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Vertex Announces CASGEVY® Reimbursement Agreement for the Treatment of Transfusion-Dependent Beta Thalassemia and Sickle Cell Disease in Italy
- CASGEVY is the first, and only, gene editing therapy approved for the treatment of transfusio...
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[{"type":"text","content":"Vertex Announces CASGEVY® Reimbursement Agreement for the Treatment of Transfusion-Dependent Beta Thalassemia and Sickle Cell Disease in Italy\n\n\n .bwalignc { text-align: center; list-style-position: inside }\n \n\n\n\n\n - CASGEVY is the first, and only, gene editing therapy approved for the treatment of\n \n transfusion-dependent beta thalassemia (\n \n TDT) and\n \n sickle cell disease\n \n (SCD) in Europe -\n \n\n\n\n - Italy has the largest population of people living with TDT in Europe\n \n\n -\n \n\n\n\n\n\n Vertex Pharmaceuticals\n \n (Nasdaq: VRTX) announced today a reimbursement agreement with the Italian Medicines Agency (AIFA) for eligible transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD) patients to access the CRISPR/Cas9 gene-edited therapy, CASGEVY\n \n ®\n \n (exagamglogene autotemcel).\n \n\n “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. “Italy has the largest TDT population in Europe, which underscores the importance of this agreement. We appreciate the collaboration with AIFA to recognize the value a one-time transformative treatment provides to patients, families and the health care system.”\n \n\n In Italy, there are approximately 5,000 people 12 years and older living with TDT and around 2,300 with SCD. Italy joins a number of countries that have signed reimbursement agreements for CASGEVY including Austria, Bahrain, Denmark, England, the Kingdom of Saudi Arabia and the United Arab Emirates.\n \n\n\n About Transfusion-Dependent Beta Thalassemia (TDT)\n \n\n\n TDT is a serious, life-threatening genetic disease. TDT patients report health-related quality of life scores below the general population and significant health care resource utilization. TDT requires frequent blood transfusions and iron chelation therapy throughout a person’s life. Due to anemia, patients living with TDT may experience fatigue and shortness of breath, and infants may develop failure to thrive, jaundice and feeding problems. Complications of TDT can also include an enlarged spleen, liver and/or heart, misshapen bones and delayed puberty. TDT requir...