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BOSTON, September 2, 2025 – The Institute for Clinical and Economic Review (ICER) today released a Final Evidence Report assessing the comparative effectiveness of apitegromab (Scholar Rock Holdings), as well as the disease-modifying therapies nusinersen (Spinraza®, Biogen), onasemnogene abeparvovec-xioi (Zolgensma®, Novartis), and risdiplam (Evrysdi®, Genentech) for spinal muscular atrophy (SMA). Apitegromab is an investigational new drug seeking FDA approval for improving motor function in patients with SMA.
ICER’s report on these therapies was the subject of the August 2025 public meeting of the Midwest CEPAC, one of ICER’s three independent evidence appraisal committees.
Downloads: Final Evidence Report | Report-at-a-Glance | Policy Recommendations
“SMA, in its most common forms, has been a devastating degenerative neurologic disease of infants and children,” said ICER’s Chief Medical Officer, David Rind, MD. “Disease modifying therapies and newborn screening have dramatically altered the course of disease and represent one of the great medical success stories in the past decade. However, as the votes of the independent appraisal committee recognized, we still have important uncertainties about how best to utilize these therapies to provide maximal benefits to those affected.”
ICER’s Virtual Public Meeting: Voting Results on Clinical Evidence
ICER assessed, and the independent appraisal committee voted on the evidence for effectiveness and value of therapies for spinal muscular atrophy:
- The majority of panelists (8-5) found that current evidence is not adequate to demonstrate a net health benefit of apitegromab in addition to standard of care (risdiplam or nusinersen) compared to the standard of care alone.
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The vast majority of panelists (12-1) found that current evidence is not adequate to demonstrate a net health benefit of using risdiplam after patients are treated with onasemnogene abeparvovec when compared to no additional treatment after receiving onasemnogene abeparvovec.
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The vast majority of panelists (12-1) found that current evidence is not adequate to demonstrate a net health benefit of using nusinersen after patients are treated with onasemnogene abeparvovec when compared to no additional treatment after receiving onasemnogene abeparvovec.
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The panelists unanimously (13-0) found that current evidence is not adequate to distinguish the net health benefit among nusinersen, onasemnogene abeparvovec, and risdiplam as first line therapy.
Panel members also weighed potential benefits and disadvantages beyond the direct health effects and weighed special ethical priorities. Voting highlighted the following as particularly important for payers and other policymakers to note:
- There is substantial unmet need despite currently available treatments.
ICER’s Virtual Public Meeting: Voting Results on Long-Term Value for Money
Apitegromab has not yet been approved by the FDA for SMA, and the manufacturer has not yet announced a US price for the therapy if approved. ICER did not perform a comparative value analysis for the disease-modifying therapies.
Consistent with ICER’s process, because there is no firm estimate yet of a potential launch price for the treatment, the panel did not take a vote on the treatment’s long-term value for money.
ICER has calculated a health benefit price benchmark (HBPB) to be between $4,600 and $30,200 per year.
Key Policy Recommendations
ICER’s independent assessment of value informs the critical decisions that stakeholders across the US health system need to make around pricing and coverage. Following the voting session, a policy roundtable of experts — including clinical experts, carers and patient advocates, and representatives from US payers — convened to discuss the pricing implications and recommendations to ensure fair access. Key recommendations stemming from the roundtable discussion include:
- The manufacturer should set prices that will foster affordability and good access for all patients by aligning prices with the patient-centered therapeutic value of their treatments. Given the small average improvement in motor function for patients treated with apitegromab and the uncertainty about serious adverse events, manufacturer pricing should reflect ICER’s value-based price range in moderating launch pricing.
- The use of SMN-directed therapy after gene therapy or in combination should only be done in the context of research studies.
- A randomized trial should be performed of first-line therapy in asymptomatic patients identified through newborn screening to better understand the comparative advantages and disadvantages of each of the three SMN-directed therapies.
ICER’s detailed set of policy recommendations is available in the Final Evidence Report and in the standalone Policy Recommendations document.
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