Dashboard/FOLD/GALAFOLD/event

PDUFAPDUFA Decision

GALAFOLD Pediatric Fabry sNDA PDUFA

FOLD·GALAFOLD·Fabry Disease·

Approved

current best date

NOV 15

2026

EXACT7 months

Takeaway

FDA decision deadline on extending GALAFOLD to pediatric Fabry patients aged 2 to <12 with amenable GLA mutations. GALAFOLD is currently approved for ages 12+; alternative ERT in same disease: Fabrazyme (Sanofi).

What’s at stake

market · comparables · base rate · prior moves

Market opportunity

—

company filings

Peer comparables

1 readout

ClinicalTrials.gov

Stage base rate

~100%

BIO Industry Analysis 2023 (2011–2020)

Avg prior-readout move

—

historical price reactions

MoApharmacological chaperone of α-galactosidase A

GALAFOLD (migalastat) is an oral pharmacological chaperone developed by Amicus Therapeutics taken every other day for Fabry disease — an X-linked lysosomal storage disorder in which GLA mutations impair the alpha-galactosidase A (alpha-Gal A) enzyme, causing glycolipid accumulation in blood vessels, kidneys, heart, and nerves that leads to renal failure, cardiomyopathy, and early stroke. Rather than replacing the enzyme from outside the cell, migalastat enters cells and stabilizes specific misfolded mutant alpha-Gal A proteins, allowing them to reach the lysosome and function. GALAFOLD is approved for patients aged 12+ with amenable GLA mutations; Amicus is pursuing approval for children aged 2 to under 12.

Indication

Rare Disease

Fabry Disease

MeSH · D000795

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

1 term detected in the takeaway

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
KRESLADImarnetegragene autotemcel	RCKT	gene therapy	lentiviral ITGB2 transfer (LAD-I)	PDUFA · Oct 26

Prior FOLD reactions to PDUFA events

1 historical event · base rate, not prediction

Median 1W move

-39.1%

Median 1M move

-27.3%

Positive outcomes

0/ 1

Negative outcomes

1/ 1

100%

Date	Headline	Outcome	1W	1M	6M
Dec 2023	GALAFOLD — PDUFA — CRL Issued	negative	-39.1%	-27.3%	-40.6%

Historical FOLD stock reaction to past PDUFA catalysts. Past performance is not a forecast — base rates anchor expectations, not outcomes. Positive rate 0%.

Disclosure trail

1 observation · sorted by confidence

Mar 30, 2026·1mo agopinned · highest confidence
HIGH confPR
top claim
NOV 152026
EXACT
“The FDA has accepted the sNDA for GALAFOLD in pediatric Fabry patients aged 2 to <12 with amenable mutations and assigned a PDUFA action date of November 15, 2026.”
conf 95%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.