Pratteln,
Santhera and ReveraGen have now commenced the NDA filing as a rolling submission following a successful pre-NDA meeting with the FDA in
“The NDA filing for vamorolone marks a tremendous milestone for Santhera and an important next step for the Duchenne community. The potential benefits of vamorolone could address significant unmet needs that represent a burden to DMD patients and their families,” said
“We are delighted about the initiation of the filing for approval for vamorolone as it represents a culmination of over a decade of scientific research for the benefit of patients with DMD. In having granted Fast Track Designation, the FDA has acknowledged the significant innovation vamorolone could bring in addressing a high unmet medical need of patients with DMD,” said
Santhera and ReveraGen expect to complete the NDA filing in the second quarter of 2022. Based on FDA review timelines, notification from the FDA on the acceptance of the filing for review is expected in
In
Vamorolone has been granted Orphan Drug status in the US and in
Vamorolone was discovered by US-based ReveraGen and is being developed in collaboration with Santhera who owns worldwide rights to the drug candidate for all indications. In
About Vamorolone and the VISION-DMD study
Vamorolone is a drug candidate with a novel mode of action that binds to the same receptor as corticosteroids but modifies its downstream activity and as such is a dissociative agonist [2-4]. This mechanism has the potential to ‘dissociate’ efficacy from typical steroid safety concerns.
VISION-DMD was a Phase 2b study comprising a (1) pivotal double-blind 24-week period to demonstrate efficacy and safety of vamorolone (2 and 6 mg/kg/day) versus placebo and prednisone (0.75 mg/kg/day), followed by a (2) 24-week period to evaluate the maintenance of efficacy and collect additional longer-term safety and tolerability data. 121 ambulant boys aged 4 to <7 years with Duchenne muscular dystrophy (DMD) were included in the study. The trial met its primary endpoint of superiority in change of time to stand from supine position (TTSTAND) velocity with vamorolone 6 mg/kg/day versus placebo (p=0.002) at 24 weeks (period 1). Vamorolone 6 mg/kg/day also met its secondary efficacy endpoints and no statistically significant differences were observed between vamorolone and prednisone. During the second 24-week period of this 48-week study, all participants received vamorolone. Participants from the placebo and prednisone arms were randomized to either the 2 or 6 mg/kg/day dose of vamorolone and the vamorolone arms continued on their existing dose. Efficacy observed at 24 weeks for vamorolone 6 mg/kg/day was maintained across multiple endpoints over 48 weeks. In addition to efficacy, the study aimed to confirm the favorable tolerability profile of vamorolone with the potential to offer an alternative to current standard of care.
In clinical studies, vamorolone was generally well tolerated. The most commonly reported adverse events versus placebo from the VISION-DMD study were cushingoid features, vomiting and vitamin D deficiency. Adverse events were generally of mild to moderate severity.
References:
[1] ClinicalTrials.gov Identifier: NCT03439670, link
[2] Heier CR at al. (2013). EMBO Mol Med 5: 1569–1585.
[3] Reeves EKM, et al (2013). Bioorg Med Chem 21(8):2241-2249.
[4] Liu X, et al. (2020).
About Duchenne Muscular Dystrophy
Duchenne muscular dystrophy (DMD) is a rare inherited X-chromosome-linked disease, which almost exclusively affects males. DMD is characterized by inflammation which is present at birth or shortly thereafter. Inflammation leads to fibrosis of muscle and is clinically manifested by progressive muscle degeneration and weakness. Major milestones in the disease are the loss of ambulation, the loss of self-feeding, the start of assisted ventilation, and the development of cardiomyopathy. DMD reduces life expectancy to before the fourth decade due to respiratory and/or cardiac failure.
About Santhera
Raxone® is a trademark of
About ReveraGen BioPharma
ReveraGen was founded in 2008 to develop first-in-class dissociative steroidal drugs for Duchenne muscular dystrophy and other chronic inflammatory disorders. The development of ReveraGen’s lead compound, vamorolone, has been supported through partnerships with foundations worldwide, including
For further information please contact:
Santhera
public-relations@santhera.com or
Phone: +41 79 875 27 80
eva.kalias@santhera.com
ReveraGen BioPharma
Phone: + 1 240-672-0295
eric.hoffman@reveragen.com
Disclaimer / Forward-looking statements
This communication does not constitute an offer or invitation to subscribe for or purchase any securities of
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Attachment
- 2022 03 29_NDA filing_e_finalz
Source:
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