ISEMBYLD (apitegromab-mstn) was approved by the FDA on September 11, 2026 for spinal muscular atrophy (SMA), adults and children 2 years and older already on an SMN2-targeted treatment, the 39th novel drug approval of 2026 on the FDA's Novel Drug Approvals list.
Verified against: FDA action day September 11, 2026 (BLA 761463 ORIG-1) · Announcement September 11, 2026 (sponsor announcement) · Goal date September 30, 2026 (Scholar Rock 8-K).
ISEMBYLD (apitegromab-mstn) approved as the first muscle-targeted treatment for spinal muscular atrophy, for adults and children 2 years and older already receiving an SMN2-targeted therapy (Sep 11 2026).
The PDUFA goal date was September 30, 2026 and the FDA approved on September 11, 19 days early. That goal date came from Scholar Rock's own 8-K of May 7, 2026, which is why the gap is quoted here as a measurement rather than an impression. The BLA was a resubmission: the FDA issued a Complete Response Letter on September 22, 2025 over observations at a third-party fill-finish facility, not over apitegromab's efficacy or safety, and Scholar Rock resubmitted on March 31, 2026 with two fill-finish facilities included.
Run-up figures are the stock's own daily closing path over the 120 trading days before the decision, from our price history. Cohort move is the historical median absolute decision-day move for this market-cap tier. History, not a prediction.
Primary source: Scholar Rock 8-K (EX-99.1), September 14, 2026
FDA record of the action: the FDA approved on September 11, 2026, per BLA 761463 ORIG-1 (FDA letter). The sponsor announced it the same day. Action dates on pdufa.bio come from the FDA's own record, not the press release. Every FDA action we hold, by FDA date: FDA approval letters.
| Fund | Shares | Value at quarter end | Filing |
|---|---|---|---|
| Redmile Group | 4,311,092 | $237.1 million | filed August 14, 2026 |
13F reports list long positions in common stock at quarter end, are due 45 days later, and show no short positions or later trades. Notes, warrants and other instruments are not counted as shares.
Yes. The FDA approved the application for ISEMBYLD (apitegromab-mstn) on September 11, 2026, per the FDA's own record. The primary source is linked on this page.
ISEMBYLD (apitegromab-mstn) is a Scholar Rock Holding Corporation (SRRK) program. The FDA decision came on September 11, 2026; the FDA's own record of the decision is linked on this page.
Measured over the 120 trading days before the decision (T-120 to T-1), SRRK moved +24.7%. That is the recorded price path into this specific event. Historical measurement, not a prediction, and not investment advice.