Valoctocogene roxaparvovec
- Generic Name
- Valoctocogene roxaparvovec
- Brand Names
- Roctavian
- Drug Type
- Biotech
- Chemical Formula
- -
- CAS Number
- 1819334-78-5
- Unique Ingredient Identifier
- 681K1JDI8M
- Background
Valoctocogene roxaparvovec is an adeno-associated virus serotype 5 (AAV5) based gene therapy vector that expresses the B-domain deleted SQ form of human coagulation factor VIII (hFVIII-SQ). The expression of hFVIII-SQ is driven by a liver-specific promoter, which enables hepatocytes to produce factor VIII protein and increase the levels of active factor VIII...
- Indication
Valoctocogene roxaparvovec is indicated for the treatment of severe hemophilia A (congenital factor VIII deficiency) in adult patients without a history of factor VIII inhibitors and without detectable antibodies to adeno-associated virus serotype 5 (AAV5).
- Associated Conditions
- Severe Hemophilia A
- Associated Therapies
- -
Gene Therapy Study in Severe Hemophilia A Patients With Antibodies Against AAV5
- Conditions
- Interventions
- First Posted Date
- 2018-05-11
- Last Posted Date
- 2024-04-24
- Lead Sponsor
- BioMarin Pharmaceutical
- Target Recruit Count
- 3
- Registration Number
- NCT03520712
- Locations
- 🇨🇳
Kaohsiung Medical University Chung-Ho Memorial Hospital, Kaohsiung, Taiwan
🇨🇳National Taiwan University Hospital, Taipei, Taiwan
🇰🇷Kyung Hee University Hospital at Gangdong, Seoul, Korea, Republic of
Drug Development Updates
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