Express treatment of hemophilia A, FDA accepted marketing applications for potential blockbuster gene therapy

BioMarin Pharmaceutical announced that the US FDA has accepted the company's resubmitted biologics license application (BLA) for valoctocogene roxaparvovec, an AAV gene therapy under investigation, for the treatment of adult patients
with severe hemophilia A.
The FDA expects to make a review decision
by March 31 next year.
The press release states that if approved, Valoctocogene Roxaparvovec would be the first FDA-approved gene therapy
to treat hemophilia A.

Hemophilia A is a rare inherited bleeding disorder
caused by the lack of clotting factor VIII.
Deficiency of clotting factors leads to insufficient thrombin production, leading to coagulopathy
.
Hemophilia A has recurrent bleeding and related complications as the main clinical manifestations, about 80% of which are joint bleeding, 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 infusion brings great inconvenience
to the patient's life.

Valoctocogene roxaparvovec is a transgene
expressing coagulation factor VIII delivered using an AAV5 viral vector.
It has the advantage that patients may only need to receive one treatment and hepatocytes can continue to express factor VIII, eliminating the need for long-term prophylactic coagulation factor injections
.
The US FDA granted breakthrough therapy designation (BTD) and Advanced Regenerative Medicine Therapy designation (RMAT) designation (RMAT)
for gene therapy in 2017 and 2021, respectively.
The treatment is also granted Orphan Drug Designation (ODD) by 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 (trade name: Roctavian) for the treatment of hemophilia A
.

"This BLA resubmission brings us one step
closer to our goal of bringing gene therapy treatment options to patients with severe hemophilia.
" We look forward to working closely
with the FDA in reviewing this transformative therapy.
" Dr.
Hank Fuchs, President of Global R&D at BioMarin, said
.

As WuXi AppTec's CTDMO focused on cell and gene therapies, WuXi Biologics is committed to accelerating and transforming the development, testing, manufacturing and commercialization
of gene and cell therapies and other high-end therapies.
WuXi Biologics can help customers around the world bring more innovative therapies to market sooner rather than later, benefiting patients
.
If you have related business needs, please click the picture below to fill in the specific information
.