Regulatory efforts were finally rolling along at Santhera Pharmaceuticals for its Duchenne muscular dystrophy treatment. But now, a manufacturing snag has set the timeline for an FDA inspection back, and the company will need to go back to the agency to figure out a new path forward.
It’s been a tough couple years for the Swiss biotech. In 2020, the company underwent a restructuring after a phase 3 DMD asset called idebenone flopped. The company switched focus to vamorolone, also in DMD, cut 50 staffers and saw its chief medical officer pack her bags. The cuts meant $10 million in savings, and the company managed to borrow another $16 million to stay afloat.
Fast-forward to 2022, and Santhera finally got things moving again with a rolling new drug application for vamorolone in March. The company secured about $42 million in additional funds (PDF) and negotiated a pending milestone payment with partner ReveraGen, which owns vamorolone, to garner more time.
But now, time is not on their side. The company revealed Wednesday (PDF) that its contract manufacturing partner responsible for prepping for an FDA inspection for the vamorolone application is not ready. The facility will not be available for inspection until later this year, pushing back the timeline by four to six months, according to Santhera. This inspection is needed for the FDA to deem the vamorolone application complete and accept it for review.
Santhera will now have to chat with the FDA and figure out an updated timeline for completion of the rolling submission, which the company hopes can be wrapped up by the fourth quarter. The company had initially expected to have the approval in hand via priority review by the third quarter.
Vamorolone holds orphan-drug status in the U.S. and EU, fast-track and rare pediatric disease tags from the FDA and promising innovative medicine status in the U.K. Santhera is preparing a marketing authorization request for the EU as well, with no changes to that plan at this time.