Does Evrysdi cure spinal muscular atrophy (SMA)?
Evrysdi does not cure spinal muscular atrophy (SMA), it just replaces a protein that is low or missing in newborns, children, or adults with the condition, improving muscle strength and allowing some relief from some of the symptoms of SMA. Evrysdi must be taken for a person’s lifetime; if Evrysdi is stopped the newborn, child, or adult’s SMA symptoms will progressively worsen at a faster rate.
How does Evrysdi work?
Evrysdi (risdiplam) works by targeting the SMN2 gene (survival motor neuron gene 2), causing it to make more functional SMN protein. This increases SMN protein levels throughout the central nervous system and body, helping to improve motor nerve and muscle function in children and adults with SMA.
Evrysdi was approved by the FDA on August 7, 2020 and is available as an oral tablet that can be swallowed whole or dispersed in unchlorinated water, or an oral liquid that is given by mouth or through a gastrostomy tube (g-tube). Both formulations are taken once a day after a meal. It may be used to treat all types of spinal muscular atrophy (SMA) in adults, children, and newborns. A label extension for Evrysdi in May, 2022 to include pre-symptomatic infants aged under 2 months (or newborns) allows healthcare providers to intervene as early as possible in treating babies with SMA.
References
- Drug Trials Snapshots: EVRYSDI US Food and Drug Administration https://www.fda.gov/drugs/drug-approvals-and-databases/drug-trials-snapshots-evrysdi
- Evrysdi Cure SMA Genentech https://www.curesma.org/evrysdi/
- Evrysdi Prescribing Information. https://www.gene.com/download/pdf/evrysdi_prescribing.pdf
- Evrysdi Package Insert. https://www.gene.com/download/pdf/evrysdi_patientinfo.pdf
Read next
Evrysdi vs Spinraza: How do they compare?
Evrysdi (risdiplam) and Spinraza (nusinersen) work in different ways to increase the level of functional SMN (survival of motor neuron) protein in people with spinal muscular atrophy (SMA). A key difference between the two drugs is that Evrysdi is taken orally, whereas Spinraza needs to be given via intrathecal injection. Continue reading
How does Evrysdi work for SMA?
Evrysdi (risdiplam) works by targeting the SMN2 gene (survival motor neuron gene 2), causing it to make more functional SMN protein. This increases SMN protein levels throughout the central nervous system and body, helping to improve motor nerve and muscle function in children and adults with SMA. Continue reading
How effective is Evrysdi?
Several major trials have reported that Evrysdi significantly improves survival motor neuron (SMN) protein levels in newborns, children, and adults with SMA, allowing an increase in muscle strength and the achievement of milestones, such as being able to sit independently for 5 or more seconds at a time. Continue reading
See also:
Aimovig
Learn about Aimovig (erenumab-aooe) a once-monthly, injectable medication that can be ...
Nurtec ODT
Nurtec ODT (rimegepant) is used to treat acute migraines and prevent episodic migraines, by ...
Botox
Botox is used for cosmetic purposes and to treat overactive bladder symptoms, urinary incontinence ...
Vyepti
Vyepti (eptinezumab-jjmr) is used for the preventive treatment of migraine. Includes Vyepti ...
Depakote
Depakote is used to treat various types of seizure disorders. Learn about side effects ...
Topamax
Topamax (topiramate) is used to prevent seizures in adults and children and to prevent migraine ...
Emgality
Emgality (galcanezumab-gnlm) is a CGRP antagonist for the preventive treatment of migraine and for ...
Inderal
Inderal (propranolol) is used to treat angina, high blood pressure and heart rhythm disorders ...
Ajovy
Ajovy (fremanezumab-vfrm) is a preventive migraine treatment. Includes Ajovy side effects ...
Valproate Sodium
Valproate Sodium is used for epilepsy, seizure prevention, seizures
Related medical questions
- How is Evrysdi administered?
- Why is Zolgensma so expensive?
- Zolgensma vs Spinraza: What are the key differences?
- Is Zolgensma a cure for SMA?
- How is Zolgensma administered?
- How is Spinraza administered?
Drug information
- Evrysdi Information for Consumers
- Evrysdi prescribing info & package insert (for Health Professionals)
- Side Effects of Evrysdi (detailed)
Related support groups
- Evrysdi (5 questions, 3 members)
- Spinal Muscular Atrophy (10 questions, 3 members)