Agency grants same-day approval to the first two gene therapies for sickle cell disease; bluebird’s lentiviral-based lovo-cel carries a boxed label warning on hematologic malignancies, while Vertex’s CRISPR-Cas9 exa-cel carries a warning and precaution about potential off-target effects. Only the Vertex product qualified for a rare pediatric disease priority review voucher.
Although the US Food and Drug Administration approved the first two gene therapies for sickle cell disease in a sweeping same-day action, product labeling reflects potential risks specific to each therapy's mechanism of action and clinical program to date.
On 8 December, the FDA approved Vertex Pharmaceuticals Incorporated/CRISPR Therapeutics AG’s Casgevy (exagamglogene autotemcel, or exa-cel) on the...
<p>Executive Summary</p> <p>A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.</p>
<p>Executive Summary<p> <p>The Pink Sheet's list of EU centralized approvals of new active substances has been updated to add six new products, incl/ding Ixchiq, Valneva's chikungunya virus vaccine</p>
<p>Executive Summary</p> <p>A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.</p>
<p>Executive Summary</p> <p>Zanidatamab, Jazz and BeiGene’s investigational treatment for biliary tract cancer, is among the latest products that have been filed for review by the European Medicines Agency for potential EU marketing approval.</p>
<p>Executive Summary</p> This is an update of recommendations from the European Medicines Agency's Committee for Medicinal Products for Human Use on the authorization of new medicines in the EU, and updates on EU marketing authorization changes recommended by the CHMP.
<p>Executive Summary</p> <p>A list of EU biosimilar filings, CHMP opinions and EU marketing authorizations, including details of the biosimilar company, the brand name/INN, indication(s), the reference product/company, and the date and type of event.</p>
