Vertex Pharmaceuticals
NEWS
Along with its gene editing therapy Casgevy, Vertex is offering fertility preservation support for its patients—a program that the HHS claims violates anti-kickback statutes.
Among the FDA’s pending decisions for this quarter are Vertex’s non-opioid pain drug and Sanofi’s RNA interference therapy for hemophilia A and B.
The new combination, dubbed Alyftrek, is designed to improve on the Trikafta, a product that generated sales of $8.9 billion in 2023. It’s welcome good news for Vertex following last week’s subpar clinical results for its non-opioid analgesic.
Vertex Pharmaceuticals’ investigational non-opioid analgesic suzetrigine failed to outperform placebo. Investors voiced their concerns as the company’s share price fell 13% in premarket trading.
By far, the largest acquisition of 2024 was Novo Holdings’ yet-to-be-closed buyout of manufacturer Catalent at $16.5 billion. Outside of that, the leading pharmaceutical companies kept to less than $5 billion per deal.
GSK, Gilead and Arcellx, Vertex and more present new data at the American Society of Hematology annual meeting just as sickle cell therapies Casgevy and Lyfgenia have a new outcomes-based payment model; Eli Lilly and Novo Nordisk pump new funds into manufacturing; and AbbVie makes a Cerevel comeback while uniQure clears a path toward accelerated approval in Huntington’s disease.
Vertex unveiled long-term durability data for Casgevy, while Beam presented Phase I/II findings for its investigational base editor BEAM-101, building up to a BLA by late 2026.
The payment scheme will tie gene therapy payments to improvements in health outcomes—and could potentially boost the uptake of these sickle cell disease treatments.
Eli Lilly topped the list of the 20 biggest pharmas by market cap with a more than 39% improvement year-to-date in its share price. Other companies have not been so lucky.
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