An experimental triple combination treatment for cystic fibrosis has landed Vertex Pharmaceuticals at the top of EvaluatePharma’s “World Preview 2018” list.
An experimental triple combination treatment for cystic fibrosis has landed Vertex Pharmaceuticals at the top of EvaluatePharma’s “World Preview 2018” list.
In its annual list, EvaluatePharma said Vertex’s triple combination treatment VX-659, which includes tezacaftor and ivacaftor (Kalydeco), will become the most valuable project in the pharmaceutical industry pipeline” with a net present value of $13 billion. The Vertex treatment is a new entry for the EvaluatePharma list. As it soared to the top spot it replaces Biogen’s anticipated Alzheimer’s disease treatment aducanamab, which held the top spot last year. The Biogen drug, which is in Phase III, fell to third place behind AbbVie’s investigational JAK1-inhibitor upadacitinib, which is being developed as a monotherapy for rheumatoid arthritis patients.
Vertex initiated two Phase III trials for VX-659 earlier this year. In February the company initiated a trial in CF patients who have one F508del mutation and one minimal function mutation. Then in March Vertex began a trial to study the medication as a treatment for cystic fibrosis patients who have two copies of the F508del mutation, the most common genetic form of the disease.
If the triple-combination treatment scores well in Phase III and is approved, it is predicted to generate sales of about $3.4 billion by 2024, according to EvaluatePharma. In his analysis of the EvaluatePharma rankings, Keith Speights wrote in The Motley Fool that Vertex Chief Executive Officer Jeffrey Leiden noted at the Cowen healthcare conference earlier this year that VX-659, if approved, would be more profitable for the Boston-based company than its three approved CF treatments. The reason is that Vertex pays tiered royalties to Cystic Fibrosis Foundation Therapeutics Incorporated (CFFT) for the three approved drugs, Kalydeco, Orkambi, and Symdeko.
Smydeko is the latest Vertex CF treatment to be approved. In February the U.S. Food and Drug Administration gave the go-ahead to the company’s third CF drug. Vertex already markets CF drugs Kalydeco and Orkambi, which allows the company to already treat about 30,000 CF patients. There are an estimated 75,000 CF patients people in North America, Europe and Australia who have cystic fibrosis.
In addition to VX-659, earlier this year Vertex initiated Phase III studies of VX-445, tezacaftor and ivacaftor as an investigational triple combination regimen for people with cystic fibrosis (CF). Vertex is keeping its promise of advancing triple combination treatments for CF patients. Vertex believes the combination treatment will target the underlying cause of cystic fibrosis in patients who have one F508del mutation and one minimal function mutation (F508del/Min) – one of the most difficult types of CF to treat.
While Vertex is sitting pretty right now on the list, EvaluatePharma did make a prediction that Swiss-based Novartis will become the leading prescription drug company in 2024 with projected sales of $53.2 billion. That puts Novartis ahead of both Pfizer and Roche, which will compete for the number two spot, EvaluatePharma noted. The Novartis projection of $53 billion in sales in 2024 is certainly a lot of money, but the EvaluatePharma prediction pegs global prescription drug sales to reach $1.2 trillion in 2024, primarily due to “novel therapies addressing key unmet needs, as well as increasing access to medicines globally.”
