Piper Jaffray expects today's FDA approval of Orkambi to accelerate top-line growth and return Vertex to sustainable profitability. Piper thinks the cystic fibrosis combo will be priced in line with the company's Kalydeco drug at $311,000 per year, except with steeper gross-to-net discounts due to the larger patient population. It reiterates an Overweight rating on Vertex with a $148 price target.

Vertex Pharmaceuticals announced that the European Commission has granted approval for the label expansion of KALYDECO for the treatment of infants down to 1 month of age with cystic fibrosis who have one of the following mutations in the cystic fibrosis transmembrane conductance regulator gene: R117H, G551D, G1244E, G1349D, G178R, G551S, S1251N, S1255P, S549N or S549R. As a result of existing access agreements in Austria, Czech Republic, Denmark, Ireland, Norway, Sweden, and The Netherlands, eligible patients will have access to the expanded indication of KALYDECO shortly following regulatory approval by the European Commission. Vertex will continue to work with reimbursement authorities across the European Union to ensure access for all other eligible patients. In the U.K., following MHRA approval at the end of 2023, and as a result of the existing reimbursement agreement between Vertex and the National Health Service, eligible infants ages 1 month and older in the U.K. have access to this expanded indication for KALYDECO.