Dashboard/BMRN/VOXZOGO/event

PDUFAPDUFA Decision

VOXZOGO Under-5 sNDA PDUFA

BMRN·VOXZOGO·Achondroplasia·

Approved

current best date

NOV 20

2026

EXACT7 months

Takeaway

FDA decision deadline on extending VOXZOGO into achondroplasia patients aged 4 to <60 months. VOXZOGO is currently approved for ages 5+ and is BioMarin’s largest growth franchise.

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

MoACNP analog (FGFR3 antagonism)

VOXZOGO (vosoritide) is a once-daily subcutaneous injection developed by BioMarin for achondroplasia — the most common form of short-limb dwarfism, caused by a gain-of-function FGFR3 mutation that suppresses bone growth at the growth plates. The drug is a synthetic analog of C-type natriuretic peptide (CNP), a signaling molecule that inhibits the overactive FGFR3 receptor, allowing growth plates to develop more normally and increasing annualized height velocity in children. VOXZOGO is approved in the US for patients aged 5 and older; BioMarin is pursuing a label extension for infants from age 4 months.

Indication

Rare Disease

Achondroplasia

MeSH · D000130

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
GALAFOLDmigalastat	FOLD	small molecule	pharmacological chaperone of α-galactosidase A	PDUFA · Nov 26
KRESLADImarnetegragene autotemcel	RCKT	gene therapy	lentiviral ITGB2 transfer (LAD-I)	PDUFA · Oct 26

Prior BMRN reactions to PDUFA events

1 historical event · base rate, not prediction

Median 1W move

+39.9%

Median 1M move

+29.9%

Positive outcomes

1/ 1

100%

Negative outcomes

0/ 1

Date	Headline	Outcome	1W	1M	6M
Dec 2024	VOXZOGO — PDUFA — Approved	positive	+39.9%	+29.9%	+43.5%

Historical BMRN stock reaction to past PDUFA catalysts. Past performance is not a forecast — base rates anchor expectations, not outcomes. Positive rate 100%.

Disclosure trail

1 observation · sorted by confidence

Jan 15, 2026·3mo agopinned · highest confidence
HIGH confPR
top claim
NOV 202026
EXACT
“BioMarin plans to submit the supplemental NDA in mid-2026, with a target PDUFA action date of November 20, 2026 under standard review.”
conf 85%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.