March 9, 2016
By Mark Terry, BioSpace.com Breaking News Staff
Cambridge, Mass.-based Sarepta Therapeutics announced yesterday that it was shuttering its research-and-development manufacturing facility in Corvalis, Ore. Most of the employees there are expected to move to Sarepta’s facilities in Andover and Cambridge, Mass. About 30 people are expected to be laid off.
On Jan. 21, Sarepta announced that, with an impending snowstorm on the east coast, the U.S. Food and Drug Administration (FDA)’s meeting to review the company’s New Drug Application (NDA) for eteplirsen to treat Duchenne Muscular Dystrophy (DMD) was postponed.
DMD is a muscle wasting disease caused by mutations in the dystrophin gene. The disease is progressive and generally causes death in early adulthood. Complications include serious heart or respiratory-related problems. It mostly affects boys, about 1 in every 3,500 to 5,000 male children.
On Jan. 15, an FDA advisory committee decided to reschedule the meeting, at which point a recommendation or approval decision will be made. That meeting of the Peripheral and Central Nervous System Advisory Committee has not been rescheduled yet, but Sarepta believes it will be prior to May 26, which is the PDUFA date. The Prescription Drug User Fee Act (PDUFA) is a law that allows the FDA to collect an application fee from drug companies when an NDA or Biologics License Application (BLA) is submitted.
The DMD drug arena has been fraught with failures and bad news this year. San Rafael, Calif.-based BioMarin Pharmaceutical Inc. ’s application for its DMD drug Kyndrisa (drisapersen) was turned down by the FDA on Jan. 15. The FDA argued that Kyndrisa didn’t show enough benefit.
On Jan. 25, Cambridge, Mass.-based Akashi Therapeutics announced that it had halted its DMD trial for HT-100 after one of its patients developed serious, life-threatening health problems. In that DMD is a serious, life-threatening health problem in itself, it’s not clear if the patient’s problems are directly related to the drug. The patient was receiving the highest dose in the HALO trial, while others in the trial with lower doses were not showing adverse side effects.
Sarepta plans to consolidate its operations in four waves starting in May, and following in October, November and December of this year. It plans to have the shift completed by Dec. 30, 2016.
“For a biotechnology company of our size and stage of development, it is operationally important to centralize our facilities and focus our efforts on the R&D, manufacturing, and pre-commercialization of eteplirsen,” said Edward Kaye, Sarepta’s interim chief executive officer and chief medical officer, in a statement. “To that end, we will consolidate operations to Massachusetts over the coming year to optimize the efficiency of our R&D and manufacturing teams.”
The Oregon facility is 53,000 square feet and was leased. The company’s headquarters in Cambridge are 88,000 square feet. It has two facilities in Massachusetts, the other is a 60,000-square-foot planned manufacturing center in Andover. It is not currently in use. The company originally launched in Oregon before shifting to Seattle, then to Massachusetts.
Sarepta hopes the FDA will grant conditional approval for the drug, and has plans to conduct a larger confirmatory trial. The initial study was based on 12 patients and has been in progress for four years. The study’s small size is at least part of the problem with possible approval, although most patients seem to respond to the drug.
The Boston Business Journal reports that if the company continues operations on the drug, it will cost around $100,000 annually to manufacture the drug for clinical trials. As of the end of 2015, it had $204 million in cash and cash equivalents. Investors have not liked the uncertainty. Sarepta traded for $41.79 on Oct. 5, 2015 and is currently trading for $14.44.
