Shares drop at Santhera after FDA requires more trial data DMD drug
pharmafile | July 15, 2016 | News story | Medical Communications, Research and Development | Duchenne Muscular Dystrophy, FDA, santhera
Santhera Pharmaceuticals (SIX: SANN) has seen shares fall as much as 38% after the US Food and Drug Administration (FDA) indicated that it would require further Phase III results before it could approve Raxone (idebenone) for the treatment of Duchenne muscular dystrophy (DMD).
The FDA issued written correspondence to Santhera, indicating that results from the SIDEROS Phase III trial, for which results aren’t expected until the second half of 2019, should be provided at the time of filing to support a new drug application for Raxone.
The rare, degenerative Duchenne muscular dystrophy currently has no available treatments, but several have come before the FDA for approval in recent months. Shares fell sharply, and then jumped again, at Sarepta Therapeutics after the FDA expressed reservations about the efficacy of the drug, but then delayed its final decision. PTC Therapeutics had its drug candidate rejected by the FDA, and cut jobs as a result, while Marathon Pharma submitted an application for approval to the regulator last month.
Santhera had proposed a partial approval of the drug, in the subpart H pathway for Raxone in patients not taking concomitant glucocorticoids, with data from the SIDEROS trial expanding the label to include the treatment of glucocorticoid-using patients, but the US regulator rejected this suggestion.
Shares at the Swiss firm dropped sharply as a result, dropping as much as 38%.
Thomas Meier, CEO at Santhera, says: “We are disappointed that the FDA does not support our plan to file an NDA for Raxone under subpart H for patients not using concomitant glucocorticoids, we now have clarity that successful completion of the SIDEROS trial will provide the necessary data to support NDA filing for Raxone in DMD patients irrespective of the glucocorticoid use status.
“Enrolment in the SIDEROS trial will start shortly and we are committed to working closely with the FDA, clinical experts and the DMD patient community to make Raxone available for all DMD patients in the US as quickly as possible.”
Raxone is currently under regulatory review by the European Medicines Agency for the treatment of DMD patients with respiratory function decline who are not taking concomitant glucocorticoids.
Sean Murray
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