PPMD received an email from Dr. Rohit Batta, Global Medical Affairs and Patient Relations Leader in GSK's Rare Disease Unit following the recent story regarding his presentation on drisapersen at the Duchenne Parent Project Onlus meeting on February 24:
Since my last email you may have seen a recent story that appeared on an investment tips website regarding a presentation on drisapersen made at the Duchenne Parent Project Onlus meeting on 24th February 2013 in Rome, Italy.
We appreciate that the story may raise questions for you, so I wanted to write and provide you with a little more detail and context. At the meeting, I provided an overview of our clinical programme, which is the largest investigation of a medicine in DMD boys ever conducted involving over 300 boys.
The story that appeared on the investor website highlighted the point that a small number of boys in the clinical trial programme were hospitalised due to thrombocytopenia (a decrease in the number of platelets or small cells that help blood to clot) and proteinuria (excessive protein in the urine). The boys have all recovered, following withdrawal of drisapersen and appropriate medical management. I would like to assure you that maintaining the safety of the boys in the drisapersen clinical studies is of paramount importance to GSK. Over the course of conducting the drisapersen programme, there have been a small number of serious adverse events of thrombocytopenia and proteinuria. In the clinical trials, a close watch is being kept on platelet counts and on proteinuria through blood and urine tests. It is important to note that it is usual practice for an investigator to admit a boy to hospital if necessary, for the boy to be appropriately treated and to recover.
We are confident that the current safety monitoring procedures set out in the study protocols, and agreed by regulatory authorities, are appropriate to ensure the safety of the boys participating in our clinical trials. The GSK Safety Review Team continually monitors the safety in the clinical trials and an external panel of medical and statistical experts also periodically review the drisapersen safety data (the Independent Data Monitoring Committee). The adverse events have also been disclosed in a timely manner to the appropriate regulatory authorities and study investigators.
Although a small number of subjects withdrew from medication due to adverse effects, approximately 96% of boys randomised have continued to receive assigned study treatment. The large database includes approximately 180 boys who have completed 48 weeks of double-blind treatment. In the open label extension study a further 117 boys have completed 24 weeks and 41 have completed 48 weeks. Currently, all drisapersen clinical trials are progressing as planned and boys are continuing to receive medicine as per study protocol.
As part of GSK’s commitment to transparency, we have shared safety information on our clinical program on an ongoing basis at a number of patient advocacy meetings, including the recent Duchenne Parent Project Onlus meeting in Italy, and past US Parent Project Muscular Dystrophy (PPMD) and UK Action Duchenne meetings (among others). We also plan to share the results of our completed Phase IIb study (DMD114117) in the coming months, with scientific conference presentations and a manuscript submission.
The following is still very concerning, especially when SRPT's drug appears to have no safety issues:
"The boys have all recovered, following withdrawal of drisapersen and appropriate medical management."
So apparently 4 percent of patients have reactions severe enough to drop out of the study. God knows how many had "less severe" reactions where they chose the ability to continue to walk in spite of the side-effects. No numbers given here, but SRPT CEO already laid out some pretty significant numbers. They will definitely lose in heads up competition all other things being equal - which looks to be the case. Could pricing itself become a battleground? Just wondering if anyone has any idea.
Also not being able to clot blood in the short term is a pretty scary thing to have as a problem especially in children who aren't exactly the most careful when it comes to running around. That plus the kidney disease risk makes these huge hurdles for GSK to overcome.
A naive question due to my lack of intimate familiarity with orphan drug indications. How is this drug - costing 350-500K per year for the rest of a patient's life paid for. Does insurance automatically cover without limits. Does anyone have any first hand knowledge of what financing is typically worked out. Just curious since it is a little hard to believe insurers willingly swallow 25M in treatment costs for each boy over a lifetime. Any insight is appreciated.
ivan, my question exactly. It's hard for me to believe that any insurance company is going to pay for this. I'm sure this must have been discussed here before, but I haven't seen anything. The cost is exactly the reason I'm hesitant to buy. I don't believe the insurers will cover it.
I can see the headlines now: "Hey, we're going to 'do the right thing' and offer Drisapersen for a whopping 25% less than the folks at SRPT charge for their drug, and ours makes just as much dystrophin. That way, you'll have enough left over to purchase your own dialysis machine, and a few nice tourniquets too, for the bleeding issues that (may) arise. GSK/Prosena cares"
Gimme a break. It seems that all GSK will be able to offer is trading one life threatening problem for a different life threatening problem, or two. The King has no clothes despite what he would like others to believe.
Also no denying that the medication causes SAE's. Straight from GSK's mouth. We have no safety issues and evidence that the drug does not cause safety issues based on 400 other patients using the chemistry for other indications. Seems very convincing on the safety side. AA lock and GSK not an issue. Worrywarts have it all wrong. Now I just need to hear whether they have met with FDA alredady and I am good to go.