Cystic fibrosis
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Vertex Pharmaceuticals’ three cystic fibrosis drugs—Kaftrio/Trikafta, Symkevi and Orkambi—will now be available through England’s National Health Service, following the signing of a long-term reimbursement agreement.
With a total revenue of nearly $2.7 billion, Vertex exceeded analyst estimates in the first quarter, bolstered by strong uptake of its cystic fibrosis products.
Sionna Therapeutics on Wednesday announced it raised another round of funding as the company looks to challenge Vertex Pharmaceuticals’ dominant cystic fibrosis business.
The company’s three-drug combination met all primary and key secondary endpoints in two randomized controlled trials in cystic fibrosis patients, paving the way for an application to regulators.
The biopharma company reported sales of $2.24 billion in the second quarter for the cystic fibrosis treatment, beating analysts’ estimate, after it won FDA approval in children ages two to five in April.
Drugs that act on the CFTR protein only work in patients who produce the protein in the first place. That leaves 6% of patients hanging.
Vertex is kicking off a Phase I trial assessing VX-522, an mRNA therapy designed to treat the underlying cause of cystic fibrosis lung disease, while SpliSense is targeting a specific mutation.
Sionna hit the ground with a pipeline of first-in-class small molecules designed to fully restore the function of the cystic fibrosis transmembrane conductance regulator (CFTR) protein in CF patients.
Vertex has made a point over the past decade to make research and development a priority, investing over 70% of its expenses back into it.
Suliman anticipates filing an Investigational New Drug application with the FDA for the PCD asset this year, and next year filing one for the cystic fibrosis asset.
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