The US Food and Drug Administration on Tuesday approved Hemgenix, a new drug to treat hemophilia. Manufacturer CSL Behring set the price at $3.5 million per treatment, making it the most expensive drug in the world.
Hemgenix is a gene therapy to treat adults with hemophilia B, a genetic bleeding disorder in which people do not produce a protein needed to create blood clots. About 1 in 40,000 people have the disease, most of whom are men.
“Gene therapy for hemophilia has been on the horizon for more than two decades. Despite advancements in the treatment of hemophilia, the prevention and treatment of bleeding episodes can adversely impact individuals’ quality of life,” Dr. Peter Marks, director of the FDA’s Center for Biologics Evaluation and Research, said in a statement. “Today’s approval provides a new treatment option for patients with Hemophilia B and represents important progress in the development of innovative therapies for those experiencing a high burden of disease associated with this form of hemophilia.”
The Institute for Clinical and Economic Review, an independent nonprofit research organization that analyzes the value of prescription drugs and other medical products, confirmed for CNN that Hemgenix is now the most expensive drug in the world.
In a recent cost-effectiveness analysis of the drug, weighing health benefits against offset costs, ICER suggested that a fair price for the drug to be between $2.93 million and $2.96 million.
According to an analysis by GoodRx, the most expensive drug in the US previously was Zolgensma, which was approved by the FDA in 2019 to treat spinal muscular atrophy and priced at $2.1 million for a course of treatment.
FDA approved Hemgenix based on safety and effectiveness evaluated in two studies of about 60 adult men. The most common side effects included high liver enzymes, headaches, flu-like symptoms and mild infusion-related reactions.
News
Silver nanoparticles show promise in fighting antibiotic-resistant bacteria
In a new study, scientists with the University of Florida have found that a combination of silver nanoparticles and antibiotics is effective against antibiotic-resistant bacteria. The researchers hope to turn this discovery into viable [...]
Combating severe cancer with a new drug delivery system
Peritoneal cancer is difficult to treat and has a poor survival prognosis. But a new and effective nanomedicine delivery system is offering some hope. The company is called NaDeNo and is well underway with [...]
New Research Shows How Ketamine Acts As “Switch” in the Brain
According to a new study by researchers at Penn Medicine, ketamine, which is well-known as an anesthetic and is becoming increasingly popular as an antidepressant, dramatically reorganizes activity in the brain, almost as if [...]
Supercharged T Cells: A New Way To Kill Pancreatic Cancer With Minimal Side Effects
A new immunotherapy releases cancer-killing cytokines only within the tumor. Researchers at the University of California San Francisco (UCSF) have developed a new T cell-based immunotherapy that selectively targets cancer cells, producing a powerful anti-cancer cytokine [...]
AI has designed bacteria-killing proteins from scratch – and they work
An AI was tasked with creating proteins with anti-microbial properties. Researchers then created a subset of the proteins and found some did the job. An AI has designed anti-microbial proteins that were then tested [...]
Using nanoparticles, researchers can identify and deliver synergistic combinations of cancer drugs
Treating cancer with combinations of drugs can be more effective than using a single drug. However, figuring out the optimal combination of drugs, and making sure that all of the drugs reach the right [...]
Humanity May Reach Singularity Within Just 7 Years, Trend Shows
By one unique metric, we could approach technological singularity by the end of this decade, if not sooner. A translation company developed a metric, Time to Edit (TTE), to calculate the time it takes for professional [...]
HYPER (Highly Interactive Particle Relics) – A New Model for Dark Matter
Phase transition in early universe changes strength of interaction between dark and normal matter. Dark matter remains one of the greatest mysteries of modern physics. It is clear that it must exist, because without [...]
New Nanoparticles Deliver Therapy Brain-Wide and Edit Alzheimer’s Gene
Summary: Researchers have developed a new family of nano-scale capsules capable of carrying CRISPR gene editing tools to different organs of the body before harmlessly dissolving. The capsules were able to enter the brains of [...]
Cancer’s Secret Weapon? Enzyme That Protects Against Viruses May Fuel Tumor Evolution
An enzyme that defends human cells against viruses can help drive cancer evolution towards greater malignancy by causing myriad mutations in cancer cells, according to a study led by investigators at Weill Cornell Medicine. The [...]
Scientists Uncover Japanese Fruit Juice That May Help Prevent Lung Cancer
Using a mouse model, Japanese researchers unleash the likely mechanism of action of Actinidia arguta (sarunashi) juice on lung cancer development. Lung cancer is a leading cause of death in Japan and across the [...]
In-place manufacturing method improves gas sensor capabilities, production time
When used as wearable medical devices, stretchy, flexible gas sensors can identify health conditions or issues by detecting oxygen or carbon dioxide levels in the breath or sweat. They also are useful for monitoring [...]
In the core of the cell: New insights into the utilization of nanotechnology-based drugs
Novel drugs, such as vaccines against covid-19, among others, are based on drug transport using nanoparticles. Whether this drug transport is negatively influenced by an accumulation of blood proteins on the nanoparticle’s surface was [...]
The costly lesson from COVID: why elimination should be the default global strategy for future pandemics
Imagine it is 2030. Doctors in a regional hospital in country X note an expanding cluster of individuals with severe respiratory disease. Rapid whole-genome sequencing identifies the disease-causing agent as a novel coronavirus. Epidemiological [...]
How Artificial Intelligence Found the Words To Kill Cancer Cells
A predictive model has been developed that enables researchers to encode instructions for cells to execute. Scientists at the University of California, San Francisco (UCSF) and IBM Research have created a virtual library of thousands of “command sentences” [...]
Next-generation, light-activated nanotech for antibiotic-resistant superbugs
It's "lights out" for antibiotic-resistant superbugs as next-generation light-activated nanotech proves it can eradicate some of the most notorious and potentially deadly bacteria in the world. Developed by the University of South Australia and [...]
