The biotech company said it would price Zynteglo for beta-thalassemia at about $1.8 million for the European market. Data for the therapy in that disease and also sickle cell disease are being presented at EHA.
Two beta-thalassemia advocacy organizations - one British and one Italian - issued a press release Monday that claimed the EMA had approved the gene therapy under the brand name Zynteglo.
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The company is developing a drug that targets the same pathway as the chemotherapy agent hydroxyurea, but is designed for a better safety profile.
The Phase I/II trial is in beta-thalassemia, a rare blood disorder. Another Phase I/II study of the same drug, in sickle cell disease, is expected to infuse its first patient in mid-2019.
A company analysis deemed the patient's myelodysplastic syndrome was unrelated to LentiGlobin, but a news report indicated it may have stemmed from the conditioning regimen used in the trial.
The agency also accepted the two companies' IND to begin the Phase I/II study of CTX001 in patients with sickle cell disease, sending CRISPR shares up more than 11 percent Thursday morning.
A new report by Paubox calls for healthcare IT leaders to dispose of outdated assumptions about email security and address the challenges of evolving cybersecurity threats.
FDA slaps clinical hold on Crispr Therapeutics and Vertex Pharmaceuticals' upcoming gene-editing trial.