- Vertex Pharmaceuticals and CRISPR Therapeutics said Monday they have finished submitting their application for U.S. approval of a gene editing medicine for two rare blood disorders, beating a rival that has faced delays in advancing a similar type of treatment.
- The Food and Drug Administration now has until early June to decide whether to accept the application for their medicine, which is named exa-cel and treats sickle cell disease and beta thalassemia. If the FDA does and begins a “priority,” or expedited, review, the agency could make a decision later this year.
- The news comes days after competitor Bluebird bio, which is developing a gene therapy for sickle cell, missed its goal to submit an application by the end of March. Bluebird is waiting for FDA feedback on its manufacturing protocol and anticipates hearing back “within a matter of weeks,” it said Wednesday.
The FDA’s review of exa-cel will be a milestone for genetic medicine.
The treatment could become the first marketed medicine based on CRISPR, the landmark gene editing technology that won a Nobel Prize in 2020. Last September, the FDA allowed Vertex and CRISPR to begin a rolling approval application after clinical testing showed treatment could alleviate the most serious symptoms of both sickle cell disease and beta thalassemia.
The review — and whether the regulator schedules an advisory committee meeting to discuss the application — will provide a “window into how FDA thinks about gene editing,” wrote Stifel analyst Dae Gon Ha, in a research note on Monday.
An FDA OK would bring to the U.S. another gene-based treatment for inherited diseases. Last year, the regulator approved three treatments for rare blood and brain diseases, adding to earlier clearances for gene therapies used to treat vision loss and spinal muscular atrophy. Two more could come soon, as the FDA is currently reviewing genetic medicines for Duchenne muscular dystrophy and hemophilia.
For sickle cell and beta thalassemia patients, exa-cel offers the potential of long lasting, if not lifelong, benefit.
Sickle cell causes painful and life-threatening blockages triggered by misshaped red blood cells, while beta thalassemia hinders formation of functional hemoglobin, an essential oxygen-carrying protein.
Beta thalassemia patients in the U.S. already have one genetic medicine available to them, Bluebird’s gene therapy Zynteglo, which the FDA approved last year. Sickle cell patients don’t yet, but that could soon change with the review of exa-cel and, in the near future, Bluebird’s treatment as well.
The completion of Vertex and CRISPR’s application puts the partners a step ahead of Bluebird in the U.S. They’ve also completed applications in Europe and the U.K.