February 17, 2017
By Alex Keown, BioSpace.com Breaking News Staff
BURLINGON, Mass. – Switzerland-based Santhera Pharmaceuticals (SANN.SW), the maker of a treatment for Duchenne Muscular Dystrophy, has opened its first office in the United States.
Santhera’s U.S. facility will be staffed by about 20 employees, but has plans for possible expansion, the Boston Business Journal reported this morning. It is unknown what expansion plans Santhera has for its U.S. offices, but it’s likely they hinge on its second Phase III trial for Raxone, its experimental DMD drug. Raxone is being developed to treat all DMD patients regardless of mutation. In July last year, Santhera suffered a setback when the U.S. Food and Drug Administration (FDA) told the company it must conduct a second Phase III trial before it could file a New Drug Application. At the time the FDA issued its notice, Thomas Meier, chief executive officer of Santhera, said they now have “clarity” that successful completion of the additional Phase III trial will “provide the necessary data to support NDA filing for Raxone in all DMD patients irrespective of the glucocorticoid use status.”
Santhera expects results from its second Phase III trial in 2019. The first Phase III trial for Raxone showed the drug was effective in slowing the loss of respiratory function in DMD patients and reduced bronchopulmonary complications. The FDA told Santhera that it needed to include additional information in order to file the NDA, hence the second Phase III trial.
Meier told the Journal that opening an office in the United States will allow the company to “expand our engagement with physicians, their patients, and families” as the company moves forward with the Phase III trial.
As Santhera moves into the greater Boston area, the company will become neighbors with rival companies developing their own DMD treatments, including Catabasis Pharmaceuticals (CATB) and Summit Therapeutics, among others. Cambridge-based Sarepta Therapeutics (SRPT) became the first company to win FDA approval for a DMD treatment called Exondys51, which targets a specific genetic mutation of the drug found in about 13 percent of patients, the Journal said.
Illinois-based Marathon Pharmaceuticals won approval for its DMD drug Emflaza earlier this month, to treat patients five years of age and older regardless of genetic mutation. However, that company has put its commercialization plans on hold following an outcry over the $89,000 price tag the drug has.
Duchene muscular dystrophy is a fatal genetic disease. Most patients are diagnosed by the time they reach five years of age and live until their mid-20s.