On Tuesday (January 16th) local time, American biotech company Vertex Pharmaceuticals announced on its official website that the US Food and Drug Administration (FDA) approved its Casgevy therapy for the treatment of "transfusion dependence" in patients aged 12 and above that day β Mediterranean anemia (TDT).
It is understood that Casgevy was jointly developed by Foton Pharmaceuticals and CRISPR Treatment Company in Switzerland. It extracts stem cells from the patient's bone marrow, and then edits the genes in the cells in the laboratory - using molecular "scissors" to trim the defective parts of the genes, and finally transfusing them back into the patient's body to produce functional hemoglobin.
A month ago, the FDA first approved Foton Pharmaceuticals' gene therapy for the treatment of sickle cell disease (SCD), becoming the world's first approved "gene scissors" CRISPR therapy. At that time, the company also expected the approval date for TDT indications to be around March 30, 2024. This means that the FDA's action is more than two months ahead of expectations.
Vertex CEO Reshma Kewalramani wrote in the latest announcement that following the FDA's historic approval of Casgevy for the treatment of SCD, it is very exciting that it has now obtained approval for the treatment of TDT earlier.
Foton Pharmaceuticals introduced in a press release that TDT is a serious and life-threatening hereditary blood disease, and patients may need frequent blood transfusions and the use of iron chelators for life. Due to anemia, patients may experience fatigue and shortness of breath, and infants may experience developmental delays. The median age of death for patients is 37 years old.
According to data from Boston Children's Hospital, it is estimated that over 100000 people worldwide suffer from TDT, with at least 1200 patients in the United States. Kewalraman added, "TDT patients deserve new curative options, and we expect the therapy to be delivered to eligible patients who are waiting."
Like SCD, the only clinical cure for TDT before this was hematopoietic stem cell transplantation, and drug innovation and development were also relatively slow. Nowadays, gene therapy has become a new method for treating this disease. A week ago, the Saudi Food and Drug Administration (SFDA) had already granted market authorization for the treatment of SCD and TDT to Casgevy.
But Casgevy's pricing is also very expensive, with treatment costs of up to $2.2 million per patient. Regarding this, investment bank Oppenheimer analyst Hartaj Singh said that data shows that Casgevy has a good effect, and he expects its peak sales to be close to $400 million.
Analysis suggests that gene therapy can target the root causes of diseases and even achieve therapeutic effects, and its rapid development can be applied to various disease fields, which may open a new chapter in the pharmaceutical field. Research institutions predict that the global gene editing market will reach $36 billion by 2030, with a compound annual growth rate of 22.3%.
Last year, the stock price of Foton Pharmaceuticals, which was listed on NASDAQ, rose by 40%, and this year it has risen by over 7.5% on this basis. As of Tuesday's close, the company's market value was $112.73 billion, approaching the multinational pharmaceutical giant Sanofi.