P/>BOSTON ( TheStreet) -- A GlaxoSmithKline (GSK - Get Report) scientist, Dr. Rohit Batta, may have been trying to soothe the nerves of Duchenne muscular dystrophy parents when he provided another update about drisapersen on Monday. I believe he raised even more concerns about the experimental drug's safety profile.
Here's the passage from Batta's email, sent to Parent Project Muscular Dystrophy, that stood out to me:
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.
Toxicity requiring treatment with drisapersen to be stopped is not good thing! Drug withdrawal is more serious than if treatment was merely interrupted.Batta adds: 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. Fine, but Batta is totally downplaying the seriousness of these toxicities. The boys required hospitalization! Yes, they recovered -- and that's a good thing -- but why is drisapersen causing some boys -- even a small number -- to be hospitalized for thrombocytopenia and proteinuria? GlaxoSmithKline has much more safety data on drisapersen already collected from a completed phase II study but has so far declined to make it public. Perhaps it's time to do that. My story from last week on the patients hospitalized after drisapersen therapy can be found here. Meantime, Sarepta Therapeutics (SRPT - Get Report) , presenting this afternoon at an investor conference in Boston, said its meeting with the FDA has not yet taken place but will before the end of March.