Vertex CRISPR therapy demonstrates early success in children with sickle cell and thalassemia, potentially expanding its market reach.
Browsing: Regulatory & Approvals
Regulatory updates, drug approvals, submissions, and compliance developments from authorities like FDA, EMA, and others.
Praxis shares rise 40% following positive updates on two drugs, while Bristol Myers’ Breyanzi gains approval for a fifth cancer indication.
CDC panel delays vote on hepatitis B vaccine for newborns, impacting public health.
• CDC panel delays decision on hepatitis B vaccine.
• Vote affects millions of newborns annually.
• Public health implications are significant.
FDA chiefs critique plans to toughen vaccine standards, impacting COVID-19 protocols.
UniQure Huntington’s gene therapy faces challenges as FDA deems current data insufficient for approval, affecting market outlook.
Capricor Duchenne cell therapy shows positive results, potentially reversing FDA rejection and quadrupling shares.
FDA animal testing guidance reduces non-human primate use, aiding U.S. drugmakers amid global competition.
Belite Stargardt drug shows promising results in a late-stage trial, positioning it for potential regulatory approval.
Otsuka secures FDA approval for its first anti-APRIL therapy targeting IgA nephropathy, marking a significant advancement in rare kidney disease treatment.
Otsuka FDA Approval marks a significant milestone with the first anti-APRIL therapy for IgA nephropathy, impacting the rare kidney disease market.