PharmaFocus Today – Page 319 – media

FDA approves Duchenne gene therapy with hefty $3.2m price tag

Children with a confirmed mutation of the DMD gene – responsible for causing Duchenne muscular dystrophy (DMD) – are closer to receiving the first gene therapy for the disease after the US Food and Drug Administration (FDA) approved Sarepta’s highly anticipated Elevidys (delandistrogene moxeparvovec-rokl). On a conference call following the approval, Sarepta CEO Douglas Ingram said the company has set the wholesale acquisition cost of Elevidys at $3.2m. After a narrow US Food and Drug Administration Advisory Committee (AdCom) panel vote and a delay to the Prescription Drug User Fee Act (PDUFA) date, Sarepta’s therapy crossed the approval finish line. The decision, which came through the accelerated approval pathway, is for children ages four to five with a confirmed mutation in the DMD gene. As per Sarepta, Elevidys’ price is below the cost-effectiveness range, which is estimated to be between $5m and $13m. Sarepta’s adeno-associated virus (AAV) mediated therapy delivers ...
- Source: drugdu
- 99
- June 25, 2023
Treatment for opioid use disorder varies widely among states, study finds

Despite a national opioid overdose epidemic supercharged by a surge of illicit fentanyl, new research from Oregon Health & Science University reveals wide discrepancies among U.S. states in effectively treating opioid use disorder among people covered by Medicaid. The study, published in the journal JAMA Health Forum, found that in many states, fewer than half of people diagnosed with opioid use disorder received proven medications to treat it. “We fail people by not providing adequate treatment to people with opioid use disorder enrolled in Medicaid,” said lead author Stephan Lindner, Ph.D., associate professor in the OHSU Center for Health Systems Effectiveness. Medicaid provides health care coverage to more than 90 million Americans. Evidence strongly suggests that medication should be nearly universal treatment for people with opioid use disorder, said co-author Dennis McCarty, Ph.D., professor emeritus of public health in the OHSU School of Medicine and the OHSU-Portland State University School ...
- Source: drugdu
- 88
- June 25, 2023
Study reports drug that significantly reduces bacteria’s ability to develop antibiotic resistance

A team of researchers at Baylor College of Medicine is gaining ground in their search for solutions to the global problem of bacterial antibiotic resistance, which was responsible for nearly 1.3 million deaths in 2019. The team reports in the journal Science Advances a drug that, in laboratory cultures and animal models, significantly reduces the ability of bacteria to develop antibiotic resistance, which might prolong antibiotic effectiveness. The drug, called dequalinium chloride (DEQ), is a proof-of-concept for evolution-slowing drugs. “Most people with bacterial infections get better after completing antibiotic treatment, but there are also many cases in which people decline because the bacteria develop resistance to the antibiotic, which then can no longer kill the bacteria,” said corresponding author Dr. Susan M. Rosenberg, Ben F. Love Chair in Cancer Research and professor of molecular and human genetics, biochemistry and molecular biology and molecular virology and microbiology at Baylor. She also ...
- Source: drugdu
- 100
- June 25, 2023
New study finds that plant extracts used by Indigenous people hold promise in treatment of ataxia

A University of California, Irvine-led team of researchers has discovered that extracts from plants used by the Kwakwaka’wakw First Nations peoples in their traditional botanical medicine practices are able to rescue the function of ion channel proteins carrying mutations that cause human episodic ataxia. The study, “Native American ataxia medicines rescue ataxia-linked mutant potassium channel activity via binding to the voltage sensing domain” was published in June in Nature Communications. “Episodic ataxia 1 (EA1) is a movement disorder caused by inherited mutations in the human KCNA1 gene, which encodes Kv1.1, a voltage-gated potassium channel essential for normal function of the human nervous system,” said Geoffrey W. Abbott, Ph.D., vice dean of basic science research and professor in the Department of Physiology & Biophysics at the UCI School of Medicine. “We found that extracts of stinging nettle, bladderwrack kelp and Pacific ninebark can all correct function of the mutation-carrying proteins causing ...
- Source: drugdu
- 110
- June 23, 2023
Omega-3 fatty acids linked with slower progression of ALS

Consuming omega-3 fatty acids—particularly alpha-linolenic acid (ALA), a nutrient found in foods including flaxseeds, walnuts, and chia, canola and soybean oils—may help slow the progression of disease in patients with amyotrophic lateral sclerosis (ALS), according to a new study led by Harvard T.H. Chan School of Public Health. The study is published on June 21, 2023, in Neurology. “Prior findings from our research group have shown that a diet high in ALA and increased blood levels of this fatty acid may decrease the risk of developing ALS. In this study, we found that among people living with ALS, higher blood levels of ALA were also associated with a slower disease progression and a lower risk of death within the study period,” said lead author Kjetil Bjornevik, assistant professor of epidemiology and nutrition. “These findings, along with our previous research, suggest that this fatty acid may have neuroprotective effects that could ...
- Source: drugdu
- 125
- June 23, 2023
Empress Therapeutics emerges with $50m to fund small molecule development

Life sciences venture capital firm Flagship Pioneering has unveiled Empress Therapeutics, a small molecule drug development startup. The Moderna-backed venture fund has launched Empress with a $50m investment. This will support development of its proprietary Chemilogics platform and drug discovery pipeline. So far, Empress claims to have generated 15 drug leads across multiple indications. These span multiple structural classes and target several classes of proteins, including cytokines, enzymes, G protein-coupled receptors (GPCRs), and ion channels. With this pipeline, Empress hopes to address unmet clinical needs in immune and inflammatory conditions, and metabolic, neurologic, oncologic, and pain disorders. The company expects to file multiple IND applications in these areas over the next couple of years. Empress benefits from a wealth of clinical data, and recent advances in genomics, artificial intelligence (AI) and synthetic biology. This allows the company’s platform to read genetic code and use biosynthetic instructions to find, make and ...
- Source: drugdu
- 118
- June 23, 2023
Calliditas files sNDA to US FDA for TARPEYO to treat primary IgAN

Calliditas Therapeutics has submitted a supplemental new drug application (sNDA) to the US Food and Drug Administration (FDA) for complete approval of TARPEYO (budesonide). The TARPEYO delayed-release capsule is a corticosteroid indicated for reducing proteinuria to treat primary immunoglobulin A nephropathy (IgAN) in adult patients at risk of quick disease progression, generally having a urine protein-to-creatinine ratio greater than or equal to 1.5g/g. The therapy received accelerated approval based on showing a reduction in proteinuria. The submission of sNDA to the regulator was based on the findings obtained from the Phase III NefIgArd clinical trial. The multicentre, randomised, double-blind trial was designed to evaluate the safety and efficacy of TARPEYO 16 mg compared with placebo against a background of optimised RASi (renin–angiotensin system inhibitor) therapy in primary IgA adult patients. TARPEYO showed a highly statistically significant benefit in estimated glomerular filtration rate (eGFR) over a two-year period of treatment. The ...
- Source: drugdu
- 121
- June 23, 2023
ADVANZ PHARMA and Veeva Systems link for digital operations in Europe

ADVANZ PHARMA has collaborated with Veeva Systems to set up a unified digital-first commercial foundation in Europe. ADVANZ intends to drive a unified field strategy across all markets and strengthen field engagement with healthcare professionals (HCPs) using Veeva Commercial Cloud, a family of data, software and services for advancing commercial excellence in life sciences. ADVANZ is also using Veeva OpenData and Veeva CRM Suite to consolidate its operations and to gain a clear understanding of customers throughout the healthcare ecosystem, with a focus on products within oncology, anti-infectives, critical care, endocrinology and rare diseases. This allows the company’s newly merged field teams to coordinate more effectively with HCP touchpoints across channels and regions. Veeva Europe commercial strategy vice-president Philipp Luik stated: “With Veeva Commercial Cloud, ADVANZ now has a digital foundation to sustain growth as it expands operations in Europe. “By reaching the right people sooner and leading more impactful ...
- Source: drugdu
- 114
- June 23, 2023
SFDA approves Amarin’s VASCEPA to reduce CV risk in Saudi Arabia

The Saudi Food and Drug Authority (SFDA) has granted approval to Amarin’s VASCEPA (icosapent ethyl) capsules for reducing cardiovascular (CV) risk. VASCEPA is indicated as an adjunct to statin therapy for use in adult patients with elevated levels of triglycerides (TG). This means levels greater than or equal to 150 mg/dL. It has also been approved for use in patients at increased risk of CV events caused by at least one other CV disease risk factor and established CV disease, or who have diabetes mellitus. The therapy is the first and only medicine to receive approval from the regulator to reduce CV risk beyond cholesterol-lowering therapy in patients on high-risk statin treatment and who have increased TG levels. Amarin stated that the country’s National Heart Center (NHC) and the Saudi National Diabetes Center (SNDC) have mentioned icosapent ethyl (IPE) as an adjunct to statin therapy for CV risk reduction in ...
- Source: drugdu
- 127
- June 23, 2023
Philips gets slapped with two legal challenges by French patients in ‘Respiratorgate’

Dive Brief A lawyer has filed civil and criminal complaints against Philips in France over its recalled respiratory medical devices. The civil complaint covers 1,341 claimants who are seeking compensation for non-material damages tied to Philips’ failure to inform them of the danger the devices may present and for “anxiety-related harm.” A related criminal complaint was lodged by 215 people who accuse Philips of endangering the lives of others, aggravated deception and administration of harmful substances. Dive Insight France has emerged as a hotspot for action against Philips. Last year, the French device regulator ordered Philips to repair or replace all of its recalled respiratory devices by the end of 2022. When Philips missed the deadline, the agency applied more pressure to the company and referred the case to a prosecutor who could bring criminal proceedings. In parallel, lawyers behind a collective legal action platform have been preparing cases against ...
- Source: drugdu
- 198
- June 23, 2023

your submission has already been received.

OK

Subscribe

Please enter a valid Email address！

Submit

The most relevant industry news & insight will be sent to you every two weeks.

more

more

Customer Services
Contact Us
Help Center

Buy on Drugdu.com
Browse Product Categories

Free APP

Follow Us

Terms & Conditions | Privacy Policy

粤ICP备16008861号

Copyright © 2016-2018 Drugdu.com. All Rights Reserved.