The European Medicines Agency said it is asking healthcare providers to conduct liver tests on patients treated with Zolgensma, after recent reports of deaths related to the gene therapy.
Tag Archive for: Zolgensma
With Novartis facing a potential loss of $9 billion through 2026 from patent expirations, the company announced topline results for Pluvicto for PSMA-positive metastatic castration-resistant prostate cancer (mCRPC).
A separation of Sandoz via a 100 percent spin-off will create the leading European generics company and a global leader in biosimilars, and a more focused Novartis.
Swiss pharma giant Novartis said it would make growth in the United States its top geographic priority, even after laws were passed to rein in drug prices in the world’s largest pharmaceutical market.
The U.S. Food and Drug Administration on Wednesday approved bluebird bio’s gene therapy for patients with a rare disorder requiring regular blood transfusions, and the drugmaker priced it at a record $2.8 million.
The two fatal cases of acute liver failure took place in Russia and Kazakhstan after 5 to 6 weeks of Zolgensma infusion and about 1-10 days following the initiation of corticosteroid taper, according to the company.
Even with the COVID-19 emergency measures, payers still had an eye on the potential flood of gene therapies for rare diseases brewing in the pipeline, and were pondering new ways to pay for them.
Advisers to the U.S. Food and Drug Administration on Thursday voted to recommend approval of bluebird bio’s treatment for a rare neurological disorder, bringing it closer to becoming the third gene therapy to be available in the United States.