PharmaFocus Today – Page 251 – media

FDA Blocks Alnylam’s Bid to Expand Onpattro Label

By Tristan Manalac Pictured: Alnylam headquarters in Cambridge, Massachusetts/iStock, hapabapa The FDA on Monday denied approval of Alnylam Pharmaceuticals’ siRNA therapy Onpattro (patisiran) for the treatment of cardiomyopathy of transthyretin-mediated amyloidosis. In its Complete Response Letter (CRL), the regulator said that Alnylam had not provided enough evidence of the therapy’s benefit in the proposed indication. At the same time, the FDA did not flag any problems with patisiran’s clinical safety, drug quality, manufacturing processes or study conduct. “The CRL indicated that the clinical meaningfulness of patisiran’s treatment effects for the cardiomyopathy of ATTR amyloidosis had not been established,” according to the company’s announcement. In light of the rejection, Alnylam will no long work toward an expanded label for Onpattro in the U.S. Monday’s CRL runs counter to the recommendations of the FDA’s own panel of external advisers. In September 2023, the Cardiovascular and Renal Drugs Advisory Committee voted 9-3 in ...
- Source: drugdu
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- October 11, 2023
FDA Approves Novartis’ New Rheumatic Disease Treatment

Cosentyx is the first intravenous formulation interleukin-17A antagonist to focus on disease. Image Credit: Adobe Stock Images/Seventyfour Novartis officially announced that the FDA approved n intravenous (IV) formulation of Cosentyx (secukinumab) for the treatment of adults with psoriatic arthritis (PsA), ankylosing spondylitis (AS) and non-radiographic axial spondyloarthritis (nr-axSpA). Currently, Cosentyx is the only treatment approved in an IV formulation that specifically targets and blocks interleukin-17A (IL-17A), and the only non-tumor necrosis factor alpha (TNF-α) IV option available in all these indications. “At Novartis, we are committed to ensuring healthcare providers and patients have treatment options available to meet their unique needs. With this approval of Cosentyx as an IV formulation, along with the subcutaneous formulation, we can broaden the use of Cosentyx to help more patients manage their condition with a medicine backed by more than a decade of clinical research and eight years of real-world experience,” said Christy Siegel, ...
- Source: drugdu
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- October 11, 2023
FDA Rejection Is a Delay, Not a Detour for Alnylam’s Aim to Treat Heart Disorder

Despite an affirmative FDA advisory committee vote, the agency declined to approve Alnylam Pharmaceuticals’ Onpattro for treating the heart complications caused by a rare, inherited protein disorder. But Alnylam has other drugs candidates for the disease, including one expected to post Phase 3 data in the first half of 2024. By FRANK VINLUAN An Alnylam Pharmaceuticals drug developed to treat a rare disease’s potentially fatal effects on the heart has fallen short in its bid for FDA approval, costing the company a chance to immediately challenge a Pfizer product that has become a blockbuster seller in the indication. But the biotech still has another chance with a different drug in late-stage development that could offer patients a better alternative. Alnylam was seeking approval of its drug, Onpattro, as a treatment for cardiomyopathy caused by hereditary transthyretin amyloidosis (hATTR). According to the Cambridge, Massachusetts-based company, the FDA’s complete response letter said ...
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analysts

Dive Brief Johnson & Johnson’s spinoff of its consumer health business has positioned the company to achieve top-tier medtech growth and profitability, according to analysts at RBC Capital Markets. J&J recently split off its consumer health unit to make a standalone company, turning itself into the only global healthcare company with pharma and medtech in a single portfolio. RBC analysts expect the new structure to support the growth of a “bellwether” healthcare stock. On the medtech side, the analysts identified J&J’s leadership position in 11 categories, shift to fast-growing markets, investment in robotics and capacity for M&A as factors that position it to outperform its peers. Dive Insight The RBC analysts praised J&J’s current medtech portfolio, noting that the company is a leader in 11 categories and has 12 platforms with sales of more than $1 billion. The portfolio is the result of a “clear winning strategy,” they wrote in ...
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- October 11, 2023
Compound found to inhibit influenza virus replication by targeting host enzyme

How can the propagation of influenza viruses be stopped? For a new approach in the therapy of influenza infections, Prof. Hiroki Kato from the Institute of Cardiovascular Immunology at the University Hospital Bonn (UKB) and the Cluster of Excellence ImmunoSensation2 of the University of Bonn receives an Open Philanthropy grant of 2.2 million US dollars. Together with his team, he found a compound that inhibits the body’s own methyltransferase MTr1 and thus prevents the replication of influenza viruses. The funded project now aims to identify further MTr1 inhibitors with influenza-inhibiting activity that could be considered for clinical trials in the near future. When a virus enters our body, it binds to the host cell and introduces its genetic information in form of ribonucleic acid (RNA). Using these blueprints, the host cell is now forced to produce numerous new viruses. “This is because viruses have evolved various mechanisms, including modifications of ...
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- October 11, 2023
We’re Going To Find Out if Blue Shield of California’s New Drug Model Will Lower Costs

Blue Shield of California recently launched a new experiment for 2025: It will largely cut out CVS Caremark as its pharmacy benefit manager and will instead put the company in charge of just specialty pharmacy services. Meanwhile, Amazon Pharmacy will be in charge of home delivery of prescription drugs, Mark Cuban Cost Plus Drug Company will create a more transparent pricing model, Abarca will handle the payment of prescription drug claims and Prime Therapeutics will negotiate savings with drug manufacturers. Will this experiment of distributing pharmacy benefit responsibilities to different vendors work? Remains to be seen, said Creagh Milford, senior vice president of retail health at CVS Health, during a panel discussion on Sunday at Engage at HLTH in Las Vegas. CVS Health owns CVS Caremark. “The problem with that generally — and not saying this specific example [of California] — is that you actually see costs go up because ...
- Source: drugdu
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- October 10, 2023
WHO experts recommend the use of Takeda’s dengue vaccine QDENGA

Takeda today announced that the World Health Organization’s (WHO) Strategic Advisory Group of Experts (SAGE) on Immunization shared recommendations for use of QDENGA® (Dengue Tetravalent Vaccine [Live, Attenuated]) (TAK-003). In the coming months, the WHO will consider the SAGE recommendation and update its position paper on dengue vaccines to include final guidance on the use of QDENGA in public vaccination programs. SAGE made the following recommendations: The vaccine to be considered for introduction in settings with high dengue disease burden and high transmission intensity to maximize the public health impact and minimize any potential risk in seronegative persons. The vaccine to be introduced to children aged 6 to 16 years of age. Within this age range, the vaccine should be introduced about 1-2 years prior to the age-specific peak incidence of dengue-related hospitalizations. The vaccine should be administered in a 2-dose schedule with a 3-month interval between doses. The vaccine ...
- Source: drugdu
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Takeda to Pull Lung Cancer Drug from Market After Failed Confirmatory Study

Takeda Pharmaceutical drug Exkivity failed the confirmatory study required of its 2021 accelerated approval. Our recap of other recent regulatory developments includes a partial clinical hold on a cancer drug, a Covid-19 vaccine authorization, and several drug approvals in the U.S. and beyond. By FRANK VINLUAN Accelerated approval offers a way to more quickly bring patients drugs for diseases that have few treatment options. But that speedy path to the market comes with the understanding that the FDA can take its regulatory blessing away—unless the company takes its drug away first. That is what Takeda Pharmaceutical has decided to do with its cancer drug, Exkivity. Following discussions with the FDA, the Japanese pharmaceutical giant is voluntarily withdrawing Exkivity from the market. Exkivity treats non-small cell lung cancer by targeting epidermal growth factor (EGFR). That cancer protein must have exon 20 mutations—the same genetic signature addressed by Johnson & Johnson’s Rybrevant. ...
- Source: drugdu
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- October 10, 2023
FDA wants more testing in Philips recall

BY SEAN WHOOLEY The FDA has issued a communication stating its desire for more testing in the massive respiratory device recall at Philips (NYSE: PHG)+.The morning after the FDA’s evening announcement, PHG shares were down more than 7% on the NYSE to $18.31 apiece. PHIA shares in Amsterdam closed the day down more than 8%. (MassDevice’s MedTech 100 Index was up slightly.) Philips has been handling a Class I recall of certain respiratory devices since mid-2021. Respironics recalled millions of ventilators, bi-level positive airway pressure (BiPAP) and continuous positive airway pressure (CPAP) machines. Here’s a timeline of the events of the ongoing saga. FDA’s statement follows a scathing investigational report from ProPublica and the Pittsburgh Post-Gazette. The report outlined the alleged withholding of information around the issues with the devices even as they became more alarming. “The FDA remains unsatisfied with the status of this recall, and we continue to ...
- Source: drugdu
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- October 10, 2023
Roche presents positive results for spinal muscular atrophy treatment Evrysdi in babies

Roche has presented positive results from an ongoing study evaluating its spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam) in babies aged from birth to six weeks with pre-symptomatic disease. SMA is a severe and progressive neuromuscular disease affecting approximately one in every 10,000 babies. Those living with the condition have insufficient levels of the SMN protein, which is essential to the function of nerves that control muscles and other functions such as swallowing, speaking, breathing and movement. Roche’s Evrysdi, which can be administered at home in liquid form by mouth or by feeding tube, is designed to treat the disease by increasing and sustaining the production of SMN protein in the central nervous system and peripheral tissues. Clinical studies have shown that the loss of motor neurons may begin before symptoms start, so beginning treatment early is seen as critical for improved outcomes. Results from the ongoing RAINBOWFISH study, which ...
- Source: drugdu
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- October 10, 2023

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