ICER flags concerns over Scholar Rock’s apitegromab and other SMA drugs

The Institute for Clinical and Economic Review (ICER) has cast doubt on the value of a new spinal muscular atrophy treatment from US biotech Scholar Rock, while reaffirming concerns about limited comparative data across the class of SMA therapies.

In a new Evidence Report published July 17, the  ICER said apitegromab—an investigational myostatin inhibitor currently under FDA review—offers “promising but inconclusive” clinical benefit for patients with Type 2 or Type 3 SMA who are already receiving Spinraza (nusinersen) or Evrysdi (risdiplam).