Paris, France – The U.S. Food and Drug Administration (FDA) has granted fast track designation to SAR446597, a one-time intravitreal gene therapy for the treatment of geographic atrophy (GA) due to age-related macular degeneration (AMD). The fast track designation process aims to facilitate the development and expedite the review of...
treatment News
PARIS, France – Following the positive opinion by the European Medicines Agency’s Committee for Medicinal Products for Human Use on June 19, 2025, the European Commission has approved Sarclisa in combination with bortezomib, lenalidomide, and dexamethasone (VRd) for the induction treatment of adult patients with newly diagnosed multiple myeloma (NDMM) who...
Paris, France — Positive results from the LEAP2MONO phase 3 study (clinical study identifier: NCT05222906) demonstrated that venglustat met the primary and three out of four key secondary endpoints in adults and pediatric patients (12 years and older) with neurological manifestations of Gaucher disease type 3 (GD3), a rare lysosomal storage...
Paris, France – The European Commission has approved Wayrilz (rilzabrutinib), a novel, oral, reversible, Bruton’s tyrosine kinase (BTK) inhibitor, as a new treatment for immune thrombocytopenia (ITP) in adult patients who are refractory to other treatments. This follows the positive opinion by the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP)....
Pratteln, Switzerland – Santhera Pharmaceuticals (SIX: SANN) and ReveraGen Biopharma announce new clinical data of 2.5-year treatment outcome with vamorolone in patients with Duchenne muscular dystrophy (DMD). These Phase 2a long-term treatment data demonstrate a maintenance of treatment effect, equivalent to a delay of about two years in decline for...
Pratteln, Switzerland, and Rockville, MD, USA – Santhera Pharmaceuticals (SIX: SANN) and ReveraGen BioPharma, Inc (US: private) announce positive key results from the VISION-DMD study, demonstrating robust efficacy across multiple efficacy endpoints and favorable safety and tolerability of vamorolone in the treatment of patients with DMD. VISION-DMD is a pivotal...
Pratteln, Switzerland – Santhera Pharmaceuticals (SIX: SANN) today notes that Health Canada has approved AGAMREE® (vamorolone) for the treatment of Duchenne muscular dystrophy (DMD) in patients aged 4 years and older following Priority Review, marking the first approved treatment for the disease in Canada. The Canadian Neuromuscular Disease registry estimates...
Santhera Announces Completion of First 6-Month Period of Pivotal VISION-DMD Trial with Vamorolone in Duchenne Muscular Dystrophy Pratteln, Switzerland Santhera Pharmaceuticals (SIX: SANN) announces that the last patient has completed the last visit for the first period of the placebo-controlled pivotal VISION-DMD study with vamorolone in patients with Duchenne muscular...
Pratteln, Switzerland – 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...
Pratteln, Switzerland – Santhera Pharmaceuticals (SIX: SANN) announces positive results from its multiple ascending dose Phase 1b study with lonodelestat, a potent inhibitor of human neutrophil elastase (hNE), in patients with cystic fibrosis (CF). Lonodelestat is a potent and selective peptide inhibitor of human neutrophil elastase (hNE), currently being developed...