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Evrysdi vs Spinraza: How do they compare?

Medically reviewed by Philip Thornton, DipPharm. Last updated on June 3, 2022.

Official answer

by Drugs.com

Evrysdi (risdiplam) and Spinraza (nusinersen) are both drugs that are used to treat spinal muscular atrophy (SMA) in adults and children. SMA is a type of motor neuron disease that destroys motor neurons - the muscle-controlling nerve cells.

Evrysdi and Spinraza work by targeting SMN2 (survival of motor neuron 2) and increasing levels of the SMN protein throughout the body, however, they go about it in two slightly different ways. Increasing SMN protein helps to improve muscle and nerve function in people with SMA.

One of the key differences between these drugs is that Evrysdi is taken orally, unlike Spinraza which is given via intrathecal injection. When Evrysdi was approved for use in August 2020 it not only provided an alternative treatment option for people with SMA who were already receiving treatment, but it also provided the first oral and at-home treatment option for people with SMA, which is particularly important for those people who are unable to receive intrathecal injections.

The table below compares Evrysdi and Spinraza across a range of areas.

Evrysdi (risdiplam) Spinraza (nusinersen)
Company Genentech Biogen
Approval date First approved August 2020 First approved December 2016
Dosage Form Powder for oral solution Injection in a single-dose vial
Administration Self administered via oral syringe, gastrostomy or nasogastric tube. The medication is made up by a pharmacist and can then be given at home following the instructions of a healthcare provider Intrathecal injection (injection into the subarachnoid space of the spinal cord/the cerebrospinal fluid)
Dosing schedule Given daily Four loading doses required:
  • First three given at 14 day intervals
  • Fourth given 30 days after the third.
A maintenance dose is then given once every four months
Indication / usage Used to treat spinal muscular atrophy (SMA) in pediatric and adult patients Used to treat spinal muscular atrophy (SMA) in pediatric and adult patients
Drug type Small molecule drug An antisense oligonucleotide
Mechanism of action Survival of motor neuron 2 (SMN2) splicing modifier. It works by increasing the ability of the SMN2 gene to make more functional SMN protein, which is lacking in people with SMA Survival of motor neuron 2 (SMN2)-directed antisense oligonucleotide. It works by rebinding at exon 7 so that the SMN protein produced by the SMN2 gene becomes functional
Side effects / adverse effects The most common adverse reactions in later-onset SMA occurring in 10% of patients include:
  • Fever
  • Diarrhea
  • Rash

Similar reactions were seen in patients with infantile-onset SMA, plus the following also occurred in at least 10% of patients:

  • Upper respiratory tract infections
  • Lower respiratory tract infections
  • Constipation
  • Vomiting
  • Cough
 The most common adverse reactions occurring in at least 20% of patients and at a frequency of at least 5% more than controls who didn’t receive Spinraza include:
  • Lower respiratory infection and constipation in patients with infantile-onset SMA
  • Pyrexia, headache, vomiting and back pain in patients with later-onset SMA
Efficacy
  • Clinical trial results show that treatment with Evrysdi resulted in 7 of 17 infants being able to sit independently for ≥ 5 seconds as measured by Item 22 of the Bayley Scales of Infant and Toddler Development - Third Edition (BSID-III) gross motor scale. Without treatment, no such children with infantile-onset SMA would be expected to be able to sit independently. Without treatment, no more than 25% of such children would also be expected to survive without permanent ventilation beyond 14 months of age, however, 17 of 21 patients (median age 32 months) who were treated with Evrysdi were alive without permanent ventilation after a minimum of 23 months' treatment.
  • Interim clinical trial results from a trial conducted in pre-symptomatic infants who received their first dose of Evrysdi at up to 6 weeks of age showed that of the 6 patients who have 2 or 3 copies of SMN2, 100% achieved sitting, 67% were able to stand and 50% could walk independently (measured by HINE-2 at 12 months). All were alive without permanent ventilation at 12 months.
  • In patients with later-onset SMA, 12 months of treatment with Evrysdi also resulted in a clinically meaningful and statistically significant change in Motor Function Measure 32 (MFM32) scores compared with patients treated with placebo.
  • Clinical trial results show that treatment with Spinraza was associated with a 47% reduction in the risk of death or permanent ventilation compared with infants who received the control. In the control group the median time to death or permanent ventilation was 22.6 weeks, but this event was not reached in the Spinraza-treated group. Improvements in motor function were also more commonly observed in infants treated with Spinraza.
  • In patients with later-onset SMA, treatment with Spinraza resulted in a statistically significant improvement in Hammersmith Functional Motor Scale - Expanded (HFMSE) scores at 15 months compared with patients treated with the sham-control.
  • Treatment with Spinraza was also shown to be effective in the small number of infants with presymptomatic SMA who were treated in a clinical trial.
Warnings and precautions Requires baseline tests before each administration because of thrombocytopenia and coagulation abnormalities and renal toxicity
Special patient populations May cause fetal harm
Avoid in hepatic impairment		 May cause fetal harm
References

Read next

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

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

Evrysdi does not cure spinal muscular atrophy (SMA), it just replaces a protein that is low or missing in 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. Continue reading

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