Vanzacaftor

Orphan drug sales reached $168 billion globally in 2023, with growth expected to moderate to just under 10% annually through 2028, according to Evaluate's latest report.

The European Medicines Agency (EMA) has recommended eight new products for EU-wide approval, expanding treatment options for various conditions.

The FDA approved Alhemo (concizumab) from Novo Nordisk for hemophilia A and B with inhibitors, offering a once-daily subcutaneous option.

The FDA has approved Vertex's ALYFTREK for cystic fibrosis (CF) in patients aged 6 years and older with specific genetic mutations.

Vertex Pharmaceuticals reported a 12% revenue increase in Q3 2024, reaching US$2.77 billion, driven by strong demand for its cystic fibrosis therapies.