Alice Melao,  —

Articles by Alice Melao

EMA Regulatory Committee Grants Accelerated Assessment to Patisiran to Treat FAP

Alnylam Pharmaceuticals’ lead investigational therapeutic patisiran, for the treatment of hereditary ATTR (hATTR) amyloidosis, also known as familial amyloid polyneuropathy (FAP), was granted accelerated assessment by the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP). “We are pleased the CHMP has granted accelerated assessment for patisiran…

Inotersen, Potential FAP Treatment, Under Review for Approval in Europe

Ionis Pharmaceuticals has asked the European Medicines Agency (EMA) to approve its lead drug candidate inotersen to treat hereditary TTR amyloidosis (ATTR), also known as familial amyloid polyneuropathy (FAP). The request, known as a Marketing Authorization Application (MAA), is supported by positive data from the Phase 3 NEURO-TRR trial, and…

Inotersen Seen to Improve Neurological Symptoms, Quality of Life of FAP Patients in Phase 3 Trial

The investigative drug inotersen was shown to significantly reduce neurological progression and improve quality of life in patients with familial amyloid polyneuropathy (FAP) – also referred to as hereditary transthyretin amyloidosis with polyneuropathy (hATTR-PN). These findings resulted from the Phase 3 NEURO-TTR study (NCT01737398), and were subject of a…