Chiesi has recently garnered a favorable recommendation from the National Institute for Health and Care Excellence (NICE) in relation to their cutting-edge Elfabrio treatment, scientifically known as pegunigalsidase alfa. This pioneering therapy has been meticulously crafted to cater to adult patients officially diagnosed with Fabry disease, offering them renewed hope and improved prospects.
Fabry disease, an exceptionally rare genetic disorder, afflicts an estimated 1,150 individuals in England. This condition is characterized by a deficiency of the vital enzyme alpha-galactosidase A, which plays a pivotal role in breaking down various fatty acids within the body. Pegunigalsidase alfa stands as a revolutionary enzyme replacement therapy, administered through intravenous infusion once every two weeks. This innovative treatment provides a modified version of the alpha-galactosidase enzyme, addressing the root cause of Fabry disease.
The resounding endorsement from NICE is the result of an exhaustive evaluation encompassing the safety and effectiveness of pegunigalsidase alfa. This evaluation was founded upon an extensive clinical trials program involving 142 individuals diagnosed with Fabry disease, comprising 94 males and 48 females. Out of this cohort, 112 participants were subjected to pegunigalsidase alfa treatment at a dosage of 1mg/kg every other week.
The research findings emanating from these trials resoundingly support the treatment’s merit. Notably, the therapy exhibited remarkable tolerability among patients, with the most frequently reported adverse reactions being infusion-related reactions, affecting 6.3% of patients, followed by asthenia (5.6%) and hypersensitivity (5.6%).
Dr. Kamran Iqbal, the head of medical affairs for global rare diseases at Chiesi UK&I, conveyed his profound optimism in response to NICE’s commendation. He emphasized the immense significance of this endorsement, stating, “We are thrilled that NICE has endorsed pegunigalsidase alfa, offering a new therapeutic option for individuals living with Fabry disease throughout England.” Dr. Iqbal further underscored the complex nature of Fabry disease symptoms, highlighting the importance of a diversified range of treatment options. Recognizing that one therapy may not suit everyone affected, he championed the need for tailored solutions.
Bob Stevens, the group chief executive of MPS Society, echoed this sentiment by expressing heartfelt gratitude for NICE’s pivotal decision to make pegunigalsidase alfa accessible to the Fabry community. This recommendation marks an expansion of the available treatment choices for individuals grappling with Fabry disease. Stevens concluded by underlining the critical importance of support and informed decision-making for those living with Fabry disease, ensuring they are empowered to pursue the lives they envision.
