Vertex Pharmaceuticals Misses Sales Estimates; UK May Discontinue Use of Its Costly Cystic Fibrosis Treatments

Q3 sales decline due to weak demand for older treatments; UK's NICE deems cost of Vertex's treatments too high, could deny future access to new CF patients.

• Vertex Pharmaceuticals' Q3 sales fell short of estimates due to weak demand for its older CF treatments; the company now expects annual sales of about $9.85 billion from its CF treatments, compared to estimates of $9.86 billion.
• Sales of Vertex's top-selling CF drug, Trikafta, were reported at $2.27 billion in Q3, though total sales of $2.48 billion missed estimates by $20 million.
• The National Institute for Health and Care Excellence (NICE) has deemed Vertex's groundbreaking CF treatment, Trikafta, and two other drugs as not cost-effective despite the clinical benefits, posing a potential denial of future access to new CF patients in the UK.
• Existing Vertex CF treatments - the first to address the root causes of the disease rather than just the symptoms - are considered game-changing, life-extending advances, but regulatory proposals could restrict access to them for new patients on cost grounds.
• If enacted, NICE's draft guidance would not affect current patients of ages 1 and over, but new CF patients would not be guaranteed treatment under National Health Service (NHS) care, a proposal that has generated fear and uncertainty among the CF community.

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