Sarepta Therap

$Sarepta Therap(SRPT.US) | Mizuho reiterates 𝐎𝐮𝐭𝐩𝐞𝐫𝐟𝐨𝐫𝐦 on 𝐒𝐚𝐫𝐞𝐩𝐭𝐚, maintains 𝐏𝐓 𝐚𝐭 $𝟑𝟏 following 𝐩𝐨𝐬𝐢𝐭𝐢𝐯𝐞 𝐅𝐃𝐀 𝐧𝐞𝐰𝐬

Analyst sees FDA's sNDA acceptance for two DMD drugs as a positive, de-risking event given past trial data concerns.

📢 𝐉𝐔𝐒𝐓 𝐈𝐍: FDA Accepts $Sarepta Therap(SRPT.US) Sarepta's sNDAs for AMONDYS 45 and VYONDYS 53

👉 𝐊𝐞𝐲 𝐇𝐢𝐠𝐡𝐥𝐢𝐠𝐡𝐭𝐬:

➤ FDA accepted 𝐒𝐚𝐫𝐞𝐩𝐭𝐚'𝐬 sNDAs for 𝐀𝐌𝐎𝐍𝐃𝐘𝐒 𝟒𝟓 and 𝐕𝐘𝐎𝐍𝐃𝐘𝐒 𝟓𝟑.

➤ Applications seek to convert both therapies from 𝐚𝐜𝐜𝐞𝐥𝐞𝐫𝐚𝐭𝐞𝐝 to 𝐭𝐫𝐚𝐝𝐢𝐭𝐢𝐨𝐧𝐚𝐥 approval.

➤ FDA set a 𝐏𝐃𝐔𝐅𝐀 action date of 𝐅𝐞𝐛. 𝟐𝟖, 𝟐𝟎𝟐𝟕.

➤ Filings are supported by 𝐏𝐡𝐚𝐬𝐞 𝟑 𝐄𝐒𝐒𝐄𝐍𝐂𝐄 data, real-world evidence, and safety results.

➤ ESSENCE missed its 𝐩𝐫𝐢𝐦𝐚𝐫𝐲 𝐞𝐧𝐝𝐩𝐨𝐢𝐧𝐭, but post-hoc analyses favored treatment.

➤ VYONDYS 53 was associated with a 𝟕.𝟓-𝐲𝐞𝐚𝐫 delay in nighttime ventilation.

➤ AMONDYS 45 was associated with a 𝐬𝐭𝐚𝐭𝐢𝐬𝐭𝐢𝐜𝐚𝐥𝐥𝐲 𝐬𝐢𝐠𝐧𝐢𝐟𝐢𝐜𝐚𝐧𝐭 slowing of lung function decline.

➤ More than 𝟏,𝟖𝟎𝟎 patients have received Sarepta's exon-skipping therapies worldwide.

👉 𝐖𝐡𝐲 𝐈𝐭 𝐌𝐚𝐭𝐭𝐞𝐫𝐬:

➤ Traditional approval could strengthen the therapies' 𝐫𝐞𝐠𝐮𝐥𝐚𝐭𝐨𝐫𝐲 standing and long-term availability.

➤ FDA acceptance marks a key 𝐫𝐞𝐠𝐮𝐥𝐚𝐭𝐨𝐫𝐲 milestone for Sarepta's DMD franchise.

➤ Positive FDA decisions could support broader 𝐩𝐡𝐲𝐬𝐢𝐜𝐢𝐚𝐧 and payer confidence.

👉 𝐄𝐱𝐩𝐞𝐫𝐭 𝐒𝐭𝐚𝐭𝐞𝐦𝐞𝐧𝐭:

➤ "The submissions draw on the ESSENCE study and years of published real-world evidence, which together offer a fuller understanding of how these therapies benefit patients and change the progression of disease." — 𝐋𝐨𝐮𝐢𝐬𝐞 𝐑𝐨𝐝𝐢𝐧𝐨-𝐊𝐥𝐚𝐩𝐚𝐜, Ph.D., President of Research and Development and Technical Operations at Sarepta Therapeutics.

$Sarepta Therap(SRPT.US) | 𝐒𝐚𝐫𝐞𝐩𝐭𝐚: H.C. Wainwright reiterates 𝐒𝐞𝐥𝐥, maintains 𝐏𝐓 𝐚𝐭 $𝟓

Analyst sees rising efficacy risks from new Roche study, with limited upside and significant downside to core U.S. ELEVIDYS franchise.

Source: Hardik Shah