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Hemophilia A

All articles tagged with #hemophilia a

healthcare2 years ago•4 min saved

"Baudax Bio's TI-168 Receives FDA Orphan Drug Designation for Hemophilia A with Inhibitors"

Baudax Bio has announced that the U.S. FDA has granted orphan drug designation to its lead clinical candidate, TI-168, for the treatment of Hemophilia A with inhibitors. TI-168 is a next-generation T cell receptor therapy designed to address Hemophilia A patients with FVIII inhibitors. The designation provides various benefits to support the development of novel drugs, including potential market exclusivity for seven years upon FDA approval. Baudax Bio plans to activate the Phase 1/2a clinical trial of TI-168 in early 2024.

via Yahoo Finance|

#baudax-bio #fda #healthcare

healthcare2 years ago•0 min saved

Roche Advances Hemophilia Gene Therapy in Phase III, Terminates Another

Roche has announced the removal of RG6358, or SPK-8016, from its gene therapy pipeline for hemophilia A, while initiating a Phase III trial for RG6357, or SPK-8011. The company plans to open enrollment for the study, named Keystone1, later this year.

via Endpoints News|

#clinical-trial #gene-therapy #healthcare

health2 years ago•1 min saved

FDA Approves BioMarin's Gene Therapy for Hemophilia A

The FDA has approved Roctavian, a gene therapy treatment for severe hemophilia A, a rare and potentially fatal blood disorder. The treatment involves a single infusion that inserts missing genes and could eliminate the need for frequent injections. Roctavian is the latest in a series of FDA-approved gene therapies, signaling the potential for gene therapy to revolutionize the treatment of various diseases. However, the high cost of gene therapies remains a significant barrier to widespread use.

via The Washington Post|

#fda #future-of-medicine #gene-therapy

health2 years ago•3 min saved

FDA Approves BioMarin's Gene Therapy for Severe Hemophilia A

The FDA has approved Roctavian, the first gene therapy for adults with severe hemophilia A. Hemophilia A is a rare genetic bleeding disorder caused by a deficiency in clotting factor VIII. Roctavian is a one-time gene therapy administered through intravenous infusion, which increases blood levels of factor VIII to reduce the risk of uncontrolled bleeding. The safety and effectiveness of Roctavian were evaluated in a study of adult men with severe hemophilia A, and it was found to significantly reduce bleeding rates. Common side effects include mild changes in liver function, headache, nausea, and fatigue. The approval of Roctavian provides a new treatment option for patients with severe hemophilia A.

via FDA.gov|

#bleeding-disorder #fda #gene-therapy

healthcare2 years ago•0 min saved

FDA Approves BioMarin's Gene Therapy Roctavian for Hemophilia A

The FDA has approved BioMarin's gene therapy, Roctavian, for the treatment of hemophilia A, following a turbulent regulatory journey that included a surprise rejection in 2020 and a review extension earlier this year. BioMarin aims to commercialize Roctavian and hopes it will become a key blockbuster drug, potentially doubling the company's revenues to $4 billion to $5 billion by the middle of the decade. The list price for the therapy has not been disclosed yet.

via Endpoints News|

#biomarin #commercialization #fda-approval