Nusinersen - Biogen/Ionis Pharmaceuticals
Alternative Names: BIIB 058; IONIS-SMNRx; ISIS-396443; ISIS-SMNRx; SpinrazaLatest Information Update: 21 Jun 2024
At a glance
- Originator Genzyme Corporation; Isis Pharmaceuticals
- Developer Biogen; Ionis Pharmaceuticals
- Class Antisense oligonucleotides
- Mechanism of Action RNA interference; Survival of motor neuron 2 protein expression stimulants
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Orphan Drug Status
Orphan designation is assigned by a regulatory body to encourage companies to develop drugs for rare diseases.
- New Molecular Entity Yes
Highest Development Phases
- Marketed Spinal muscular atrophy
Most Recent Events
- 30 May 2024 Biogen completes the phase II/III DEVOTE trial in Spinal muscular atrophy (In children, In adolescents, In adults, In the elderly) in USA, Australia, Brazil, Canada, Chile, China, Colombia, Estonia, France, Greece, Germany, Hungary, Ireland, Israel, Italy, Japan, South Korea, Latvia, Lebanon, Mexico, Netherlands, Poland, Russia, Saudi Arabia, Spain, Turkey, Taiwan and the UK (NCT04089566) (EudraCT2019-002663-10)
- 27 Sep 2022 Launched for Spinal muscular atrophy (In adolescents, In children, In infants, In neonates, In adults) in Canada (Intrathecal)
- 30 Aug 2022 The Canadian Agency for Drugs and Technologies in Health's (CADTH) announces their decision to not give reimbursement to adult patients with Spinal muscular atrophy (SMA) in Canada