Shots:
- The company received the CHMP’s positive opinion for the label expansion of Kalydeco (Ivacaftor) to include the treatment of infants (aged 1mos.-4mos.) with cystic fibrosis with specific mutations in the CFTR gene (R117H/G551D/G1244E/G1349D/G178R/G551S/S1251N/S1255P/S549N/S549R)
- Kalydeco has been approved by the EU for the treatment of cystic fibrosis patients aged ≥4mos. with specific CFTR gene mutations
- Kalydeco is an oral medicine designed to enhance the ability of the CFTR protein to transport salt and water across the cell membrane thereby hydrating and clearing mucus from the airways
Ref: Vertex Pharmaceuticals | Image: Vertex Pharmaceuticals| Press Release
Related News:- Orna Collaborates with Vertex to Develop Gene Therapies for Sickle Cell Disease (SCD) and Transfusion-Dependent Beta Thalassemia (TDT)
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