← Back to Calendar

KRESLADI (marnetegragene autotemcel (RP-L201))

Rocket Pharma · $RCKT
Priority Review Breakthrough Therapy Orphan Drug Rare Pediatric Disease BLA
PDUFA Date
March 28, 2026
Date Status
16d ago (past)
Review Type
Priority (6 mo)
93%
Baseline PoA
Orphan/rare disease designation approval rate
Based on FDA historical approval rates by submission type (2015–2025)
Dynamic PoA
Coming soon
xAI Sentiment

FDA Decision

✅
✓ FDA Approved
Decision issued: March 26, 2026
FDA APPROVED March 26, 2026 — accelerated approval, 2 days before PDUFA target date of March 28. First FDA-approved gene therapy for severe LAD-I. Also received Rare Pediatric Disease Priority Review Voucher.
Source: https://www.fda.gov/news-events/press-announcements/fda-approves-first-gene-therapy-severe-leukocyte-adhesion-deficiency-type-i

Live Company Data NGM

Updated 1m ago · Data: FMP
Current Price
$3.49 -29.35%
$-1.45 today
Day: $3.48 – $3.64
Market Cap
N/A
Shares out: 109.12M
Float: 87.08M
52-Week Range
$2.19
$8.26
Current price is at 21% of 52-week range
Avg Volume
3.41M
Beta
0.57
vs. S&P 500
Sector
Healthcare
Biotechnology
⚠️ Financial data is for informational purposes only. Not investment advice. Prices may be delayed.
Track $RCKT catalysts → get weekly FDA alerts Subscribe Free on Substack →

Indication

Leukocyte adhesion deficiency-I (LAD-I)

Key Notes

FDA APPROVED March 26, 2026 — accelerated approval, 2 days before PDUFA target date of March 28. First FDA-approved gene therapy for severe LAD-I due to biallelic ITGB2 variants without HLA-matched sibling donor. Rocket Pharma's first commercial product. Approved via RMAT pathway. Also received Rare Pediatric Disease Priority Review Voucher — Rocket plans to monetize the voucher for non-dilutive capital. "Minimal viable launch" planned; company priorities remain Danon disease and cardiovascular gene therapy pipeline.
⚠️ Not Investment Advice: This information is provided for educational purposes only. PDUFA dates are FDA target action dates, not guaranteed approval dates. The FDA may extend the review period, issue Complete Response Letters, or take unexpected actions. Probability of approval scores are based on historical base rates and do not account for drug-specific factors. Always consult a financial advisor before making investment decisions.

Discussion

Share your analysis, research, or questions about this catalyst. No account required.

Rate limit: 5/hr per email
Loading comments...
← Back to Full Calendar