​Sarepta Therapeutics (SRPT) Plunges on FDA Panel’s Vote on Eteplirsen

Henry Truc  |

Shares of Sarepta Therapeutics (SRPT) plunged by as much as 50% to $7.86 Tuesday after an FDA advisory panel voted against recommending the approval of the company’s eteplirsen treatment for Duchenne muscular dystrophy (DMD). Shares were able to pare losses by half to close the rough trading day at $11.02. While the FDA isn’t obligated to follow the recommendation, the results obviously don’t bode well for Sarepta, which will have to wait another month for the FDA’s official decision.

The point of contention between the FDA and Sarepta has been the lack of a placebo group in its clinical trial and the reported levels of dystrophin—a protein that patients suffering from DMD lack. For the placebo issue, instead of a control group, Sarepta opted to compare the data of its treatment group with data from external patient registries. Regarding dystrophin levels, the FDA panel called the reported 0.9% increase in patients receiving the experimental treatment “disappointing” and said the data was also unreliable since it only involved 12 patients.

For what it’s worth, Sarepta has a significant amount of public support for the development of eteplirsen. There currently is no FDA-approved treatment for DMD on the market. Earlier this year, both BioMarin Pharmaceutical (BMRN) and PTC Therapeutics (PTCT) saw their efforts for a DMD treatment denied as well. Over 50 patients and family members were present and made impassioned arguments to the advisory committee, but to no avail.

DMD is a rare genetic disorder that affects about 1 in 3,600 young boys. The disorder stems from a mutation of the dystrophin gene, which disables the normal production of dystrophin protein. Patients suffering from the disorder experience severe progressive muscle loss—mostly during their adolescent years—and premature death by their 20s and 30s. The devastating nature of the disorder is a reason for the strong public support from patients, advocacy groups and even government officials for eteplirsen.

For Sarepta, the FDA’s decision isn’t necessarily a surprise. The company’s stock price plunged from almost $40 a share to around $12 a share in January after a negative briefing document from the FDA highlighted similar issues. Over the past two months or so, Sarepta’s stock more than doubled off the low as investors hoped for more positive news from Monday’s AdComm review panel. They didn’t get it.

DISCLOSURE: The views and opinions expressed in this article are those of the authors, and do not represent the views of equities.com. Readers should not consider statements made by the author as formal recommendations and should consult their financial advisor before making any investment decisions. To read our full disclosure, please go to: http://www.equities.com/disclaimer

Companies

Symbol Name Price Change % Volume
SRPT Sarepta Therapeutics Inc. 149.26 -2.37 -1.56 1,274,911 Trade
PTCT PTC Therapeutics Inc. 43.02 -0.97 -2.21 321,912 Trade
BMRN BioMarin Pharmaceutical Inc. 81.57 -1.62 -1.95 893,882 Trade

Comments

Watchlist

Symbol Last Price Change % Change
AAPL

     
AMZN

     
HD

     
JPM

     
IBM

     

Blockchain in Mobility - Discussion at the EU Parliament

From the recent Blockchain For Europe Summit in Brussels: Panel on Mobility and Transportation