VOXZOGO
vosoritidepeptideCNP analog (FGFR3 antagonism)
Achondroplasia
Approved
1 program·1 trial·View drug →
DashboardSell13,854$57.40−$795.2KSell81,444$61.97−$5.0MBuy12,062$78.57+$947.7KOption ex.14,089$74.21$1.0MSell4,163$64.74−$269.5KOption ex.15,400$59.40$914.8KOption ex.5,552$72.05$400.0K CardiologyCo-Investigator$575.5K5Interventional CardiologyCo-Investigator$539.7K6Heart FailureCo-Investigator$514.6K7Heart FailureCo-Investigator$501.0K7Interventional CardiologyCo-Investigator$449.3K7CardiologyCo-Investigator$424.1K4CardiologyCo-Investigator$413.7K7Interventional CardiologyCo-Investigator$390.9K7CardiologyResearch Grant$385.8K4Heart FailureResearch Grant$379.2K4
BMRNNASDAQ
BioMarin
BioMarin Pharmaceutical Inc. · San Rafael, CA · founded 1997
BioMarin Pharmaceutical, based in San Rafael, California, specializes in therapies for rare genetic diseases that affect children and adults. The commercial portfolio spans enzyme replacement therapies (NAGLAZYME, VIMIZIM, BRINEURA), oral small molecules for phenylketonuria (KUVAN, PALYNZIQ), the dwarfism drug VOXZOGO, and ROCTAVIAN — the first FDA-approved gene therapy for severe hemophilia A.
- Lead asset
- VOXZOGO · Approved · Achondroplasia
- peptide · CNP analog (FGFR3 antagonism)
- Pipeline
- 1 drug · 1 program
- 1 Rare Disease
- Modalities
- peptide×1
FocusRare DiseaseGene Therapy
$76.00+4.81%1Y
BMRN · daily close · illustrative · 1 catalyst marked
P3 readout+16%
1Y high$77.781Y low$64.94range$12.84(20%)past catalysts
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1 programmatch: modality
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Rhythm Pharmaceuticals
Rel.
0.32
Obesity / GLP-1peptide
2 programsmatch: modality
Pipeline & catalysts
Pipeline & catalysts
Pipeline by phase1programs
- Preclin0
- Ph10
- Ph20
- Ph30
- Filed0
- Approved1
Past catalyst impact
DateKindEventOutcome+1W+1M+6M
Jan 15, 2026ReadoutVOXZOGO Under-5 CANOPY ToplinePositive+15.5%+33.4%+26.5%
Dec 1, 2024PDUFAVOXZOGO — PDUFA — ApprovedPositive+39.9%+29.9%+43.5%
Nov 1, 2024Conf.VOXZOGO — ASH Late-Breaker — Updated SurvivalPositive+4.3%+10.0%+3.2%
Feb 5, 2024Enroll.VOXZOGO — Pivotal Enrollment CompleteNegative-5.0%-4.3%-4.0%
% change vs ticker close on the announcement date · backtest illustrative
Smart money
Insider activity
Insider activity · last 90 days
Form 4 trail · 7 transactions · 7 insiders
Net flow
−$5.2M
Buys
$947.7K
1 txns
Sells
$6.1M
3 txns
Largest
−$5.0M
CEO sell
Net flow per quarter · last 8Q
7 txns · sum −$5.16M
24Q325Q125Q326Q126Q2
buys · $948Ksells · $6.11M
Insider · roleActionSharesPriceValueDate
C. Iyer
CSO
Apr 5, 2026
L. Reyes
CEO
Mar 30, 2026
D. Park
Director
Mar 19, 2026
P. Park
COO
Mar 7, 2026
T. Park
COO
Feb 28, 2026
P. Müller
COO
Feb 7, 2026
K. Park
Director
Jan 25, 2026
Congressional trading
Congressional trading · STOCK Act · last 365d
6 trades · 4 members
Est. net flow
−$100K
midpoint · brackets only
Buys
3
Sells
3
Party mix
1 D4 R1 I
Member · chamberActionBracket~ midpointOwnerDate · disclosure
R
Partial sell$500K–$1.00M~$750KselfRep. N. Schaefer
House · WI
Apr 25, 2026
filed +29d
R
Buy$500K–$1.00M~$750KselfRep. M. Hartwell
House · FL
Apr 9, 2026
filed +22d
I
Buy$1.00M–$5.00M~$3.00MdependentSen. F. Park
Senate · ME
Mar 7, 2026
filed +26d
R
Buy$50K–$100K~$75KselfRep. N. Schaefer
House · WI
Nov 13, 2025
filed +17d
R
Sell$1.00M–$5.00M~$3.00MselfRep. M. Hartwell
House · FL
Nov 10, 2025
filed +35d
D
Partial sell$100K–$250K~$175KselfRep. H. Patel
House · IL
May 5, 2025
filed +26d
Source: Senate eFD & House Clerk PTR feeds · members must disclose within 45 days · midpoints reflect bracket midpoint, not actual transaction value
Top ETF holders
ETF holders · NAV disclosures
2 ETFs hold the position
Held by ETFs
$30.7M
aggregate position
Of market cap
0.27%
aggregate ETF share
Top holder
IBB0.36%
iShares Biotechnology ETF
TickerETF · familyThemeNAV% of NAVPosition
IBBBroad biotech$8.40B0.36%$30.2M
iShares Biotechnology ETF
iShares
PBEBroad biotech$175.0M0.25%$438K
Invesco Dynamic Biotechnology
Invesco
Source: ETF NAV disclosures + quarterly 13F filings · narrow-theme ETFs disclose holdings daily; broad ETFs quarterly · % of NAV is the position weight inside the fund
KOL network
KOL network · CMS OpenPayments
21 physicians paid · 102 disclosed records
Total 2025+2024
$7.03M
YoY
+24%
Co-Investigator$3.75MResearch Grant$1.76MEquity / Ownership$978.4KConsulting$252.4KSpeaking$248.4KTravel & Lodging$27.0KFood & Beverage$3.6K
Physician · affiliationSpecialtyTop categoryTotalRecords
Dr. Liam Romano
MD Anderson
Dr. Olivia Hoffman
Vanderbilt
Dr. Liam Lindberg
University of Michigan
Dr. Sofia Nakamura
UCSF
Dr. Kavya Tanaka
Mayo Clinic
Dr. Priya Nakamura
Stanford Medicine
Dr. Nadia Park
Johns Hopkins
Dr. Yuki Davies
UCSF
Dr. Liam Walsh
NYU Langone
Dr. Ahmed Nakamura
Stanford Medicine
Source: CMS OpenPayments · quarterly drops · physicians identified by NPI · aggregated across all programs
Government & IP
Federal lobbying
Federal lobbying · LDA · last 8 quarters
$1.09M disclosed · 3 firms engaged
YoY change
+22%
last 4Q vs prior 4Q
24Q3Q425Q1Q2Q3Q426Q1Q2
Top lobbying firms · last 8Q
Invariant$476K
6 quarters active
Brownstein Hyatt Farber Schreck$402K
6 quarters active
Squire Patton Boggs$215K
3 quarters active
Top issues lobbied
- Medicare coverage for CAR-T$402K
- BIOSIM Act / Biosimilars$238K
- Patent Trial and Appeal Board reforms$238K
- Orphan Drug Act amendments$72K
- Rare Cancer Funding Act$72K
Source: Senate LDA database (lda.senate.gov) · quarterly registrant filings · spend is the company-wide total disclosed
Government contracts
Federal contracts · USAspending.gov
1 award · 1 agency
Total awarded
$10.1M
across all programs
Active
$10.1M
option periods incl.
Top agency
VA$10.1M
largest active: Veterans Health Administration — Specialty Drug Supply
AgencyTitle · type · award IDStatusValueAwarded → ends
VAactive$10.1MAug 2025→ Dec 2027
Veterans Health Administration — Specialty Drug Supply
Contract · VA-87145913
Source: USAspending.gov · SAM.gov · every federal contract / grant / cooperative agreement is public · values are obligated awards (may include option periods)
U.S. Patents
U.S. Patents · USPTO
9 granted · 3 pending
Total in portfolio
12
Granted in last 12mo
1
Expiring < 2yr
0
Nearest expiry
Apr 2032
Patent #Title · inventor · drugTypeStatus · filedExpiry
US 2026/2337448 A1Composition of Matter
Small molecule modulators of VOXZOGO
D. Kowalski + 1 · VOXZOGO
pendingfiled Apr 2026
in prosecution
11,296,474Method of Use
Combination therapy comprising VOXZOGO
L. Kowalski + 4 · VOXZOGO
grantedfiled Apr 2021
exp. Apr 2041
15.0y left
11,836,463Method of Use
Pediatric methods of use for VOXZOGO
J. Nguyen + 3 · VOXZOGO
grantedfiled Apr 2022
exp. Apr 2042
16.0y left
US 2024/0040996 A1Process
Scalable cell-line manufacturing for VOXZOGO
S. Iyer + 4 · VOXZOGO
pendingfiled Apr 2024
in prosecution
10,205,202Method of Use
Pediatric methods of use for VOXZOGO
A. Goldstein + 1 · VOXZOGO
grantedfiled Apr 2019
exp. Apr 2039
13.0y left
US 2023/7921753 A1Formulation
Subcutaneous injectable formulations of VOXZOGO
K. Patel + 1 · VOXZOGO
pendingfiled Apr 2023
in prosecution
10,630,926Composition of Matter
Lipid nanoparticle formulations comprising VOXZOGO
L. Reyes + 4 · VOXZOGO
grantedfiled Apr 2018
exp. Apr 2038
12.0y left
11,209,933Method of Use
Combination therapy comprising VOXZOGO
C. Park + 1 · VOXZOGO
grantedfiled Apr 2019
exp. Apr 2039
13.0y left
showing 8 of 12 · full portfolio available on Pro
Source: USPTO PEDS & Patent Public Search · composition-of-matter is the headline asset · expiration = 20 years from filing
News
News
News · last 90 days
10 items from 4 categories
corporate5clinical2partnership2earnings1
1d ago
BMRN appoints new Chief Medical Officer
Business Wire·corporate
1d ago
BMRN adds independent director to Board
Business Wire·corporate
11d ago
BMRN appoints new Chief Medical Officer
Business Wire·corporate
24d ago
BMRN appoints new Chief Medical Officer
Business Wire·corporate
1mo ago
BMRN doses first patient in Phase 3 VOXZOGO study
PR Newswire·clinical
1mo ago
BMRN reports Q1 financial results and pipeline update
Business Wire·earnings
2mo ago
BMRN expands co-development partnership for next-gen pipeline
PR Newswire·partnership
2mo ago
BMRN enters licensing agreement with major pharma for VOXZOGO
PR Newswire·partnership
2mo ago
BMRN completes enrollment in VOXZOGO Phase 2 trial
FierceBiotech·clinical
3mo ago
BMRN expands manufacturing capacity at lead facility
PR Newswire·corporate
Sources: PR wires + biotech-trade publications + company IR · paywalled outlets ($) link to publisher · we never paraphrase the headline
Financials
Financials
Trading
Previous close
$76.00
Open
$76.53
Day Δ
-0.53
-0.69%
Day range
$75.39 – $77.14
52W range
$64.94 – $77.78
Avg daily volume
1.41M
Valuation & ownership
Enterprise value
$10.2B
Shares out
147.37M
Float
129.69M
Insider %
13.23%
Institutional %
48.23%
Beta
2.23
vs SPY · 52w
Balance sheet & burn
Cash + invest
$1.8B
Total debt
$818M
Debt / equity
4.23
Cash burn / Q
$113M
R&D spend TTM
$429M
23% of cash
ATM available
None
no active facility
Earnings
EPS latest
$-0.08
EPS Δ vs prior
+0.03
EPS estimate
$-0.15
next quarter
EPS prior
$-0.11
Next earnings
Jun 20, 2026
Rev guidance
$90M