A Look at Vertex’s Cystic Fibrosis Patient Opportunity



CF opportunity

There are ~27,000 eligible patients for Vertex Pharmaceuticals’ (VRTX) Kalydeco and Orkambi. However, Vertex is currently treating only one-third of patients.

As per Vertex’s estimates, “There are 17,000 additional patients. . .who could benefit from one of these two medicines.” This would move the total eligible population of patients to ~44,000.

The incremental 17,000 patients can be divided into two groups that can be served by either drug. Orkambi can treat 12,000 patients below the age of 12 with the F508del mutation. Kalydeco can help 5,000 patients with residual function mutation.

With such a huge volume expansion opportunity, the revenue from these two drugs can record even higher levels. This is a major reason that the company is pursuing label expansion studies for both drugs.

Vertex’s advancing CF pipeline of next-generation corrector combinations such as VX-152 and VX-440 could enable it to expand its population base to ~60,000. These two pipeline molecules are currently in Phase 1 studies.

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Other companies exploring the CF space 

Other companies that are into the development of CF drugs include Pfizer (PFE), Shire (SHPG) and Sanofi’s (SNY) Genzyme. Pfizer is developing a gene therapy for CF by using the adeno-associated virus and the human bocavirus. The University of Iowa Research Foundation is supporting the research.

Shire has also partnered with the Cystic Fibrosis Foundation for developing a technology to maintain or restore lung function.

Proteostasis Therapeutics (PTI) recently received a fast-track designation from the FDA for “PTI-428, an investigational oral treatment for CF.”

It’s often risky to directly invest in a biotechnology company, as any news release for the success or failure of its drug can result in volatility in its stock price. To remain comparatively safe, investors can choose ETFs such as the First Trust NYSE Arca Biotechnology Index ETF (FBT), which holds 3.3% in Vertex’s stock.


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