Hemophilia A Gene Therapy

New drug for Hemophilia A Gene Therapy approved by USFDA.

US FDA approved Hemophilia A Gene Therapy. Hemophilia is a disease which causes uncontrolled excessive bleeding, due to absence of clotting factors which help in formation of solid clot that prevents excessive bleeding and wound healing. Patients affected with Hemophilia suffer bleeding bruises, bleeding in the joint due to internal injuries, blood in urine, and stool, excessive bleeding during periods, bleeding from nose, and swelling in joints.

There are two types of Hemophilia. Hemophilia A and Hemophilia B.

Hemophilia B.

Hemophilia B is a disease condition occurring due to faulty gene which do not produce factor IX which is responsible for blood clotting process of formation of semisolid blood clot, in absence of factor ix, uncontrolled bleeding from damaged tissue occurs, since the clot formation do not occur in Hemophilia B patients, sometimes the bleeding is spontaneous, from inner organs, intestine or oral cavity, etc., it can occur at any site.

Hemophilia B is X chromosome linked disease, which is inherited mostly predominantly to males, some females too suffer from Hemophilia B, patients do not have a gene that produces factor IX.

First of its kind New drug gene therapy approved by US FDA to treat Hemophilia B adult patients.

Hemophilia A :

Hemophilia A is a genetic disorder where the patients affected do not have genes that produce factor VIII (FVIII), it is an important factor which facilitates formation of clot invents of bleeding.

Earlier US FDA had approved a gene therapy drug to treat patients with Hemophilia B in June 29, 2023 US FDA has also approved a new drug a gene therapy for treatment of Hemophilia A. Hemophilia A results in severe bleeding in kidney and brain.

Hemophilia A Gene Therapy
Hemophilia A Gene Therapy

Therefore it was necessary to have a treatment for Hemophilia A. There was limited option for treatment for Hemophilia A which included, intravenous injections of factor VIII (FVIII) which would limit the disorder temporarily. With the approval of gene therapy which is a one time injection of viral vector carrier which is used to deliver the gene in the blood cells.

Name of the gene therapy drug approved for Hemophilia A is Roctavian.

It is approved by the US FDA under orphan drug designation. Orphan drugs are drugs which are approved by the US FDA for diseases which do not have any treatment option or have very limited treatments.


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