How Did Kalydeco Perform in 4Q17 and 2017?


Feb. 6 2018, Updated 12:36 p.m. ET

Kalydeco revenue trends

In 4Q17, Vertex Pharmaceuticals’ (VRTX) Kalydeco generated revenues of $256 million, a ~45% increase on a year-over-year (or YoY) basis and 20% growth on a quarter-over-quarter basis.

In fiscal 2017, Kalydeco generated revenues of $845 million, a ~20% increase on a YoY basis. Increasing uptake among individuals above two years in the US with cystic fibrosis (or CF) with particular residual function mutations primarily led to the revenue growth of the drug. Additionally, an increasing number of patients starting Kalydeco therapy in the international market where Kalydeco is reimbursed also contributed to the revenue growth in 2017.

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Recent developments

In December 2017, Vertex Pharmaceuticals presented the results of the phase three trial of Kalydeco in children with CF between one and two years with one of ten mutations in the CFTR (cystic fibrosis transmembrane conductance regulator) gene. Vertex Pharmaceuticals’ phase three ARRIVAL trial met the primary endpoint of safety. In the trial, ivacaftor demonstrated tolerability. The safety profile of ivacaftor also remained consistent with results from the prior trials of ivacaftor in children between two to five years and six to 11 years.

Vertex Pharmaceuticals plans to submit a regulatory application for label expansion of ivacaftor to the US FDA (Food and Drug Administration) and European Medicines Agency (or EMA) in 1Q18. Presently, around 75,000 individuals in North America, Europe, and Australia are affected with cystic fibrosis, a rare genetic disease.

Novartis’s (NVS) Tobi Podhaler and Tobi (tobramycin) are widely used antibiotics for cystic fibrosis. Some other CFTR potentiators under investigation include Novartis’s (NVS) QBW251, Proteostasis Therapeutics’ (PTI) PTI-428, and ProQR Therapeutics’ (PRQR) QR-010.

Sales growth of Vertex Pharmaceuticals’ Kalydeco could boost the Vanguard S&P 500 ETF (VOO). Vertex Pharmaceuticals makes up about ~0.18% of VOO’s total portfolio holding.


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