Santhera Pharmaceuticals (SIX: SANN) announces that the clinical module of its rolling new drug application (NDA) for vamorolone in Duchenne muscular dystrophy (DMD) has been submitted to the U.S. Food and Drug Administration (FDA). Finalization of the NDA submission to start the FDA official review is postponed by 4-6 months to Q4-2022 after a third-party contract manufacturing organization (CMO) communicated a delay in establishing FDA-inspection readiness.
Santhera commenced the NDA filing as a rolling submission in March 2022 following a successful pre-NDA meeting with the FDA. In its conclusions from this meeting, the FDA considered the proposed clinical efficacy and safety data sufficient to support an NDA filing of vamorolone for the treatment of DMD. The rolling submission has been proceeding according to plan with the clinical module submitted on June 28, 2022.
A third-party contract manufacturing organization has informed Santhera that establishing preparedness for an FDA pre-approval inspection is delayed until later this year. Formal confirmation of inspection readiness by the CMO is a prerequisite for the FDA to consider a filing complete and to accept an NDA for review. Therefore, Santhera will discuss with the Agency the status and updated timelines for completing the NDA filing by Q4-2022.
In Europe, preparations for a marketing authorization application (MAA) for vamorolone for the treatment of DMD to the European Medicines Agency (EMA) are proceeding according to plan.
Vamorolone has been granted Orphan Drug status in the US and in Europe for DMD, and has received Fast Track and Rare Pediatric Disease designations by the US FDA and Promising Innovative Medicine (PIM) status from the UK MHRA for DMD. Vamorolone is an investigational medicine and is currently not approved for use by any health authority.
