Piper Jaffray analyst Edward Tenthoff said he is confident that the FDA will approve Vertex's Orkambi in homozygous F508del cystic fibrosis by its PDUFA date of July 5, meaning a decision should come by either late Thursday or by Monday morning, due to the holiday. Pricing will be key, Tenthoff noted, adding that he expects it to be in-line with Kalydeco's $311,000, except with steeper gross-to-net discounts. Tenthoff expects Vertex to retain its dominance of CF therapy and keeps his Overweight rating and $146 target on its shares, which closed down $4.84 near $122 yesterday.

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.