Santhera Concludes Agreement with French Authorities on Raxone® Reimbursement and Plans to Submit a Request for an Early Access Program for Vamorolone
Pratteln, Switzerland, June 23, 2021 – Santhera Pharmaceuticals (SIX: SANN) announces positive topline results from its long-term Phase 4 LEROS study with Raxone® (idebenone) in the treatment of Leber’s hereditary optic neuropathy (LHON). The primary endpoint, proportion of eyes with clinically relevant benefit after 12 months treatment with Raxone versus untreated patients from an external control group, was met with high statistical significance (p=0.002). The efficacy data confirm and extend previous findings which demonstrated that Raxone can prevent further vision loss and promote recovery of vision in LHON patients.
Chiesi’s rare disease medicine Raxone has been recommended for use within NHS Wales for the treatment of visual impairment in adolescent and adult patients with Leber’s Hereditary Optic Neuropathy (LHON).
This recommendation applies only in circumstances where the approved Wales Patient Access Scheme (WPAS) is utilised or where the list/contract price is equivalent or lower than the WPAS price.
Pratteln, Switzerland, May 20, 2020 – Santhera Pharmaceuticals (SIX: SANN) announces full recruitment of its Phase 3 SIDEROS study with idebenone in Duchenne muscular dystrophy (DMD). The sample-size and variability re-assessment performed according to study protocol demonstrated that with the currently enrolled patients the study has a very high power (>99%). Given the strong powering of SIDEROS, the Company is now assessing the potential of conducting an interim analysis to test for overwhelming efficacy with a view of completing the trial early.
Santhera expects an opinion by the CHMP around mid 2020.
Santhera Pharmaceuticals has submitted a Marketing Authorisation Application (MAA) to the European Medicines Agency (EMA) for Puldysa (idebenone) in duchenne muscular dystrophy.
Chiesi agrees to license Raxone from Santhera for all indications, also picking up the option to outright acquire the asset.
Santhera Pharmaceuticals has entered into an exclusive license agreement with Chiesi Group Farmaceutici, under which Chiesi will in-license Raxone for the treatment of LHON for a total consideration of as much as €93 million, comprising an upfront payment of €44 million and near- to mid-term sales milestone payments of as much as €49 million.
UK regulators have renewed the scientific opinion for Santhera’s Raxone, extending its availability to patients with Duchenne muscular dystrophy (DMD) through the Early Access to Medicines Scheme (EAMS) for another year.