November 4, 2016
By Mark Terry, BioSpace.com Breaking News Staff
Despite significant controversy over the U.S. Food and Drug Administration (FDA)’s approval of its Exondys 51 for Duchenne Muscular Dystrophy (DMD), Sarepta Therapeutics plans for its new facility to be opened next year.
The FDA approved Sarepta’s Exondys 51 (eteplirsen) in September to treat DMD. DMD is a muscle wasting disease caused by mutations in the dystrophin gene. It is a progressive disease that usually causes death in early adulthood, with serious complications that include heart or respiratory-related problems. It mostly affects boys, about 1 in every 3,500 or 5,000 male children.
But there was significant controversy within the agency over the approval. Several prominent agency scientists argued that the company did not provide sufficient evidence that the drug actually worked. Janet Woodcock, the director of the FDA’s Center for Drug Evaluation and Research (CDER), overruled them for approval.
The approval was somewhat conditional. The company has to conduct a two-year-long clinical trial of the drug, with the possibility that if it doesn’t effectively make its case, the drug approval will be rescinded. Meanwhile, the drug is on the market with a price tag of $300,000 for an annual regimen.
So the company is continuing with its plans for another facility in Andover, Massachusetts. Two years ago, Sarepta acquired the building from Japanese drug company Eisai for approximately $15 million. The facility is at 100 Federal Street and is expected to open in the first quarter of 2017 after retrofitting is completed.
The facility is about 60,000 square feet. The construction is being performed by Timberline Construction for about $445,000.
“We wanted to increase our presence in Massachusetts,” Sarah Bard, a Sarepta spokeswoman, told the Andover Townsman.
Sarepta indicates that about 45 people will work at the Andover facility. Currently the company employs about 220 full-time staffers.
In March, Sarepta announced it was closing a 53,000-square-foot facility in Corvallis, Oregon, and consolidating most of its employees to Massachusetts. The company was originally founded in Corvallis. The former chief executive officer, Chris Garabedian, who reorganized the company to focus on DMD, told the Boston Business Journal several years ago that the move to Cambridge came about because when he was expanding the company, most of the people applying for jobs were either already there or said they’d be willing to move there.
On October 4, Sarepta inked an exclusive license and collaboration deal with UK-based Summit Therapeutics. Sarepta bought the rights to Summit’s utrophin modulator pipeline, including the lead clinical candidate, ezutromid, to treat DMD. Utrophin modulation has the potential to treat at least part of DMD regardless of the specific dystrophin gene mutation. The drug, ezutromid, is currently being evaluated in a Phase II clinical trial. Both the FDA and the European Medicines Agency (EMA) have granted ezutromid orphan drug status.
The utrophin protein is functionally and structurally similar to dystrophin. Preclinical studies have suggested that when utrophin is still expressed, it has a positive effect on muscle performance.
