Big Day Tomorrow

By November 23, 2015October 28th, 2016Tracy's Blog

Tomorrow is a big day. A special advisory committee convened by the FDA will spend all day reviewing the first Duchenne treatment up for approval in the US. By the end of the day, the “ad comm” will answer questions FDA reviewers have posed to help them decide whether to approve the treatment. Regardless of what that final decision may be, there is one outcome we all must insist on, and it’s not a simple “yes” or “no” on approval of Kyndrisa.

We all must insist that we apply lessons learned over the past three tumultuous years so the next round of new drugs is evaluated much more quickly and much more fairly. Our kids have lost too much time waiting to get to this point. We owe it to our kids to be ready, willing, and able to develop and evaluate new medicines faster. We have to do better before it’s too late.

The regulatory process for the first drugs to treat the #1 genetic killer of children worldwide has been fraught with inertia, misinformation, and conflict. Time and again, we have had high hopes – reasonable hopes – that were dashed when the process broke down and became completely derailed by indecision, confusion and a lack of urgency.

BioMarin’s Kyndrisa has its big day tomorrow, to be followed in a few weeks by a decision on a drug called Eteplirsen being developed by Sarepta Therapeutics. The fate of Eteplirsen – a drug with an entirely different safety and efficacy profile – has been inextricably and unjustifiably bound to Kyndrisa in regulators’ reviews and in public perception.

Finally, after three tumultuous years, “…it seems likely the situation will play out as it should,” predicts a recent article from a financial news outlet. “The better drug reaches the market, rewards Sarepta and its shareholders after years of riding a roller coaster, and patients (finally) get unfettered access to a drug that seems to be able to save lives.”

But even if this prediction comes to pass, in Duchenne all is not well that ends well. Wasted time translates to crucial independence lost for children like my son Charley. In early 2014, when Sarepta first planned to submit a new drug application, Charley could climb up and down stairs. Now he cannot. My friend’s son Joseph has completely lost the ability to walk and is losing the ability to feed and care for himself. And too many of our boys have lost their lives, leaving empty seats at their family Thanksgiving celebrations later this week.

These losses, unlike the losses to investors’ portfolios, are permanent. And there are other casualties. The “strong public outcry” described by the same financial reporter cost our community considerable resources that could have and should have been spent on medical research to develop the next generation of Duchenne treatments. The moms and dads who racked up frequent flier miles going to advocate in DC instead of baking banana bread and helping their kids with homework will never regain that time with their families.

It has been painful to watch my son lose muscle function over the past three years while this bureaucratic mess unfolded. But it is determination, not bitterness, that drives me. Charley will never bound up the stairs again and Joseph will never walk independently again. But we have a chance to save their lives. Now let’s do it.