We use cookies to understand how you use our site and to improve your experience.
This includes personalizing content and advertising.
By pressing "Accept All" or closing out of this banner, you consent to the use of all cookies and similar technologies and the sharing of information they collect with third parties.
You can reject marketing cookies by pressing "Deny Optional," but we still use essential, performance, and functional cookies.
In addition, whether you "Accept All," Deny Optional," click the X or otherwise continue to use the site, you accept our Privacy Policy and Terms of Service, revised from time to time.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
Baudax's (BXRX) Hemophilia A Drug Gets FDA's Orphan Drug Tag
Read MoreHide Full Article
Baudax Bio 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.
Several other companies like Genentech (subsidiary of Roche [(RHHBY - Free Report) ]), Sanofi (SNY - Free Report) and Pfizer (PFE - Free Report) have developed or are developing treatment for hemophilia A.
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.
See More Zacks Research for These Tickers
Normally $25 each - click below to receive one report FREE:
Image: Bigstock
Baudax's (BXRX) Hemophilia A Drug Gets FDA's Orphan Drug Tag
Baudax Bio 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.
Several other companies like Genentech (subsidiary of Roche [(RHHBY - Free Report) ]), Sanofi (SNY - Free Report) and Pfizer (PFE - Free Report) have developed or are developing treatment for hemophilia A.
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.