KRESLADI BLA PDUFA (Fanconi Anemia Type A)

RCKT·KRESLADI·Fanconi Anemia·

Filed

current best date

OCT 09

2026

EXACT5 months

Takeaway

FDA decision deadline for KRESLADI in leukocyte adhesion deficiency type I (LAD-I), an ultra-rare pediatric immune disorder. Granted Priority Review. Would be Rocket’s first commercial gene therapy approval.

What’s at stake

market · comparables · base rate · prior moves

Market opportunity

—

company filings

Peer comparables

1 readout

ClinicalTrials.gov

Stage base rate

~91%

BIO Industry Analysis 2023 (2011–2020)

Avg prior-readout move

—

historical price reactions

Drug

gene therapy

KRESLADI

marnetegragene autotemcel · INN

MoAlentiviral ITGB2 transfer (LAD-I)

KRESLADI (marnetegragene autotemcel) is a one-time ex vivo lentiviral gene therapy developed by Rocket Pharmaceuticals for leukocyte adhesion deficiency type I (LAD-I) — an ultra-rare, fatal pediatric immune disorder in which ITGB2 gene mutations prevent white blood cells from reaching sites of infection, leaving patients unable to fight bacteria. The therapy harvests a patient's own blood stem cells, inserts a functional ITGB2 gene via lentiviral vector, and reinfuses the corrected cells after myeloablative conditioning to restore immune function. Without treatment, severe LAD-I is typically fatal in infancy; KRESLADI received FDA Priority Review and would be Rocket's first commercial gene therapy.

Indication

Rare Disease

Fanconi Anemia

MeSH · D005199

No primer in glossary yet.

Source signal

This event was extracted from a primary disclosure. The full chain of citations is in the disclosure trail below.

Glossary · what this readout is measuring

2 terms detected in the takeaway

BLAregulatory
Biologics License Application
FDA submission for approval of a biologic (antibody, gene/cell therapy, vaccine). Same clock as NDA.
PDUFAregulatory
Prescription Drug User Fee Act
The FDA's self-imposed review deadline for an NDA/BLA. Standard reviews are ~10 months from filing; priority reviews are ~6 months.

Competitive landscape

3 peers in Rare Disease · ranked by indication + modality match

Drug	Company	Modality	Mechanism	Next catalyst
ELEVIDYSdelandistrogene moxeparvovec	SRPT	gene therapy	AAVrh74-microdystrophin transfer	ADCOM · May 26
VOXZOGOvosoritide	BMRN	peptide	CNP analog (FGFR3 antagonism)	PDUFA · Nov 26
GALAFOLDmigalastat	FOLD	small molecule	pharmacological chaperone of α-galactosidase A	PDUFA · Nov 26

Disclosure trail

1 observation · sorted by confidence

Feb 26, 2026·2mo agopinned · highest confidence
HIGH confPR
top claim
OCT 092026
EXACT
“The FDA has accepted for filing and granted Priority Review to the BLA for KRESLADI in leukocyte adhesion deficiency type I, with a PDUFA action date of October 9, 2026.”
conf 97%via llm

MethodologyEvery catalyst date is anchored to a primary source. Disclosures with confidence ≥ 0.85 auto-publish; the rest are reviewed by a human within 24 hours. We never interpret data — we organize public information.