Novo Nordisk, Eli Lilly fall after Trump comments on weight loss drug pricing
LONDON - Italy’s Medicines Agency (AIFA) has reached a reimbursement agreement with Vertex Pharmaceuticals (NASDAQ:VRTX), a prominent biotechnology company with a market capitalization of $99.26 billion, for CASGEVY, a CRISPR/Cas9 gene-edited therapy for eligible patients with transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD). According to InvestingPro data, Vertex maintains excellent financial health with strong profitability metrics, positioning it well for this expansion.
The agreement is particularly significant as Italy has the largest TDT population in Europe, with approximately 5,000 people aged 12 and older living with the condition and around 2,300 with SCD. This market expansion aligns with Vertex’s robust growth trajectory, evidenced by its 10.46% revenue growth over the last twelve months. InvestingPro analysis reveals 11 analysts have revised their earnings upwards for the upcoming period, suggesting strong market confidence in the company’s expansion strategy.
CASGEVY is the first and only gene editing therapy approved for these conditions in Europe. The treatment works by editing a patient’s own hematopoietic stem cells to produce high levels of fetal hemoglobin, which helps reduce or eliminate vaso-occlusive crises in SCD patients and transfusion requirements for TDT patients.
"Today is a turning point for eligible people in Italy living with transfusion-dependent beta thalassemia and sickle cell disease, two life-shortening diseases with limited treatment options," said Ludovic Fenaux, Senior Vice President, Vertex International, in a press release statement.
Both TDT and SCD are serious genetic diseases that significantly impact quality of life and life expectancy. TDT requires frequent blood transfusions and iron chelation therapy throughout a person’s life, with a mean age of death in Europe between 50-55 years. SCD causes severe pain, organ damage, and has a mean age of death in Europe of around 40 years.
Italy joins several other countries that have signed reimbursement agreements for CASGEVY, including Austria, Bahrain, Denmark, England, Saudi Arabia, and the United Arab Emirates.
The therapy is approved for patients 12 years and older with either severe SCD with recurrent vaso-occlusive crises or TDT, for whom hematopoietic stem cell transplantation is appropriate and a matched related donor is not available.
In other recent news, Vertex Pharmaceuticals reported its second-quarter 2025 revenue at $2.96 billion, surpassing both Truist Securities’ estimate of $2.93 billion and the consensus of $2.91 billion. Despite the revenue beat, Truist Securities lowered its price target for Vertex to $490, maintaining a Buy rating due to the company’s reliance on its cystic fibrosis franchise. Canaccord Genuity also adjusted its price target to $411 following mixed pipeline updates, while maintaining a Hold rating. Meanwhile, Bernstein SocGen Group raised its price target to $471, even as unfavorable updates to Vertex’s pain pipeline led to a notable share price decline. Goldman Sachs reiterated its Buy rating with a $624 price target, emphasizing Vertex’s diversification beyond cystic fibrosis. BofA Securities, however, lowered its price target to $34, citing a weaker fiscal year 2025 outlook and potential underlying issues beyond macroeconomic conditions. These developments reflect a range of analyst perspectives on Vertex’s financial health and strategic direction.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.