EU approves expanded use of Vertex's cystic fibrosis drug

LONDON - The European Commission has approved a label expansion for Vertex Pharmaceuticals' (NASDAQ:VRTX) cystic fibrosis medication, KAFTRIO® (ivacaftor/tezacaftor/elexacaftor) in combination with ivacaftor, broadening its use to patients aged 2 years and older with at least one non-class I mutation in the CFTR gene. This regulatory milestone makes the treatment available to approximately 4,000 additional individuals living with cystic fibrosis (CF) in the European Union.

The decision follows Vertex's long-term commitment to addressing the needs of the CF community, including those with rare mutations. Executive Vice President Carmen Bozic expressed satisfaction with the European Commission's decision, which she believes will allow more CF patients to benefit from the transformative effects of KAFTRIO®.

In countries such as Austria, Denmark, Ireland, Norway, and Sweden, where KAFTRIO® reimbursement agreements exist, and in Germany, where healthcare system provisions apply, eligible patients are expected to gain access to the therapy shortly. Vertex plans to continue its collaborative efforts with reimbursement authorities throughout the EU to facilitate access for all eligible patients.

Cystic fibrosis is a life-shortening genetic disease that affects over 94,000 individuals in North America, Europe, and Australia. It is a multi-organ condition that leads to progressive lung damage and other complications, with the median age of death in the 30s. However, treatments like KAFTRIO® are improving the projected survival rates.

KAFTRIO® works by increasing the quantity and function of the CFTR protein at the cell surface, which is crucial for the transport of salt and water across cell membranes. This helps to hydrate and clear mucus from the airways, addressing the fundamental cause of CF.

Vertex, a global biotechnology company with headquarters in Boston and London, is known for its scientific innovation in creating transformative medicines for serious diseases. The company's portfolio includes approved therapies for cystic fibrosis, sickle cell disease, transfusion-dependent beta thalassemia, and acute pain, among others.

The information in this article is based on a press release statement from Vertex Pharmaceuticals.

In other recent news, Vertex Pharmaceuticals has seen several developments impacting its operations and future outlook. RBC Capital Markets adjusted its price target for Vertex to $420, maintaining a Sector Perform rating, based on promising early performance of Alyftrek, which is expected to cannibalize sales from Trikafta. Meanwhile, Canaccord Genuity maintained its Hold rating with a $424 target after Vertex announced that its VX-264 program for Type 1 Diabetes would not proceed due to underwhelming clinical trial results. Despite this setback, Vertex's zimislecel program for T1D is progressing positively, with plans for global regulatory submissions by 2026.

Additionally, Vertex's non-opioid pain medication, Journavx, has been included in UnitedHealth Group's coverage but placed on a higher-cost tier, potentially affecting its commercial success. The drug was approved in the U.S. in January, and its classification by pharmacy benefits managers is crucial for its market performance. In related analyst activity, DA Davidson reiterated a Buy rating on Veritex Holdings, with a $34 target, following investor meetings discussing the company's strategic growth plans. These recent updates provide insights into Vertex's ongoing strategies and challenges in the pharmaceutical landscape.

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