GenSight Sees Positives In Pivotal RESCUE Trial, Despite Missing Primary Endpoint
The company's gene therapy didn't hit the primary endpoints in either of its two Phase III studies in a severe genetic retinal disease reported so far, but a third trial designed through a special protocol assessment with the US FDA is ongoing.
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Phase III success at 96 weeks sets up GenSight blindness therapy for EU filing, but puzzle of placebo arm benefits remain
Pipeline Watch is a weekly snapshot of selected late-stage clinical trial events announced by pharmaceutical and biotech companies at medical and industry conferences, in financial and company presentations, and in company releases and statements.
Santhera Pharmaceuticals' Raxone (idebenone) has garnered a positive recommendation from the European Medicines Agency's Committee for Medicinal Products for Human Use (CHMP) for the treatment of visual impairment in adolescent and adult patients with Leber's Hereditary Optic Neuropathy (LHON).