$3.5 Million Drug
Editor’s Letter
The biggest news that you might have missed in medicine this week were both related to drug approvals so both of our main articles are about them!
$3.5 Million Gene Therapy is World’s Most Expensive Drug
On Tuesday, the US FDA approved the first gene therapy for hemophilia, a $3.5 million one-time treatment for the blood-clotting disorder. Hemgenix, an IV treatment for adults, treats adults with hemophilia B, the less common form of the genetic.
Hemophilia B is 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.
Currently, patients are treated through IVs of that replace the missing protein. But since, the body lacks the gene to manufacture that protein on its own, this treatment must be done regularly, which is expensive. While drugmaker CSL Bering’s $3.5 million price tag is expensive, the manufacturer said that the drug would ultimately reduce health care costs because patients would have fewer bleeding incidents and need fewer clotting treatments. Bleeding incidents require hospitalization, which has a high cost.
The $3.5 million price tag makes Hemgenix the most expensive medicine in the world. But like most medicines, the cost of the treatment will be paid by insurers, not patients. Although patients will be able to pay the full price for the drug, insurers will negotiate lower prices with CSL Bering for the drug.
"Today's approval provides a new treatment option for patients with hemophilia B and represents important progress in the development of innovative therapies," said the FDA's Dr. Peter Marks.
Hemgenix delivers a working copy of the gene for the clotting protein to the liver. CSL Behring’s studies showed demonstrable benefits in long-term reduced bleeding and improved clotting. As a gene therapy, Hemgenix allows the body to produce the missing protein on its own, preventing the need for further treatment and essentially curing the disease.
Source:
A New Oral ALS Drug Shows Promise
ALS or Amyotrophic lateral sclerosis is a neurological disease that affects nerve cells that control voluntary muscle movement. In ALS these nerve cells gradually deteriorate and muscle function is lost over time. People suffering from ALS usually pass away within two to five years of the diagnosis.
Investigators from ProJenX, a biotech company recently published results from a phase 1 clinical trial with an Oral ALS drug they have been testing. It is important to point out that this is a safety trial. The drug in question, prosetin, is a MAP4K inhibitor targeting endoplasmic reticulum (ER) stress. MAP4K is a protein involved in cell signaling and inhibiting them in neurons has been shown to have a protective effect against ER stress. The working idea here is that if we can reduce ER stress among neurons, the progression of ALS can be slowed down.
In the Phase 1 trial, ProJenX was testing several doses of the drug to establish a safe dose and then look for any adverse side effects and how long the drug remains active. The Phase 1 trial is ongoing but so far the drugs has been safe in patients. If this trend continues, the company will then start moving towards phase 2 and 3 trials.
ALS is a very difficult disease to treat, with its cause still poorly understood, all treatments so far have only been able to delay the progression of the disease but not provide any sustained relief or cure. While this drug also falls in the same category, treatments like these are still important as they buy precious time to develop more advanced treatments that can hopefully completely cure the disease.
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Featured Fake News
There have been numerous different posts surfacing this week about the Vaccine Adverse Events Reporting System (VAERS) data pertaining to the COVID-19 vaccines, again. While we could address one of them individually, we would like to address the website that seems to be featured the most frequently in their sources instead:
This website is meant to be a tool to help the unfamiliar navigate through VAERS data without the need to set up advanced searches, which are extremely tedious.
However, while they have copied a lot of VAERS disclaimers, they did not copy the largest and most important one that is printed on every page while you are navigating VAERS.
“VAERS reports alone cannot be used to determine if a vaccine caused or contributed to an adverse event or illness.”
The purpose of VAERS is a place where anyone can report a vaccine adverse event. Yes, anyone, but only healthcare providers must make reports. This means that reports can be duplicated, and in the case of COVID-19 with all of the initial uncertainty around what the side effects of the vaccine could be, there were very likely many unreliable reports. This would naturally skew the numbers.
Furthermore given how politically contentious the vaccines are, the VAERS system should be approached with even more caution before any claims are made using solely its data. Now, we are NOT implying that people uploaded false information into the VAERS database. But the possibility that people could alone means you should be very skeptical of any report relying solely on VAERS data.
VAERS knows this, which is precisely why they are repeatedly warning people who visit their site that they should not use VAERS reports alone to make conjectures about rates of adverse events to vaccines.
Now, openvaers.com has been questioned before about the accuracy of the data they pull reuters fact check recently found that they were reporting vaccine associated miscarriages at 134% their actual numbers.
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