Regulatory
Friday’s FDA approval of Vertex-CRISPR’s Casgevy and bluebird bio’s Lyfgenia has immediately revealed startling differences between these two gene therapies: price and a black-box warning.
Along with CRISPR/Cas9-based Casgevy—developed by Vertex Pharmaceuticals and CRISPR Therapeutics—the regulator on Friday approved bluebird bio’s Lyfgenia, a second gene therapy for sickle cell disease.
As Vertex and CRISPR Therapeutics’ exa-cel and Verve Therapeutics’ VERVE-101 move forward, questions remain about possible drawbacks of such therapies.
The regulator has accepted Bristol Myers Squibb’s Opdivo and the chemotherapy cisplatin for priority review in patients with unresectable metastatic urothelial carcinoma, as well as Merck and Seagen’s first-line combo.
The regulator placed a partial clinical hold on Roche’s fenebrutinib—being developed for relapsing MS—after two patients experienced elevated hepatic transaminase and bilirubin levels indicative of liver injury.
The European Medicines Agency is seeking additional information from the makers of GLP-1 drugs as part of its ongoing review of the potential risk of suicide and self-harm thoughts associated with the class.
This week, the FDA could approve the first CRISPR-edited therapy in the U.S., while two other companies await decisions on topical drugs.
The regulator accepted Karuna Therapeutics’ NDA and set a PDUFA date of September 26, 2024. If approved, it would be the first new mechanism of action to treat schizophrenia in decades, the company contends.
The troubled Indian pharma company received its second FDA warning letter in months, which this time cited quality control and data integrity lapses at its manufacturing facility in Gujarat, India.
The agency on Tuesday said it is investigating the “serious risk” of T cell malignancy outcomes, including hospitalization and death, and evaluating the need for potential regulatory action.
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