Another gene therapy coming? FDA updates regulatory progress for hemophilia therapy

Today, BioMarin Pharmaceutical announced that it has been notified that the FDA no longer plans to hold an advisory committee meeting to discuss the Biologics License Application (BLA)
for Roctavian (valoctocogene roxaparvovec).
Roctavian's hemophilia A gene therapy for BioMarin, a decision that trade media outlet Endpoints pointed out, may mean that the innovative therapy will have a smoother journey to market
.

Hemophilia A is a rare inherited bleeding disorder
caused by the lack of clotting factor VIII.
Deficiency of coagulation factors leads to insufficient thrombin production, resulting in coagulation disorders
.
Hemophilia A is characterized by recurrent bleeding and related complications, of which about 80% are joint hemorrhage, and its complications are mainly chronic hemorrhagic joint lesions, which can lead to severe joint deformities
.
At present, the main treatment of hemophilia A is regular transfusion of coagulation factor VIII, but frequent transfusion brings great inconvenience
to the patient's life.

Roctavian is a transgene
expressing coagulation factor VIII using an AAV5 viral vector.
Its advantage is that patients may only need to receive one treatment for liver cells to continuously express factor VIII, eliminating the need for long-term prophylactic factor injections
.
The US FDA granted this gene therapy breakthrough therapy designation (BTD) in 2017 and RMAT designation in 2021
.
The therapy has also received orphan drug designation (ODD)
from the US FDA and the European Medicines Agency (EMA).
On August 24 this year, the European Commission (EC) approved the conditional marketing of this gene therapy for the treatment of hemophilia A
.

Image source: 123RF

BioMarin's BLA submission contains the Company's response to a previous FDA Full Response Letter (CRL), as well as 2-year results from the Phase 3 trial of GENEr8-1 and 5-year follow-up support data
from the Phase 1/2 trial.
The results of the GENEr8-1 trial showed that Roctavian was able to control bleeding
stably and persistently.
After a single infusion of Roctavian, the rate of use of coagulation factor VIII and the annual bleeding rate requiring treatment were reduced by 99% and 84%, respectively, compared with data from the year prior to enrollment, (p<0.
001).

Overall, 90% (121/134) of trial participants had no bleeding events requiring treatment or fewer
bleeding events compared with prophylactic treatment with factor VIII.

"BLA review is a dynamic process, and we are grateful for the FDA's continued engagement as we deliver a potentially transformative therapy for hemophilia A patients," said Dr.
Hank Fuchs, President of Global Research and Development at BioMarin.

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