PharmaFocus Today – Page 320 – media

BioMarin’s hemophilia gene therapy Roctavian lands FDA nod with ‘glimmers’ of enthusiasm among doctors

After an initial rejection in 2020 and a review delay earlier this year, BioMarin’s Roctavian has finally got the FDA go-ahead to introduce a gene therapy for a not-so-rare disorder.The FDA has cleared Roctavian as a one-time therapy for adults with severe hemophilia A, BioMarin said Thursday. The news comes seven months after the FDA greenlighted CSL Behring’s hemophilia B gene therapy Hemgenix, which bears a list price of $3.5 million and is currently the most expensive drug in the world. BioMarin is pricing Roctavian at a wholesale acquisition cost of $2.9 million, BioMarin’s chief commercial officer Jeff Ajer said during a call Thursday. It’s also setting up an outcomes-based warranty program. The warranty will reimburse government and commercial payers up to the full cost if Roctavian doesn’t live up to its treatment expectations. Partial reimbursement will be granted if an individual loses response to the therapy in the first four ...
- Source: drugdu
- 106
- July 1, 2023
BioXcel’s Positive Alzheimer’s Agitation Data Overshadowed by Principal Investigator Fabrications

By David Adam Pictured: Person holding a cell phone in front of computer with stock data/iStock The release of positive topline results Thursday from the Phase III trial of BioXcel Therapeutics’ Alzheimer’s-related agitation treatment was overshadowed by disclosures to the SEC about fabricated email correspondences pertaining to the study’s safety data. In a Thursday SEC filing, BioXcel said that a trial site’s principal investigator—who enrolled 40% of the study participants at that site—was found to have fabricated email correspondences related to the timing of the filing for a serious adverse event (SAE) with the company’s vendor responsible for the monitoring of drug safety. The adverse event occurred in a subject in the placebo arm, the company said. In May 2023, it “came to the company’s attention that this same principal investigator in the TRANQUILITY II clinical trial may have fabricated email correspondence purporting to demonstrate that the investigator timely submitted ...
- Source: drugdu
- 108
- July 1, 2023
Alkermes Shareholders Reject All of Sarisa’s Nominees for Its Board

By Lucy Miguel Pictured: A photograph of an empty board room with laptops at each seat/iStock, ismagilov Biopharmaceutical company Alkermes announced on Thursday that, at the company’s 2023 Annual General Meeting of Shareholders, seven of Alkermes’ director nominees were re-elected. All of Sarissa Capital Management candidates, Alexander Denner, Patrice Bonfiglio, and Sarah Schlesinger, did not receive enough votes for the election. Denner, Sarissa’s founder nominee, lost just after he stepped down from the board at Biogen, where he has sat since 2009, and from an additional role on Biogen’s board nominating committee. This action was taken to remove possible conflicts of interest with Alkermes. However, his replacement at Biogen was Susan Larger, his romantic partner, a fact not disclosed to shareholders and that became an obstacle to his election. Sarissa, one of Alkermers’ largest investors since 2019, has criticized how management has been running and the stock price has performance. ...
- Source: drugdu
- 122
- July 1, 2023
Former J&J sales rep indicted on charges of defrauding Boston hospital

Dive Brief A former DePuy Synthes sales representative and team lead was arrested this week for allegedly defrauding a Boston area hospital and lying to federal authorities investigating whether he interfered with the hospital’s sterilization processes. Matthew Capobianco, 45, of Winchester, Massachusetts, was indicted on eight counts of wire fraud and one count of making material false statements, according to the U.S. Attorney’s Office in Boston. Johnson & Johnson unit DePuy Synthes, in an emailed statement, said all government claims against the company in the matter have been resolved. Dive Insight According to the Department of Justice indictment, Capobianco defrauded the hospital out of hundreds of thousands of dollars’ worth of spine products that he falsely represented as used in surgeries from January 2016 through June 2017. The Boston area hospital was not named in the DOJ’s statement. To boost his sales numbers and his compensation, Capobianco falsely represented on ...
- Source: drugdu
- 139
- June 30, 2023
Teleflex recall of separating vascular catheters labeled Class I event

Dive Brief Teleflex’s recall of its Arrow Endurance Extended Dwell Peripheral Catheter System was categorized by the U.S. Food and Drug Administration as a Class I event, the most severe type of recall. The company began the recall in May after receiving 83 complaints, including 18 reports of injuries. If the catheter separates while in a blood vessel, fragments could spread to other parts of the body and cause adverse events including heart attack and death, the FDA said in a notice. Hospitals should check if they have any of the 262,016 devices recalled in the U.S. and stop using the affected products. Teleflex is asking clinical staff overseeing patients who are currently using an affected product to weigh the risks and benefits of removing and replacing the device. Dive Insight Teleflex acquired Arrow International in 2007 to gain control of a portfolio of vascular access devices. That portfolio now ...
- Source: drugdu
- 140
- June 30, 2023
US health alert over malaria cases in Florida and Texas

BBC NEW Florida and Texas are seeing some locally acquired cases of malaria – the first spread of the mosquito-transmitted disease inside the US in 20 years, officials warn in a health alert. Active surveillance for more cases is continuing, the Centres for Disease Control says. The risk of catching malaria in the US remains extremely low, it says. All five patients – four in Florida, one in Texas – have now had treatment. Malaria is caused by being bitten by an infected mosquito. People cannot catch it from each other. But the insects catch it from infected people – and the cycle continues. It is common in large areas of Africa, Asia and Central and South America but not the US. However, Anopheles mosquitoes, found throughout many parts of the US, can transmit malaria, if they have fed on an infected person. The risk is higher in areas where: ...
- Source: drugdu
- 113
- June 30, 2023
Bayer’s BlueRock Reports Positive Early Data for Parkinson’s Stem Cell Therapy

By Heather McKenzie https://www.biospace.com/ Bayer logo on a building under blue sky/Getty Images, Kena Betancur/VIEWpress/Corbis Bayer subsidiary BlueRock Therapeutics has taken another next step in its bid to bring a potentially curative therapy to Parkinson’s disease patients. Wednesday, the companies reported that bemdaneprocel, a stem cell therapy, was well tolerated with no major side effects in a Phase I study. Topline data from the trial of 12 patients also showed feasibility of transplantation and evidence that the cells survived and engrafted in the brain after one year, satisfying the study’s secondary endpoints. Full data will be presented in August at the 2023 International Congress on Parkinson’s Disease and Movement Disorders in Copenhagen. The therapy, also known as BRT-DA01, is the first to show positive results in a Phase I study for Parkinson’s, according to Bayer. Bemdaneprocel consists of dopamine-producing neurons derived from pluripotent stem cells. In Parkinson’s disease, patients lose ...
- Source: drugdu
- 116
- June 30, 2023
Vitamin D supplements may reduce risk of serious cardiovascular events in older people

Vitamin D supplements may reduce the risk of major cardiovascular events such as heart attacks among people aged over 60, finds a clinical trial published by The BMJ. The researchers stress that the absolute risk difference was small, but say this is the largest trial of its kind to date, and further evaluation is warranted, particularly in people taking statins or other cardiovascular disease drugs. Cardiovascular disease (CVD) is a general term for conditions affecting the heart or blood vessels and is one of the main causes of death globally. CVD events such as heart attacks and strokes are set to increase as populations continue to age and chronic diseases become more common. Observational studies have consistently shown a link between vitamin D levels and CVD risk, but randomized controlled trials have found no evidence that vitamin D supplements prevent cardiovascular events, possibly due to differences in trial design that ...
- Source: drugdu
- 200
- June 30, 2023
Living near green space makes you 2.5 years younger

City parks and green spaces help counter heat, boost biodiversity, and instill a sense of calm in the urban jungle. They also help slow biological aging, with people who have access to green spaces found to be on average 2.5 years biologically younger than those who do not, according to a new study published Wednesday in Science Advances. “Living near more greenness can help you be younger than your actual age,” Kyeezu Kim, the study’s lead author and a postdoctoral scholar at Northwestern University’s Feinberg School of Medicine, told AFP. “We believe our findings have significant implications for urban planning in terms of expanding green infrastructure to promote public health and reduce health disparities.” Exposure to green spaces has previously been linked with better cardiovascular health and lower rates of mortality. It’s thought that more physical activity and social interactions are at play, but whether parks actually slowed down aging ...
- Source: drugdu
- 226
- June 30, 2023
FDA rejection sets back Regeneron’s plan to defend top-selling eye drug

Dive Brief: Regeneron Pharmaceuticals is facing a delay for a much-anticipated new version of its best-selling eye medicine after failing to win U.S. approval on Tuesday because of a manufacturing issue. The Food and Drug Administration issued a complete response letter for the high-dose version of Eylea, citing inspection findings at a third-party facility that fills vials of the medication. Regeneron said it’s working with the FDA and the third party to resolve the issues “as quickly as possible.” Importantly, the FDA did not cite any issues with the drug’s safety or efficacy and isn’t asking for new clinical trials. The agency also did not find any issues with the labeling or manufacturing of the actual drug substance, Regeneron said. Dive Insight: While the issue sounds relatively minor, any delay is significant for Regeneron, and the company’s shares tumbled around 11% during trading sessions on Tuesday and Wednesday. Eylea is ...
- Source: drugdu
- 105
- June 30, 2023

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