MHRA approves Vertex’s Alyftrek triple therapy for cystic fibrosis

Vertex Pharmaceuticals has announced that its latest triple combination treatment for cystic fibrosis (CF), known as Alyftrek, has received approval from the UK's Medicines and Healthcare products Regulatory Agency (MHRA). The therapy is now authorized for use in individuals aged six and older who carry at least one F508del mutation or another mutation in the CF transmembrane regulator (CFTR) gene that shows a positive response to this treatment.

Cystic fibrosis is a rare, genetic disorder that affects over 11,000 people in the UK. It is caused by a defective CFTR gene which is responsible for regulating the flow of water and chloride in and out of organs such as the lungs. The malfunctioning of this gene leads to the accumulation of thick, sticky mucus in the lungs, resulting in chronic infections, progressive lung damage, and often early death.

Alyftrek, a once-daily oral CFTR modulator, has been designed to correct the defective protein produced by the CFTR gene. The MHRA's decision to approve this therapy was based on favorable outcomes from two late-stage clinical trials involving 480 cystic fibrosis patients aged 12 and above. The trials demonstrated that Alyftrek was as effective in enhancing lung function as Vertex's existing triple combination therapy, Kaftrio, and showed superior efficacy in reducing sweat chloride levels.

The decision was further supported by additional data from an open-label phase 3 study. Carmen Bozic, Vertex's executive vice president of global medicines development and medical affairs, and chief medical officer, highlighted the significance of this new development. She emphasized Vertex's two-decade-long commitment to discovering treatments that address the root cause of cystic fibrosis, ultimately aiming to improve the quality and longevity of patients' lives. Bozic regards the approval of Alyftrek as a pivotal achievement in the ongoing effort to provide better treatment options for CF patients in the UK.

Vertex is actively collaborating with the National Health Service (NHS) and the National Institute for Health and Care Excellence (NICE) to expedite the availability of Alyftrek to eligible patients. This collaboration aims to ensure that individuals who can benefit from the treatment can access it as promptly as possible.

David Ramsden, the chief executive of the Cystic Fibrosis Trust, expressed his approval of the MHRA's decision, considering it a crucial step towards making optimal treatments accessible to as many CF patients as possible. Ramsden urged NICE to swiftly complete its evaluation of Alyftrek, so it can be prescribed through the NHS, echoing the broader hope for timely and widespread access to this promising new therapy.

The MHRA's approval of Alyftrek marks an important advancement in the treatment of cystic fibrosis, offering hope to many patients and their families. The collaboration between Vertex, health authorities, and advocacy groups underscores a shared commitment to enhancing the quality of life for people affected by this challenging disease.