US FDA still appears to be open, if not optimistic, about path towards broader label for patisiran, the first-of-its-kind targeted RNA-based therapy.
Alnylam Holding Co. made headlines Aug. 10 when the US FDA approved Onpattro (patisiran) as a “first-of-its-kind targeted RNA-based therapy,” but the company landed a narrower indication than it was initially hoping for.
The agency specifically cleared the small interfering RNA (siRNA) therapy for the treatment of adults with polyneuropathy caused by hereditary transthyretin-mediated amyloidosis (hATTR), a rare and often fatal genetic disease that consists of the
Plozasiran, Arrowhead Pharmaceuticals' treatment for familial chylomicronemia syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.
The company’s failure to show its potency assays measured attributes directly related to the mesenchymal stromal cell product’s therapeutic effect highlights the importance of a robust CMC program for complex cell therapies.
Agency staff repeatedly said the BLA based on a single-arm study in 55 patients lacked substantial evidence of effectiveness in steroid-refractory acute graft-versus-host disease and a randomized trial was needed, but changed course “based on additional consideration” after a second CRL.
The Pink Sheet’s Drug Review Profile looks at the timeline for the clinical development and US FDA review of Mesoblast’s remestemcel for graft-versus-host disease.
Two US FDA advisory committees will discuss results from epidemiological studies that attempted to quantify the prevalence, incidence and risk factors for misuse, abuse, overdose and death with opioid analgesics.
CEO Len Schleifer said during the company’s first quarter earnings call that most of the complete response letters the company has received related to third-party suppliers, not efficacy or safety.
Plozasiran, Arrowhead Pharmaceuticals' treatment for familial chylomicronemia syndrome, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.
