A revolutionary breakthrough could come for patients with hemophilia | News of business markets and actions


Patients diagnosed with hemophilia B suffer a lifetime of treatment, but that could change based on recently reported data from an early-stage trial Pfizer (NYSE: PFE) and ] Spark Therapeutics [19659003] (NASDAQ: ONCE) are performing. The two companies joined SPK-9001, a gene therapy that hopes to offer a "unique and complete" functional cure for the disease. This week, The New England Journal of Medicine a prestigious industry magazine, published updated results from a study of 10 patients who were quite remarkable. Can hemophilia become a curable disease?

Progress towards a revolutionary treatment

The results of the company's phase 1/2 study show that a single dose of SPK-9001 caused patients to produce their own coagulation factor and, as a result, the number of Hemorrhages per year for these patients decreased by 97% and the need for an infused coagulation factor was reduced by 99%. Previously, these patients experienced an average of 11.1 hemorrhagic events per year, but after receiving SPK-9001, they suffered an average of only 0.4 per year.


According to the World Federation of Hemophilia, there are 151,000 people worldwide with hemophilia A and almost 30,000 people with hemophilia B. In both diseases, patients are at risk of severe bleeding and life-threatening hemorrhages due to their inability to produce a protein necessary for blood clotting. In hemophilia A, coagulant factor VIII is missing or insufficiently produced; and in hemophilia B, it is coagulant factor IX.

Currently, patients with hemophilia A and B receive prophylactic infusions of the missing coagulation factor to limit bleeding and prevent the badociated joint damage that causes the disease. However, these products are not ideal because they require regular visits to specialized hemophilia treatment centers and expose patients to the risk of complications, including unwanted immune reactions to coagulation factor concentrates.

Unlike these other existing treatment options, SPK-9001 is a genetic therapy that essentially corrects the ability of a patient with hemophilia B to produce coagulation factor IX. It does this by using an inactivated adeno-badociated virus to deliver a functional factor IX gene that allows patients to produce their own factor IX. Since they produce their own coagulation factor, the need for prophylactic infusions of factor IX could be eliminated.

More work to be done

It is true that the study is small and the research is ongoing, but the results are nonetheless impressive. The findings could transform the quality of life of patients with hemophilia B and potentially save patients and payers a large amount of money in the process.

If finally approved, SPK-9001 will likely be launched at a high tag price, but its price may seem like a bargain when compared to the costs badociated with a lifetime of treatment for hemophilia B and the potential risk of hospitalization. for hemorrhagic events.

More research is needed on SPK-9001 before it can win an FDA. go ahead and be widely available. But if this research confirms the results seen so far, it could be a great victory for everyone, including Pfizer and Spark Therapeutics. The drug market for hemophilia is worth billions of dollars per year, and SPK-9001 could one day become standard care. If the regulators finally approve SPK-9001, then Pfizer will market it globally and will pay Spark Therapeutics a royalty.

However, the implication for Spark Therapeutics could be even greater. That's because Spark Therapeutics is also working on a gene therapy for patients with hemophilia A. If you can duplicate your success there, then you could be in a position to benefit significantly because you have not yet sold the rights to that drug.

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Todd Campbell owns shares in Pfizer. Your customers can have positions in the companies mentioned. The Motley Fool has no position in any of the actions mentioned. The Motley Fool has a disclosure policy.

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