where she both started a bioterrorism program and oversaw planning for a potential pandemic flu response. Again, she was successful at the Federal level both for her expertise in the medical field and for successfully navigating Washington politics in connection with accomplishing her stated medical goals.
From my vantage point, the idea that Dr. Hamburg would agree to meet in a well-publicized manner with Mrs. Jenn McNary and her group, while knowingly planning to deny the right to submit an AA application for etelplirsen betrays a lack of knowledge as to how Washington DC operates.
I’ve interacted over the years with experts involved in interacting with the FDA on numerous drug submissions. I can tell you that all politicians within government (and make no mistake about it, Dr. Hamburg didn’t get to where she is today by lacking political judgement) seek to attach themselves to the winning horse. In this case, the winning horse is Jenn McNary and her fellow activists who are out to save the lives of children. The FDA has already met with the patient advocates at the operational level (again, see the DMDHero notes from the February 15th meeting) and Dr. Hamburg, knowing as she does the status of etelplirsen within the FDA, could have easily declined the meeting if she felt the outcome was going to be a negative one. This meeting is so close to the FDA’s decision that any new information coming out of it would fail, in my opinion to affect the FDA’s decision.
So why agree to meet? It’s simple really – I believe Dr. Hamburg is meeting with the group this close to the decision point so that when the good news is handed down, the photo ops, the Washington Post article, the lavish specialty medical journal coverage can all point to Dr. Hamburg as one of those ‘heroes’ alongside the smiling McNary clan. Seriously, does anyone who knows something of her career expect her to agree to meet with these activists while knowingly planning to shoot them down a few weeks later??
You don’t have to be a cynic or believe in the bad intentions of Dr. Hamburg – I know for a fact that she is a consummate physician. Rather, in our democratic/capitalist system, impacting public opinion is a necessary prerequisite to getting anything accomplished within government. In other words, it’s just how the business of governing our country gets done in DC. The FDA oversees 25% of all consumer purchases in this country (food and drugs are a trillion dollar a year economy) and Dr. Hamburg surely knows she needs all the good will on Capital Hill to adequately fund her plans for the FDA. Being a hero – to Jerry’s kids at the MDA no less! – burnishes her image and with the upcoming budget battle for scarce Federal dollars you can bet that Dr. Hamburg will position herself accordingly in the eyes of public opinion.
When the photo ops of the March 12th meeting take place, one needs to evaluate whether or not Wall Street will be as skeptical as it appears to be with the stock in the high 20s. I think the relatively low volatility in the in-the-money March calls are a good way to take a flyer on Wall Street being pleasantly surprised by the FDA’s acceptance of eteplirsen.
Longer term, I think the value in Sarepta is enormous. Perhaps in a future piece it would be worth laying out the case for why the FDA will ultimately approve Sarepta’s drug platform as a basis to rapidly churn out treatments for the other exon-mutation classes of DMD patients. Sarepta already has treatments for exon 45, 50 & 53 in pre-clinical development.