-
Categories
-
Pharmaceutical Intermediates
-
Active Pharmaceutical Ingredients
-
Food Additives
- Industrial Coatings
- Agrochemicals
- Dyes and Pigments
- Surfactant
- Flavors and Fragrances
- Chemical Reagents
- Catalyst and Auxiliary
- Natural Products
- Inorganic Chemistry
-
Organic Chemistry
-
Biochemical Engineering
- Analytical Chemistry
-
Cosmetic Ingredient
- Water Treatment Chemical
-
Pharmaceutical Intermediates
Promotion
ECHEMI Mall
Wholesale
Weekly Price
Exhibition
News
-
Trade Service
BioMarin's therapy, called valoctocogene roxaparvovec or 'valrox', in the Phase 3 GENER8-1 trial, 8 of 20 adults with severe hemophilia A had sufficient improvements in factor VIII levels to meet prespecified regulatory review standards
.
In June 2019, BioMarin reported three years of data on its gene therapy for hemophilia A, which was used to support its applications in Europe and the US, however, its long-term therapeutic value has also been called into question
.
Read more: BioMarin's Phase III hemophilia gene therapy hits endpoint, but its long-term effects raise questionsBioMarin reports three-year data on its gene therapy for hemophilia A that supports BioMarin's hemophilia gene therapy Phase III clinical end point, but its long-term effects are questioned
On January 9, 2022, BioMarin Pharmaceutical announced that valoctocogene roxaparvovec has achieved positive results in an ongoing Phase III clinical trial
.
This is an investigational gene therapy using an AAV5 viral vector to deliver a transgene expressing coagulation factor VIII for the treatment of adults with severe hemophilia
A.
Trial results showed that in 112 subjects (median follow-up of 110 weeks), valoctocogene roxaparvovec reduced the patient's annual bleeding rate (ABR) by 85% compared to a baseline value of 4.
FDA
However, it was also seen that the activity of FVIII continued to decrease with prolonged follow-up
However, the gene therapy will have a high price once it comes out, see: BioMarin plans to price its hemophilia A gene therapy Valrox at $2 million to $3 million
BioMarin plans to price its hemophilia A gene therapy Valrox at $2-3 million
