Tofersen

Tofersen, sold under the brand name Qalsody, is a medication used for the treatment of amyotrophic lateral sclerosis (ALS).^[3] Tofersen is an antisense oligonucleotide that targets the production of superoxide dismutase 1, an enzyme whose mutant form is commonly associated with amyotrophic lateral sclerosis. It is administered as an intrathecal injection.^[3]

The most common side effects include fatigue, arthralgia (joint pain), increased cerebrospinal (brain and spinal cord) fluid white blood cells, and myalgia (muscle pain).^[3]

Tofersen was approved for medical use in the United States in April 2023,^[3][6] and in the European Union in May 2024.^[4] The US Food and Drug Administration (FDA) considers it to be a first-in-class medication.^[7]

Tofersen is indicated to treat people with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS).^[3]

Tofersen was developed by Ionis Pharmaceuticals and was licensed to, and co-developed by, Biogen.^[8][9]

The effectiveness of tofersen was evaluated in a 28-week, randomized, double-blind, placebo-controlled clinical study in 147 participants with weakness attributable to amyotrophic lateral sclerosis and a superoxide dismutase 1 (SOD-1) mutation confirmed by a central laboratory.^[3] The study randomly assigned 108 participants in a 2:1 ratio to receive treatment with either tofersen 100 mg (n = 72) or placebo (n = 36) for 24 weeks (three loading doses followed by five maintenance doses).^[3] The participants were approximately 43% female; 57% male; 64% White; and 8% Asian.^[3] The average age was 49.8 years (range from 23 to 78 years).^[3]

The stage III clinical trial was conducted by the Neuroscience Institute and Sheffield Institute for Translational Neuroscience (SITraN), both at the University of Sheffield.^[10]

منحت إدارة الغذاء والدواء الأمريكية (FDA) طلب الموافقة على دواء توفرسن مراجعة ذات أولوية ، ودواء يتيم ، وتصنيف المسار السريع . ^{[ 3 ] [ 7 ]}

في فبراير 2024، أصدرت لجنة المنتجات الطبية للاستخدام البشري التابعة لوكالة الأدوية الأوروبية رأيًا إيجابيًا، موصيةً بمنح ترخيص تسويق، في ظروف استثنائية، للمنتج الطبي كالسودي، المُخصص لعلاج نوع من التصلب الجانبي الضموري الناجم عن خلل في بروتين سوبر أكسيد ديسموتاز 1 (SOD1). ^{[ 4 ] [ 15 ]} مقدم طلب ترخيص هذا المنتج الطبي هو شركة بيوجين هولندا بي في. ^{[ 4 ]} تمت الموافقة على استخدام توفرسن طبيًا في الاتحاد الأوروبي في مايو 2024. ^{[ 5 ]}