A Promising Uptake for Vertex’s Orkambi



Rising Orkambi volume

It’s been nine months since Vertex Pharmaceuticals (VRTX) launched Orkambi. Out of the 8,500 eligible patients, around 5,500 are using the drug. This translates to a ~65% market share in volume terms.

Although the number of patients who have initiated the treatment is growing, there have been significant discontinuations on account of adverse effects. Around 15% of patients have discontinued the treatment within the first three months of initiation.

Vertex estimates that though this percentage is significant, in the long term, ~70%–80% of patients will initiate and stay on the treatment.

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Orkambi’s recent approvals and applications

Vertex has submitted a supplemental new drug application (or sNDA) to the FDA for the approval of the drug in children aged 6–11 years with two copies of the F508del mutation. With this development, Vertex plans to bring the drug to younger patients.

If Orkambi gets a priority review, the decision on the application will be available in the second half of the year. For more information on the drug, you can refer to Orkambi: Recent Addition to the Vertex Portfolio

Vertex is conducting a Phase 3 efficacy study of Orkambi in children in Europe in the age group of 6–11 years with two copies of the F508del mutation. If approved, the drug’s population base in Europe would expand by 3,400.

During 1Q16, Orkambi was approved in Australia and Canada to treat CF in patients aged 12 and above. Orkambi’s overall eligible population in these two countries accounts for 2,500.

To gain exposure to Vertex Pharmaceuticals and control excessive company-specific risks, investors can choose to invest in the VanEck Vectors Biotech ETF (BBH). The ETF holds 70.5% of its assets in the biotechnology sector. It holds 13.4% and 4.8% in Gilead Sciences (GILD) and Regeneron (REGN), respectively. Baxalta (BXLT) makes up 4.8% of the fund’s assets, whereas Vertex accounts for 4.0% of its total holdings.


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