Deramiocel for DMD Cardiomyopathy: AdCom Votes Against Approval by 9-3

研报英文原文证据摘录

Deramiocel for DMD Cardiomyopathy: AdCom Votes Against Approval by 9-3

Update

July 29, 2026 10:54 PM GMT

Morgan Stanley MUFG Securities Co., Ltd.+MNippon Shinyaku (4516) | Japan Jaeheon Lee

Equity Analyst

Deramiocel for DMD Jaeheon.Lee@morganstanleymufg.comShinichiro Muraoka +81 3 6836-8443

Shinichiro.Muraoka@morganstanleymufg.com +81 3 6836-5424

Cardiomyopathy: AdCom Votes

Against Approval by 9-3

Key Takeaways Nippon Shinyaku (4516.T, 4516 JT)

The FDA's Advisory Committee (AdCom) voted 9-3 that the efficacy data are Pharmaceuticals | Japan

insufficient to support approval. Safety concerns and issues related to data Stock Rating Underweight

Industry View In-Line

interpretation were also discussed. Price target ¥2,600

Shr price, close (Jul 29, 2026) ¥3,550

Committee members voting against approval highlighted that the results for Mkt cap, curr, basic (bn) ¥239.1

LVEF (left ventricular ejection fraction) and PUL (Performance of Upper Limb) Avg daily trading value (bn) ¥1.6

varied significantly depending on changes to the assumptions used in the

statistical analyses.

Those voting in favor emphasized (1) the significant unmet need in DMD and (2)

regulatory flexibility for rare diseases.

The PDUFA date is scheduled for Aug 22, 2026.

Slightly negative for the share price: The market already understood the FDA's

cautious stance toward approval following the release of FDA briefing documents

on Jul 27. Many investors had viewed approval as unlikely. While we do not view the

AdCom's 9-3 vote against approval on Jul 29 as a surprise, we believe uncertainty

surrounding the FDA's final decision on Aug 22 has increased further. Given that the

stock had already priced in the possibility of a negative outcome to some extent,

downside pressure on the share price may be limited.