Scholar Rock: Countdown to the Sept. 30 PDUFA for Apitegromab in SMA; Reiterate Positive Catalyst Watch

研报英文原文证据摘录

J P M O R G A N

North America Equity Research

06 August 2026

Scholar Rock

Countdown to the Sept. 30 PDUFA for Apitegromab in

SMA; Reiterate Positive Catalyst Watch

Overweight

SRRK, SRRK US

Price (05 Aug 26):$47.87

SMid Biotechnology

As we near the Sept. 30, 2026 PDUFA date for apitegromab's BLA for spinal muscular

atrophy (SMA), we think the comments from Scholar Rock underscore that they

remain on track for a decision by that time with two independent paths to approval

with two fill-finish facilities. Importantly, Scholar Rock emphasized that its

discussions with the Agency continue to progress very well. Taking a step back, we

believe that the clinical data generated to date, and the fact that the prior CRL was

related solely to observations at the Catalent Indiana facility (i.e., there were no other

approvability concerns), support the approval of the BLA for apitegromab in SMA on

or before its PDUFA date. More broadly, we anticipate a strong launch given the high

unmet need, which should help drive the stock long term, and ultimately we see

apitegromab as having multi-billion dollar revenue potential in SMA alone. Reiterate

Positive Catalyst Watch.

What data were required by the FDA from the second fill-finish facility?

Recall, following the March 3, 2026 Type C meeting where the company

agreed to the data package required for FDA review of the second fill-finish

facility, the resubmitted BLA included the second fill-finish facility. Scholar

Rock indicated that the data package for the facility included engineering runs

and PPQ runs, which aligned with what was agreed upon prior to the BLA

acceptance and was ahead of the FDA’s timeline for submission. Scholar Rock

noted that that they are in correspondence with the Agency and “everything is

straightforward and perfunctory.” Finally, the product is at the thirdparty labeling and packaging facility awaiting approval to support the launch.

How confident is Scholar Rock on the standing of the second fill-finish

facility? Scholar Rock emphasized that the second fill-finish facility has had

successful recent site inspections by the FDA and EMA, and none of the recent

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