Dr. Derek Lowe, from In the Pipeline, writes:
So since drug pricing and FDA regulations are so much in the news, it would seem like the perfect time for a small company to game the system for big profits, right? That's apparently what Marathon Pharmaceuticals believes. They just got approval for deflazacort, a steroid, as a treatment for Duchenne Muscular Dystrophy.
[...] So what's not to like? Well, this drug has been around since the early 1990s. Marathon most certainly did not invent it. Nor did they think of applying it to DMD patients – the biggest clinical trial of the drug for that indication was done over twenty years ago, by someone else. DMD patients in the US were already taking the (unapproved) drug by importing it from Canada. Marathon just dug through the data again and ran a trial in 29 patients themselves, from what I can see. I should note that this is not any sort of cure, nor does it address the underlying pathology of the disease. The steroid treatment makes muscle strength in DMD patients stronger – barely. But even for that benefit, US patients will now have to get it from Marathon at something like 50 to 100 times the former price.
[...] So while I defend the FDA's function of making it tough on new drugs (making them prove safety and efficacy), I cannot stand how loose they are with old generic compounds. The agency hands out extremely valuable rewards like lollipops in these cases – a priority review voucher can be sold for hundreds of millions of dollars
[...] And they're also allowing the likes of Marathon to make the rest of the drug industry look like greedy sociopaths. Marathon, Catalyst, T*ring and all the rest of the people who are pulling these tricks have the word "Pharmaceuticals" in their name, but they are not drug companies. They discover nothing. They do no research. They take virtually no risks. They exist only to play legal games and watch the money roll in.
[...] As for the FDA, the agency probably can't change this on its own, though, even if it wants to – Congress has to act to give them the authority to deny market exclusivity or priority review vouchers under some conditions. Either that, or we should rethink these incentives entirely, because they are (clearly) too easy to exploit for fast bucks.
Also at ArsTechnica.
Dr. Lowe, from In the Pipeline, writes an update on a story we covered earlier this year:
You may recall Marathon Pharmaceuticals, the small company that announced plans to sell a long-used steroid treatment (Emflaza, deflazacort) in the US to Duchenne muscular dystrophy patients. The price was set to go up steeply, since the company was awarded years of market exclusivity by the FDA (under their program to reward orphan-drug indications like this one).
This business model is the same one followed by a number of other small outfits (see that link above for more), and it's infuriating. Generic drugs are off patent, by definition, and they're supposed to be cheap. Taking advantage of regulatory loopholes and perverse incentives to jack their prices up is shameful, unproductive, and expensive.
[...] Marathon itself appears to be about to disappear. And why not? They've turned a quick buck. Endpts, who have been doing a great job on this story, couldn't find anyone who thought that the company had spent more than $70 million on the drug's approval, and it was probably a lot less. So $140 million, plus milestones and royalties, is a nice return. But there's more money coming than just that – the company got a priority review voucher from the FDA for bringing a rare pediatric disease drug to the market, and they can sell that on the open market. I'd guess that it could bring in another $100 million or so
The priority review voucher is an incentive program that provides companies, that get a drug approval for a neglected or rare disease, a voucher that entitles them to a more speedy review (under six months instead of ~10 months) for a future drug. This is a great program; however, Marathon Pharmaceuticals deliberately exploited this program by conducting a very small trial using a generic drug (another loophole).
Previous Story: https://soylentnews.org/article.pl?sid=17/02/11/2116252
A new and non-viral approach to CRISPR has been used to treat Duchenne muscular dystrophy in mice:
A new version of the CRISPR-Cas9 gene-editing technology called CRISPR-Gold has successfully restored the correct sequence of the dystrophin gene in a mouse model of Duchenne muscular dystrophy (DMD), a new study revealed.
Researchers found that an injection of CRISPR-Gold into DMD mice led to an 18-times-higher correction rate and a two-fold increase in a strength and agility test compared to control groups, according to a press release.
The study, "Nanoparticle delivery of Cas9 ribonucleoprotein and donor DNA in vivo induces homology-directed DNA repair," [DOI: 10.1038/s41551-017-0137-2] [DX] was published in the journal Nature Biomedical Engineering.
[...] Unfortunately, methods of delivering the components of this system, which include an RNA molecule called a guide RNA, a protein called the Cas9 nuclease, and the correct DNA sequence to replace the mutation (via donor DNA), have not been fully developed for human use. A primary technique used to deliver the components of this system relies on viruses, but this technique is plagued by complications and unwanted side effects.
In response, researchers at the University of California, Berkeley have developed a new approach called CRISPR-Gold, which used gold nanoparticles to deliver the components of this system in a mouse model of DMD. This method works by using gold nanoparticles to coat a modified DNA molecule that binds the donor DNA, which in turn is bound to Cas9 and the guide RNA.
This entire system is then coated by a polymer that will interact with a cell membrane and allow entry into a cell. Then, the components of the system are released into the cell as the coat breaks apart upon entry. The guide RNA, the Cas9 nuclease, and the donor DNA can then make their way into the nucleus and correct the mutation.
Also at TheScientist.
Previously: FDA Panel Recommends Rejection of Duchenne Muscular Dystrophy Treatment
Marathon Pharmaceuticals is Part of the Problem
Marathon Pharmaceuticals Cashes Out on Regulatory Loopholes
What is a Muscle Protein Doing in the Brain?
As the world knows, we face an emerging virus threat in the Wuhan coronavirus (2019-nCoV) outbreak. The problem is, right now there are several important things that we don't know about the situation. The mortality rate, the ease of human-human transmission, the rate of mutation of the virus (and how many strains we might be dealing with – all of these need more clarity. Unfortunately, we've already gone past the MERS outbreak in severity (which until now was the most recent new coronavirus to make the jump into humans). If we're fortunate, though, we'll still have something that will be worrisome, but not as bad as (say) the usual flu numbers (many people don't realize that influenza kills tens of thousands of people in the US each year). The worst case, though, is something like 1918, and we really, really don't need that.
[Ed note: The linked story is by Derek Lowe who writes a "commentary on drug discovery and the pharma industry". He is perhaps best known for his "Things I Won't Work With" blog entries which are as hilarious as they are... eye opening. I have found him to be a no-nonsense writer who "tells things as they are", holding no punches. The whole story is worth reading as he clearly explains what a coronavirus is, about the current one that reportedly originated in Wuhan, China, what could be done about it, how long that would likely take, and what can be done for those who have already been infected. --martyb]
Previous Stories Referencing Derek Lowe:
Machine Learning Comes to Biochemistry
Ignition! The Funniest, Most Accessible Book on Rocket Science is Being Reissued
Another Failed Alzheimer's Disease Therapy
Marathon Pharmaceuticals is Part of the Problem
Lobbying Results in FDA Approval for Controversial Drug
"Right to Try" New Experimental Medicine and the Value of Experts
Cancer Hazard vs. Risk - Glyphosate
A Terrific Paper on the Problems of Drug Discovery
Things I Won't Work With