The FDA approved Vertex Pharmaceuticals’ gene therapy Casgevy for treating beta thalassemia, an inherited blood disease that leads to low levels of functioning hemoglobin. Last month, the one-time treatment won its first FDA nod for treating sickle cell disease.
Casgevy, from Vertex Pharmaceuticals and CRISPR Therapeutics, is now approved in the United Kingdom for treating the blood disorders sickle cell disease and beta thalassemia. It’s the first regulatory approval in the world for a CRISPR-based therapy.
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Phase III data on bluebird bio's Zynteglo, as well as Vertex and CRISPR Therapeutics' CTX001, showed patients achieving transfusion independence via two gene therapy approaches.
An analyst wrote that it is unlikely bluebird would provide full data from its registration-directed study of the CAR-T bb2121 by year end, which is more likely to come early next year.
An analyst wrote that bluebird bio would likely launch Zynteglo first in Germany, followed by other countries, but scaled back sales forecasts due to the therapy's high price tag of 1.575 million euros.
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.
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Approval of luspatercept in myelodysplastic syndrome and beta-thalassemia is expected in April 2020, or as early as December of this year if it receives FDA priority review, an analyst wrote.
An analyst called the beta-thalassemia data - for a cell therapy gene-edited using zinc finger nuclease technology - early but a solid proof of concept for the technology's capability.
The EMA's Committee for Human Medicinal Products recommended a conditional approval for Zynteglo in beta-thalassemia. A third-party press release erroneously stated the agency had approved the product earlier this week.
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.
FDA slaps clinical hold on Crispr Therapeutics and Vertex Pharmaceuticals' upcoming gene-editing trial.