FDA grants fast track status to 1-time stem cell therapy for hemophilia A
WHY IT MATTERS
If successful, this one-time stem cell therapy could eliminate the need for regular blood clotting factor infusions that hemophilia A patients currently require multiple times per week.
The FDA has given special fast-track status to a new stem cell therapy being developed by Expression Therapeutics for hemophilia A, a bleeding disorder. This designation means the therapy can be reviewed faster than usual. The therapy is designed to be a one-time treatment that could provide lasting relief from hemophilia A symptoms.
The U.S. Food and Drug Administration (FDA) has granted both fast track and rare pediatric disease designations to Expression Therapeutics’ investigational stem cell therapy for hemophilia A. Each of these designations is intended to speed development of a candidate therapy. This stem cell therapy was designed as a potentially lasting, one-time treatment for hemophilia A, a […] The post FDA grants fast track status to 1-time stem cell therapy for hemophilia A appeared first on Hemophilia N
ASK YOUR DOCTOR
Ask your hematologist if you or your child might be eligible for upcoming clinical trials of this therapy, as fast track status suggests trials could begin or expand soon.