Novartis's new sclerosis drug is approved by breakthrough therapy
Novartis's new sclerosis drug is approved by breakthrough therapy December 22, 2017 Source: WuXi PharmaTech Novartis has announced that the US FDA has awarded the company's Gilenya (fingolimod) breakthrough therapy for the treatment of children with recurrent multiple sclerosis (MS) (age 10 and older). Gilenya has been approved in the United States for the treatment of recurrent MS adult patients, but has not been approved for use in children. MS is an autoimmune disease that occurs in the central nervous system. It causes inflammation and tissue damage that destroys the normal function of the brain, optic nerve, and spinal cord. The main age of onset of MS is 20 to 40 years old, which is the cause of disability of young and middle-aged people after trauma. MS affects approximately 400,000 people in the United States. There are three types of MS in adults: relapsing-remitting (RRMS), secondary progressive (SPMS), and primary progressive (PPMS). In children, up to 98% of MS cases are RRMS. Advances in MS have led to increased loss of physical and cognitive functions such as memory. This has a great negative impact on the lives of MS patients. It is estimated that 3-5% of MS patients are children, but there is no palliative therapy specifically for this group. Therefore, there is still a large medical need in this group. Gilenya is the first oral drug approved for the treatment of recurrent MS (once a day). As a first-line disease remission therapy (DMT), it helps to slow down the physical problems caused by RRMS and reduce the frequency of MS onset (relapse), and because it is an oral medication, it also protects patients from injection. The mechanism by which Gilenya plays a role in MS patients remains to be fully elucidated, but may involve reducing the migration of lymphocytes to the central nervous system. There is ample evidence that T cells play a central role in mediating and regulating MS pathology. It is currently believed that Gilenya may, by binding to T cells, reside in lymph nodes, thereby reducing immune cells entering the central nervous system and reducing the number of MS episodes and the effects on bodily functions. This time, Gilenya's breakthrough therapy was based on data from the Phase 3 clinical study PARADIGMS, which evaluated the use of Gilenya or interferon beta-1a in children with recurrent MS (10 years or older). Interferon beta-1a) safety and efficacy. PARADIGMS is the first randomized controlled clinical trial designed specifically for children with recurrent MS. It is a double-blind, randomized, multicenter study for up to 2 years followed by a 5-year open label Extended period experiment. The study included 215 children with MS between the ages of 10 and 17 and the Extended Disability Status Scale (EDSS) score of 0-5.5. Patients were randomized to receive Gilenya (0.5 mg or 0.25 mg, depending on the patient's weight) once a day or intramuscularly with interferon beta-1a once a week. The primary endpoint of the study was the frequency of recurrence within two years (annual recurrence rate). The data showed that Gilenya's treatment reduced the recurrence rate (annual recurrence rate) of the patient group by 82% (p < 0.001) within 2 years compared with intramuscular injection of interferon beta-1a. The safety demonstrated by Gilenya in this study is consistent with that previously observed in clinical trials in adult patients. ▲Principal Investigator of PARADIGMS Research, Dr. Tanuja Chitnis, Director of the Pediatric Multiple Sclerosis Center, Massachusetts General Hospital, Boston, USA (Source: Partners Pediatric Multiple Sclerosis Center) “Although children with MS have experienced approximately two to three times the number of relapses in adult patients, there is currently no approved disease remission therapy for children,†said PARIDIMS Research Principal Investigator, Boston Massachusetts General Hospital Partner Pediatric Multiple Sclerosis Dr. Tanuja Chitnis, director of the Partners Pediatric Multiple Sclerosis Center, said, “There are important differences between MS children and adults, and there is a need to provide more treatment options for children.†▲ Dr. Fabrice Chouraqui, President of Novartis Pharmaceuticals Corporation (Source: Novartis official website) “We are proud of the milestones in this approval process, which is part of our commitment to advance treatment options for young MS patients,†said Dr. Fabrice Chouraqui, President of Novartis Pharmaceuticals Corporation: “Nova is looking forward to working with the FDA as soon as possible. This treatment for adult patients with recurrent MS is brought to this young patient population." We congratulate Novartis and hope that more new drugs will help the MS patient population. Reference materials: [1] Novartis multiple sclerosis therapy fingolimod granted FDA Breakthrough Therapy designation for pediatric MS [2] Novartis official website Roxadustat Intermediate Roxadustat Intermediate,Fg-4592 Intermediate,Cas 1455091-10-7,Cas 1455091-10-7 Roxadustat Intermediate Shijiazhuang Dingmin pharmaceutical Sciences Co.,Ltd , https://www.dingminpharma.com
