Baudax Bio BXRX announced that the FDA has granted the Orphan Drug designation to its next-generation, FVIII specific Treg therapy, TI-168, for treating patients with hemophilia A with inhibitors.
The designation is granted to experimental treatments intended for rare diseases affecting less than 200,000 individuals in the United States. It expedites the development and review of drugs, and also grants seven years of post-approval marketing exclusivity.
In June 2022, the FDA approved an investigational new drug application (IND), seeking to initiate a clinical study on TI-168 to treat hemophilia A patients with inhibitors. Following the approval, BXRX plans to initiate phase I/IIa study on TI-168 by early 2024.
Hemophilia A is a rare genetic blood disorder which is caused by a missing clotting protein known as FVIII. The protein prevents normal blood from clotting. If the blood doesn’t clot properly, it can lead to painful bleeding inside the joints that can cause scarring and damage.
Roche’s Hemlibra (emicizumab) was the first approved treatment for the disease. Emicizumab is a bispecific antibody mimicking the action of FVIII. It is administered subcutaneously. Hemlibra continues to perform well and the drug is one of the top contributors to Roche’s sales growth. In the first half of 2023, RHHBY generated revenues of CHF 2.1 billion from the drug, indicating 20% growth year over year.
Sanofi’sAltuviiio (efanesoctocog alfa), a new class of FVIII therapy for hemophilia A, was approved by the FDA in February 2023. The drug was launched in the United States in March 2023. Sanofi generated sales of 19 million euros from Altuviiio in the first half of 2023.
Pfizer is developing its investigational anti-tissue factor pathway inhibitor, marstacimab, as a treatment for hemophilia A or B patients, without inhibitors to FVIII or Factor IX. In May, the company reported upbeat data from the phase III BASIS study that evaluated marstacimab for hemophilia A or B patients. The study met its primary endpoints, which showed that those treated with marstacimab achieved a statistically significant and clinically relevant reduction in annualized bleeding rate compared with the current standard-of-care treatment for hemophilia patients.
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