palbociclib and fulvestrant alone. A “clear positive trend” towards improved survival was also observed for the inavolisib combination, Roche said, although overall survival data was immature at the time of the analysis and will continue to be analysed. Breast cancer is the most common cancer worldwide, with more than 290,000 people in the US expected to be diagnosed with the disease in 2023. HR-positive breast cancer is ...
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- December 11, 2023
Study identifies new amyloid-forming protein linked to early-onset dementia

A new study led by the Medical Research Council’s (MRC) Laboratory of Molecular Biology (LMB) has identified a new protein as a potential therapeutic target for frontotemporal dementia, a type of early-onset dementia. Dementia is a progressive neurodegenerative disorder that affects more than 944,000 people in the UK. Frontotemporal dementia occurs from the degeneration of the frontal and temporal lobes of the brain, which control emotions, personality and behaviour, along with speech. Most neurodegenerative diseases involve proteins aggregating into filaments known as amyloids. When identified, researchers can target these proteins for diagnostic tests and treatments. However, in around 10% of frontotemporal dementia cases, researchers had to identify the TAF15 protein. Published in Nature, MRC LMB researchers successfully pinpointed the aggregated structures of the protein using cutting-edge cryo-electron microscopy to study the brains of four patients who had this type of early-onset dementia. The FUS protein is already known to be ...
- Source: drugdu
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- December 11, 2023

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