1. The US trial showed no consistent production of dystrophin till 24 weeks. If I recall the 12 wk 50 m, gave hints of some dystrophin increases, but nothing approaching certainty.
2. The UK trial showed some very inconsistent dystrophin increases at 12 weeks at much smaller dosages.
3. Dystrophin measurements are only noted where the biopsies are taken, It is quite possible the dystrophin may increase faster or slower in other parts of the body.
4. It is not obvious to know how much dystrophin is really meaningful.
5. Isn't it likely dystrophin may increase in a body in a very different way, if that particular body has had large muscle masses atrophy due to being wheelchair bound?
6. And last not being least, it is quite likely some small portion of the gains are due to placebo effect.
Hopefully the FDA is ready to step up and do their job as they have done on previous rare diseases. It is a shame it took them so long to get there.
Going from memory the A.Rus patent didn't state the Kole patent as prior art. Even if it had expired it is still prior art. Therefore they really didn't address the test as to whether the A.Rus patent was a non obvious step over Kole and other prior art combined. Furthermore there was an A.Rus publication out of the window for time till a patent would have to be filed and they appeared to have ignored whether the patented material was a non obvious step over this earlier A.Rus disclosure. Furthermore the A.Rus patent is very broad and many claims should be narrowed to assure they are 1) non obvious over prior art and 2) They are enabled by the disclosures in the patent.
On a bigger topic, I have a real issue with universities granting exclusive licences to firms on IP that drives life and death issues. I would have no problem, if they granted a license with some signficant first licensee advantage, Possibly involving lower fees, some short exclusivity, but not to the point where it should be restrictive. Now, if it was a truly private university, I might be more inclined to allow the present system, Thouh I question whether there is really such a thing as a truly private university, since most depening on some government funding and tax benefits.
Feels to me like a European University got a big home field advantage in deciding to allow the A.Rus patents. I also am not a patent lawyer.
We really have no idea whether the doors were being pounded on at Sarepta. We know Sarepta publically stated they weren't interested in a partnership. One big question with Sarepta's technology, if it will be possible to manufacture PMO's in volume at a viable price, the answer appears to becoming clearer that it will be. The next question is will it be reasonable enough to attack non rare disease markets? If so, and if the testing continues to be good Sarepta may have an incalculable value! It just needs a cash generator to fund going forward.
It looked clear that based on precedent in rare diseases that the cash generator was there until the FDA set aside science and went off on the not ready tangent based on the screwed up Prosensa/GSK results.
Now 2 years later, there is a lot more data and the FDA appears ready to do what it should have been ready to do long ago. Never mind the innocent kids that died and lost the ability to walk because of its sad faulty logic.