Scholar Rock updates apitegromab BLA progress, starts FSHD Phase 2 trial

Scholar Rock reported on August 6, 2026 that its Biologics License Application for apitegromab in spinal muscular atrophy continues under FDA review, with the FDA reviewing the BLA using both the Catalent Indiana fill-finish site and a second, separate fill-finish facility as two independent routes toward an approval decision¹. The company said the FDA and Scholar Rock had agreed at a March 2026 Type C meeting on the data package needed to review the second facility, and that package has since been submitted, with the agency's review of it progressing¹. Scholar Rock said supply from the second facility is sufficient for commercialization if the drug is approved.¹ Separately, the FDA's classification of the April 2026 general site inspection at Catalent Indiana has not yet been issued.¹ The PDUFA date for apitegromab remains September 30, 2026.

On the regulatory front in Europe, the EMA's review of the apitegromab marketing application includes the Catalent Indiana facility and is waiting on the FDA's inspection classification, while Scholar Rock is separately discussing with EMA the possibility of adding the second fill-finish site to that application¹.

In pipeline news, Scholar Rock said it started a Phase 2 trial called FORGE testing apitegromab in about 60 patients with facioscapulohumeral muscular dystrophy, randomized 1:1 to receive either 10mg/kg IV apitegromab or placebo every 4 weeks for 52 weeks¹, with the primary endpoint set as change in mean lean muscle volume from baseline at 12 months¹. The company also said enrollment and dosing continue in the Phase 2 OPAL study (NCT07047144)¹, and that a Phase 1 study of SRK-439 in healthy volunteers is ongoing, with topline data expected in late 2026¹.

On finances, Scholar Rock reported cash, cash equivalents, and marketable securities of $492.1 million as of June 30, 2026, reflecting $62.8 million in net proceeds from its at-the-market program during the quarter¹.