Dashboard/BMRN/VOXZOGO/event

ReadoutTopline Readoutreported

VOXZOGO Under-5 CANOPY Topline

BMRN·VOXZOGO·Achondroplasia·

Approved

·NCT04638543

reported

JAN 15

2026

EXACT

Takeaway

Reported 2026-01-15: VOXZOGO produced a mean +1.10 cm/yr greater growth velocity vs. placebo (p=0.0009) at week 52 in achondroplasia patients aged 4 to <60 months. Supports the November 20 sNDA PDUFA.

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.

Trial

completed

NCT04638543 ↗

CANOPY Phase 2 Vosoritide in Children Under 5

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.

Reference data · comparable readouts

How Appr readouts in Rare Disease have landed.

Reference, not prediction. We surface the historical record so you can read it yourself. We never extrapolate to the upcoming event.

Positive

0/0

0% in record

Primary endpoint hit

across 0 readouts

Drug · sponsorPhase · yearOutcomeKey metricSource

eladocagene exuparvovec

PTCT · AADC deficiency

Ph3 · Nov 2023positivemotor function gains durable to 5yPR

vutrisiran

ALNY · hATTR amyloidosis

Ph3 · Sep 2021positivemNIS+7 reduction 56% vs pboPR

sorted by phase match, then recency · sources span PR wires, CT.gov, FDA notices, and conference presentations · we never editorialize the outcome label

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 Readout events

1 historical event · base rate, not prediction

Median 1W move

+15.5%

Median 1M move

+33.4%

Positive outcomes

1/ 1

100%

Negative outcomes

0/ 1

Date	Headline	Outcome	1W	1M	6M
Jan 2026	VOXZOGO Under-5 CANOPY Topline	positive	+15.5%	+33.4%	+26.5%

Historical BMRN stock reaction to past Readout 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
JAN 152026
EXACT
“Children aged 4 to <60 months treated with VOXZOGO achieved a mean annualized growth velocity 1.10 cm/yr greater than placebo at week 52 (95% CI 0.46–1.74; p=0.0009).”
conf 97%via human

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.