- Stealth BioTherapeutics (NASDAQ:MITO) plans to meet with the FDA's Division of Cardiology and Nephrology (DCN) regarding its upcoming NDA submission for elamipretide for the treatment of cardiomyopathy in Barth syndrome.
- This Type B Pre-NDA meeting follows prior interactions during which the Agency encouraged the Company to generate additional clinical data to support its Barth NDA submission, including by implementing a withdrawal protocol for the patients remaining on open-label extension.
- During the recent meeting, DCN suggested that, based on the short half-life of elamipretide, a short-term withdrawal period could be sufficient to measure durable effect on functional or structural cardiac endpoints by demonstrating that improvement is not dependent on continuous exposure to the drug.
- Although DCN reiterated the prior feedback regarding the insufficiency of the current data package, the Company believes that the existing data with additional analyses could meet the requirements for an NDA.
- The Company is proposing that a short-term withdrawal protocol for open-label extension participants be conducted in parallel with NDA review and that a larger Phase 3b/4 withdrawal study in treatment-naïve patients be completed as a post-marketing confirmatory study.
- The Company plans to discuss this and other aspects of the anticipated NDA submission during the Pre-NDA meeting next month.
- Stealth Bio will host a conference call today at 8:30 am ET to provide additional detail regarding its interactions with DCN and anticipated NDA submission timelines.
- Shares up 8% premarket.