Management on sanitary inspection behind quality of foodstuff and medicines USA (FDA) approved the use of rosdiplom (Afridi®) for the treatment of spinal muscular atrophy (SMA) in adults and children aged 2 months. The drug demonstrated clinically significant improvements of motor function in two studies involving patients of different ages and with different severity of disease.
Through the use of a new drug, the babies had the ability to sit without support for at least 5 seconds — a key stage in the development of motor function, which is usually not observed in the natural course of the disease. Rosdiplom also improved survival without permanent ventilation in 12 months and 23 months in comparison with the natural course of SMA. The drug for oral administration is presented in liquid form and it can be in the home, if necessary, the drug can be administered through a nasogastric tube for enteral feeding.
In January Roche announced the launch of the global programme of pre-access therapy rosdiplom. In particular, the program was launched in Russia.
Rosdiplom (RG7916) —modifier splicing of the gene which produce a protein survival motor neuron-2 (SMN2). The drug is designed to increase and maintain the level of SMN protein in the CNS and in peripheral tissues.