-
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
CompilationTom Lee
A few days ago, the U.
The FDA's priority review decision to accept the therapy was based on data from two Phase 3 studies of the therapy, Northstar-2 and Northstar-3, and a long-term follow-up study, LTF-303
Beta-thalassemia is a serious genetic disorder in which patients have genetic mutations that lead to significantly lower levels of hemoglobin, leading to severe anemia and lifelong dependence on blood transfusions
Anne Virginie Eggimann, chief regulatory officer of Bluebird Bio, said that for a long time, beta-thalassemia patients who depended on regular blood transfusions had to endure a huge burden associated with the disease
Regulatory filing for the gene therapy beti-cel has been a long road for Massachusetts-based Bluebird Bio, which reported its Phase 3 data in June 2017 and received it in Europe two years later approved
In Germany, the only country that has released pricing details, local regulators have offered to pay $790,000 per patient, which would rise to $950,000 after five years if the treatment was still effective
Reference source: Bluebird bio's beti-cel accepted for priority review by FDA
