A US Food and Drug Administration’s (FDA) panel of experts has recommended Sarepta Therapeutics’ Duchenne muscular dystrophy (DMD) investigational gene therapy for accelerated approval. The Cellular, Tissue and Gene Therapies advisory committee voted eight to six in support of SRP-9001 (delandistrogene moxeparvovec) as a treatment for ambulatory DMD patients with a confirmed mutation in the DMD gene. DMD is a rare and inherited X-chromosome-linked disease that results in the lack of dystrophin protein, which is required to strengthen and protect muscles. Over time, this causes progressive loss of muscle strength, with most patients requiring full-time use of a wheelchair by their early teens. Eventually, increasing difficulty in breathing due to respiratory muscle dysfunction requires ventilation support, and cardiac dysfunction can lead to heart failure. The disease almost exclusively affects males, occurring in approximately one in every 3,500-5,000 newborn males worldwide. SRP-9001 is designed to address the underlying cause of DMD ...
Melinta Therapeutics and Xediton Pharmaceuticals have entered an exclusive commercialisation and licensing agreement for four novel anti-infective products. The products include Baxdela (delafloxacin), Vabomere (meropenem and vaborbactam), Orbactiv (oritavancin), and Kimyrsa (oritavancin). Under the terms of the deal, Xediton Pharmaceuticals will handle the registration and commercialisation works of these products in Canada. Melinta Therapeutics president and CEO Christine Ann Miller said: “Xediton shares our commitment to ensure that all patients who need our life-saving therapies can get them. “We are confident that Xediton brings the right amount of energy and experience to effectively bring our novel anti-infective portfolio to market throughout this region.” Baxdela is indicated to treat acute bacterial skin and skin structure infections (ABSSSI) and community-acquired bacterial pneumonia (CABP), which is caused by designated susceptible bacteria in adult patients. Vabomere will be used to treat complicated urinary tract infections (cUTI), including pyelonephritis, that are caused by the susceptible ...
By Brenda Goodman, CNN CNN — Pfizer’s vaccine to protect newborns from respiratory syncytial virus, or RSV, by vaccinating their moms late in pregnancy cuts the risk that infants will need to see a doctor or be admitted to the hospital with a moderate to severe infection before 6 months of age, according to a new analysis by government regulators. Many parents have been eagerly anticipating this news, particularly after last year’s long and severe RSV season. RSV is a major cause of hospitalization in infants and the elderly each year, and it typically hits hardest during the winter months. Scientists have been working on an effective RSV for young children for roughly 60 years, so there’s plenty of excitement around the prospect of having a candidate get so close to the finish line. But the news isn’t all rosy. Safety data published in an agency analysis Tuesday also showed ...
Patients are now enrolling in an early stage clinical trial to test a universal flu vaccine based on messenger RNA technology, the National Institutes of Health announced Monday. Scientists hope the vaccine will protect against a wide variety of flu strains and provide long-term immunity so people do not have to receive a shot every year. Messenger RNA, or mRNA, is the technology behind Moderna ’s and Pfizer ’s widely used Covid vaccines. NIH played a crucial role in developing the mRNA platform used by Moderna. “A universal flu vaccine could serve as an important line of defense against the spread of a future flu pandemic,” Dr. Hugh Auchincloss, acting director of National Institute of Allergy and Infectious Diseases, said in a statement Monday. The universal flu vaccine trial will enroll up to 50 healthy people ages 18 through 49 to test whether the experimental shot is safe and produces ...
In a recent study published in the journal Eurosurveillance, researchers performed whole genome sequencing (WGS) on 874 Escherichia coli (E. coli) isolates carrying the bla NDM-5 gene, which encodes New Delhi metallo-β-lactamase (NDM)-5. They retrieved the study sample set from 13 European Union (EU)/European Economic Area (EEA) countries between 2012 and June 2022. Background In a survey of carbapenem- and/or colistin-resistant Enterobacterales (CCRE survey) performed across 36 European countries in 2019, researchers detected 62 of 201 carbapenemase-producing E. coli isolates carrying NDM-5 encoding gene ‘blaNDM-5’ in 15 countries. Additionally, they observed that some of these 62 E. coli isolates belonged to high-risk sequence types (STs), which increased the risk of extraintestinal infections. Since these findings were concerning, the European Centre for Disease Prevention and Control (ECDC) requested further investigation into the matter. About the study In the present study, researchers initiated the collection of WGS and epidemiological data on bla ...
Months after the U.S. and European approvals of its CSL-partnered hemophilia A gene therapy Hemgenix, uniQure is selling some royalty rights for up to $400 million. In a deal with HealthCare Royalty (HCRx) and Sagard Healthcare, uniQure agreed to part ways with a portion of its Hemgenix royalty rights. The company previously licensed the drug to Australia’s CSL Behring, and it’s entitled to milestone and royalty payments as the launch progresses. Now, though, it’s trading some of those royalty rights for a quick payday. Under the new deal with HCRx and Sagard Healthcare, uniQure will get $375 million upfront and another $25 million if an undisclosed sales threshold is achieved. The cash lets uniQure “continue to invest” in its adeno-associated virus vector gene therapy pipeline, CEO Matt Kapusta said a statement. The pipeline includes a Huntington’s disease prospect, a refractory temporal lobe epilepsy candidate and an SOD-1 amyotrophic lateral sclerosis ...
As more people use cannabis for recreational purposes, attitudes towards the drug have changed. For example, research has shown that dispensaries often recommend cannabis—also referred to as marijuana—to pregnant women to ease pregnancy symptoms, especially morning sickness. There is a growing body of literature attesting to poor child outcomes if cannabinoids are consumed during pregnancy. The exact effects on the developing fetus, however, remain unclear. Researchers in the US have now examined how timing of cannabis exposure during pregnancy impacts fetal development. “We show that even when marijuana use occurred only in the first trimester of pregnancy, birth weight was significant reduced, by more than 150g on average,” said Dr. Beth Bailey, professor and director of population health research at Central Michigan University and senior author of the study published in Frontiers in Pediatrics. “If that use continued into the second trimester, newborn head circumference was significantly decreased as well.” ...
Novartis has taken another step toward spinning off its generics and biosimilars business Sandoz, identifying eight of its new board of directors.The group includes Nestle chief financial officer Francois-Xavier Roger and former Eli Lilly chief information and digital officer Aarti Shah, Ph.D. They are among eight approved by Novartis after being recommended by Gilbert Ghostine, who was designated as Sandoz’s chairman of the board three months ago. Ghostine been the CEO of Swiss perfume and taste company Firmenich since 2014. Other new members of the board—which will begin prep work for the spinoff—include Urs Riedener, formerly the CEO of Swiss dairy company Emmi; Remco Steenburgen, the CFO at Deutsche Lufthansa AG; and Yannis Skoufalos, a former executive at Proctor & Gamble. In addition to Shah, the board will have three other women including Shamiram Feinglass, M.D., formerly the VP of medical affairs and policy at Danaher; Maria Varsellona, J.D., the ...
Advanced or metastatic estrogen receptor-positive (ER+) breast cancer is commonly treated with drugs that block the estrogen receptor. However, estrogens that stimulate the receptor can also be effective. Building on their previous studies, researchers at Dartmouth Cancer Center recently concluded a Phase II clinical trial aimed to test the efficacy of alternating between estrogen stimulation and estrogen deprivation in patients with metastatic ER+ breast cancer, and to identify tumor characteristics that predict who might benefit from this strategy. The results, newly published ahead of print in Clinical Cancer Research, support cyclical estrogen/anti-estrogen therapy as a promising strategy to treat advanced/metastatic ER+ breast cancer. The POLLY trial stands for “Phase II study of Pre-emptive oscillation of ER activity levels through alternation of estradiol/anti-estrogen therapies prior to disease progression in ER+/HER2- metastatic or advanced breast cancer.” Among 19 patients enrolled in the trial, 3 (16%) experienced tumor shrinkage during cyclical treatment and ...
Marilynn Larkin Editor’s note: Find the latest long COVID news and guidance in Medscape’s Long COVID Resource Center. Hyperbaric oxygen therapy (HBOT) increased markers of heart function in a small randomized, controlled trial of patients with long COVID. Patients with reduced left ventricular global longitudinal strain (GLS) at baseline who received HBOT had a significant increase in GLS compared with those who received sham treatment. GLS is a measure of systolic function that is thought to be a predictor of heart failure-related outcomes. The study also showed that global work efficiency (GWE) and the global work index (GWI) increased in HBOT-treated patients, though not significantly. “HBOT is an effective treatment for diabetic foot ulcers, decompression sickness in divers, and other conditions, such as cognitive impairment after stroke,” Marina Leitman, MD, of the Sackler School of Medicine, Tel Aviv, Israel, told theheart.org | Medscape Cardiology. Her team also studied HBOT in ...
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