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U.S. Approves First Gene-Editing Treatment, Casgevy, for Sickle Cell Disease
The U.S. Food and Drug Administration on Friday approved the country’s first gene-editing treatment, Casgevy, for use in patients with sickle cell disease.
The approval comes about a decade after the discovery of CRISPR technology for editing human DNA, representing a significant scientific advancement. Yet reaching the tens of thousands of people who could benefit from the treatment could be challenging given the potential hurdles — including cost — of administering the complex therapy.
Casgevy, co-developed by Vertex Pharmaceuticals and CRISPR Therapeutics, uses Nobel Prize-winning technology CRISPR to edit a person’s genes to treat disease. The treatment was approved by U.K. regulators last month.
Casgevy uses CRISPR to make an edit to a person’s DNA that turns on fetal hemoglobin, a protein that normally shuts off shortly after birth, to help red blood cells keep their healthy full-moon shape. In clinical trials, Casgevy eliminated pain crises in most patients.
The FDA approved the treatment for people 12 years and older.
New $1 Billion Plan for African Vaccine Manufacturing — GAVI Alliance
Up to $1 billion will be available to boost African vaccine manufacturing as part of a new scheme set up by Gavi, the Vaccine Alliance, the global health organization said on Thursday.
The “African Vaccine Manufacturing Accelerator” aims to address the inequality in access to vaccines that plagued the continent during the COVID-19 pandemic, as well as to use domestically-produced shots to tackle diseases that kill hundreds of thousands of African children every year, such as cholera and malaria.
The funding for the accelerator comes from leftover money in the COVAX initiative, a scheme set up during the pandemic to help vaccines reach the world’s poorest countries. It has been approved by Gavi’s board after consultation with the AU, the Africa Centres for Disease Control and Prevention (CDC), and other partners. The scheme is due to launch in June 2024 at an event hosted by the Africa CDC and France.
The initiative focuses on diseases, including cholera, plus new technologies like viral vector and mRNA vaccines, which were transformative during COVID-19 and could help in future pandemics, Kinder added.
Two Babies Infected With Dangerous Bacteria Sometimes Found in Powdered Infant Formula
The dangerous bacteria that sparked powdered formula recalls and shortages last year has infected two babies this year, killing a Kentucky child and causing brain damage in a Missouri infant.
Federal health officials confirmed Thursday that two cases of invasive infections caused by cronobacter sakazakii have been reported in 2023, both in infants who consumed powdered infant formula made by Abbott Nutrition, the company at the center of the 2022 crisis.
Food and Drug Administration officials said there was no evidence that the infections were linked to manufacturing and no reason to issue new recalls. The bacteria are found naturally in the environment and also can make their way into infant formula after the packaging is opened.
The same type of bacteria led federal investigators to shut down an Abbott formula plant in Sturgis, Michigan, last year when inspections sparked by four infant illnesses, including two deaths, showed widespread contamination in the plant.
Illinois Bill Would Require Blood Donors to Disclose COVID Vaccination Status
New legislation in Illinois would allow individuals receiving blood donations to know whether they’re receiving blood from an individual vaccinated with a COVID-19 vaccine or another messenger RNA (mRNA) vaccine.
Bill HB4243, introduced on Nov. 29 by Illinois state Rep. Jed Davis, amends the Illinois Clinical Laboratory and Blood Bank Act and would require blood banks to test donated blood for evidence of COVID-19 vaccines and other mRNA components, including lipid nanoparticles and spike protein — and requires a blood donor to disclose during each donor screening process whether they have received a COVID-19 vaccine or any other mRNA vaccine during their lifetime.
Additionally, the bill imposes labeling requirements for blood or blood components that test positive for evidence of a COVID-19 vaccine or other mRNA vaccine component or were obtained from a donor who received a COVID-19 vaccine or other mRNA vaccine.
Sanders to Target Diabetes, Weight Loss Drugs Like Ozempic
The Senate Health Committee will hold a hearing next week on the diabetes epidemic in the U.S., committee Chair Sen. Bernie Sanders (I-Vt.) said.
Sanders, who earlier this year interrogated drug manufacturers about the high cost of insulin, told The Hill the hearing will focus broadly on the underlying causes of the rise in diabetes, especially in children. Sanders also said he wanted to look into an emerging class of drugs called GLP-1 medications, like Ozempic, Wegovy and Mounjaro that are used to treat Type 2 diabetes.
They can help patients lose a dramatic amount of weight but cost more than $1,000 a month, and insurers often institute policies like lifetime coverage caps, onerous paperwork rules, or even outright refusal to cover the drugs even if they’re approved by the Food and Drug Administration.
“They’re apparently very significant drugs that could be helpful, but they’re not going to be helpful if people can’t afford them,” Sanders said. “And then the question is why we are paying in some cases eight times more for that drug than people in other countries?”
According to the health policy research group KFF, the $936 monthly list price for Ozempic is more than five times as expensive as in Japan, which has the second-highest list price at $169.
U.S. Urges Makers of Infant RSV Shots to Meet Winter Demand
U.S. officials met with manufacturers of the infant and toddler RSV immunization Beyfortus this week seeking to boost access to the shot, the White House said in a statement on Thursday after senior Biden administration officials met with the companies last week.
The officials on Tuesday met with representatives of Sanofi (SASY.PA), AstraZeneca (AZN.L) and Thermo Fisher (TMO.N) “and urged them to work expeditiously to meet the demand for immunizations this winter season through the commercial market,” the White House said in a statement.
Beyfortus, an antibody therapy developed by AstraZeneca and Sanofi, was approved by U.S. regulators in July to prevent respiratory syncytial virus (RSV) in infants and toddlers. The drug has been in tight supply since October. U.S. cases of RSV began to trend upward sharply in mid-October, which has continued, though cases are still below multiyear highs hit last winter, according to government data.
The companies have committed to producing tens of thousands of additional RSV immunizations for infants and sped up the release of 77,000 doses of the drug so far, according to the White House.
WHO Says More Contaminated Medicinal Syrups Found in New Regions
The World Health Organization on Thursday said several contaminated syrups and suspension medicines had been identified in countries in the WHO regions of the Americas, the Eastern Mediterranean, Southeast Asia and the Western Pacific.
The affected products were manufactured by Pharmix Laboratories in Pakistan, the WHO said and were first identified in the Maldives and Pakistan. Some of the tainted products have also been found in Belize, Fiji and Laos. Pharmix was not immediately available for comment.
The medicines, liquids containing active ingredients to treat various conditions, contained unacceptable levels of the contaminant ethylene glycol, WHO said.
The alert is the latest in a line of warnings from WHO about similarly contaminated medicines made in India and Indonesia, which were linked to the deaths of around 300 children worldwide last year.
