BioMarin halts several VOXZOGO Phase 2 programs

BioMarin halts several VOXZOGO Phase 2 programs

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What did BioMarin (BMRN) announce about its VOXZOGO Phase 2 trials?

Why is BioMarin stopping VOXZOGO studies in Turner, SHOX and ACAN-deficiency?

Have SCFE events been seen with VOXZOGO in achondroplasia patients at BioMarin?

Which VOXZOGO CANOPY trials at BioMarin will continue after this update?

Did BioMarin observe SCFE in its hypochondroplasia VOXZOGO trials?

How does BioMarin describe the risks around these VOXZOGO trial changes?

BioMarin Pharmaceutical Inc. is halting dosing and enrollment in its Phase 2 trials of VOXZOGO for Turner syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency.

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8-K

BioMarin Pharmaceutical Inc. is halting dosing and enrollment in its Phase 2 trials of VOXZOGO for Turner syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency. This decision follows several slipped capital femoral epiphysis (SCFE) events reported in two ongoing investigator-sponsored trials using VOXZOGO in these conditions.

BioMarin states that SCFE has not been seen in its own Phase 2 trials in these indications, nor among more than 5,000 infants and children treated with VOXZOGO for achondroplasia over about 10,000 patient-years of clinical and post-marketing safety data. No SCFE cases have appeared in VOXZOGO trials for hypochondroplasia. The Phase 2 CANOPY trials of VOXZOGO will continue for children with Noonan syndrome and for most participants with idiopathic short stature without ACAN-deficiency.

None.

Safety-driven halt of multiple VOXZOGO Phase 2 programs: BioMarin is discontinuing dosing and enrollment for VOXZOGO in Turner syndrome, SHOX-deficiency and ACAN-deficiency after SCFE events in investigator-sponsored trials, reducing near-term expansion opportunities for this therapy in several additional growth-disorder indications.

BioMarin faces a safety-related setback in several VOXZOGO expansion trials.

BioMarin is stopping enrollment and dosing in Phase 2 VOXZOGO studies for Turner syndrome, SHOX-deficiency and ACAN-deficiency after several SCFE events in investigator-sponsored trials. This narrows near-term label expansion prospects for VOXZOGO in specific growth-disorder populations.

The company emphasizes that SCFE has not been observed in its own Phase 2 programs in these conditions, nor among more than 5,000 treated achondroplasia patients with over 10,000 patient-years of accumulated safety data, and none in hypochondroplasia trials. That distinction suggests the signal may be setting- or population-specific but still requires caution.

Phase 2 CANOPY trials in Noonan syndrome and idiopathic short stature without ACAN-deficiency remain ongoing, preserving some future-growth opportunities for VOXZOGO. Actual impact will depend on further safety evaluations, regulatory interactions, and future disclosures in BioMarin’s SEC filings referenced in its risk-factor discussion.

Item 8.01 — Other Events

1 item

Item 8.01 Other Events Other

Voluntary disclosure of events the company deems important to shareholders but not covered by other items.

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BioMarin announced it will discontinue dosing and enrollment in Phase 2 VOXZOGO trials for Turner syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency. The change follows several SCFE safety events reported in two ongoing investigator-sponsored trials using VOXZOGO in these conditions.

The company is acting after several slipped capital femoral epiphysis (SCFE) events were observed in two investigator-sponsored trials involving VOXZOGO. While these events were not seen in BioMarin’s own Phase 2 trials, the safety signal led to stopping those specific programs.

BioMarin reports no SCFE events among more than 5,000 infants and children treated with VOXZOGO for achondroplasia. That experience includes 10 years of clinical research and over 10,000 patient-years of safety data from clinical studies and post-marketing surveillance.

Phase 2 CANOPY trials of VOXZOGO will continue in children with Noonan syndrome and those with idiopathic short stature without ACAN-deficiency. BioMarin states that approximately 95% of children enrolled in the idiopathic short stature trial fall into the non-ACAN-deficient group.

BioMarin states there have been no observed SCFE cases in its clinical trials of VOXZOGO for hypochondroplasia. This adds to the broader safety dataset, alongside experience in achondroplasia and BioMarin’s own Phase 2 trials for the other studied conditions.

BioMarin frames its statements as forward-looking and subject to risks, including clinical trial results, regulatory decisions and factors discussed in its Form 10-K risk factors. It cautions that actual outcomes may differ and disclaims any obligation to update these statements.

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Accepted by SEC EDGAR 03/16/2026 - 08:30 AM

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

FORM 8-K

CURRENT REPORT

Pursuant to Section 13 OR 15(d)

of The Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): March 16, 2026

BioMarin Pharmaceutical Inc.

(Exact name of registrant as specified in its charter)

Delaware

000-26727

68-0397820

(State or other jurisdiction

of incorporation or organization)

(Commission

File Number)

(I.R.S. Employer

Identification No.)


770 Lindaro Street	San Rafael	California	94901
(Address of Principal Executive Offices)			(Zip Code)

(415) 506-6700

(Registrant’s telephone number, including area code)

Not Applicable

(Former name or former address, if changed since last report.)

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions (see General Instruction A.2. below):

☐	Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

☐	Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

☐	Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

☐	Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

Securities registered pursuant to Section 12(b) of the Act:

Title of each class

Trading

Symbol(s)

Name of each exchange

on which registered

Common Stock, par value $0.001

BMRN

The Nasdaq Global Select Market

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§240.12b-2 of this chapter).

Emerging growth company ☐

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐

Item 8.01 Other Events.

On March 16, 2026, BioMarin Pharmaceutical Inc. (“BioMarin”) announced its decision to discontinue dosing and enrollment in its Phase 2 trials for VOXZOGO in Turner Syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency following the occurrence of several slipped capital femoral epiphysis (SCFE) events in two ongoing investigator-sponsored trials. SCFE events have not been observed in the Phase 2 BioMarin trials in these same conditions, nor have any cases been observed in the more than 5,000 infants and children who have received VOXZOGO for achondroplasia, which includes 10 years of clinical research and more than 10,000 patient-years of safety data in clinical studies and post-marketing surveillance. There have similarly been no observed cases in BioMarin’s clinical trials for hypochondroplasia. The Phase 2 CANOPY trials of VOXZOGO in children with Noonan syndrome, as well as those living with idiopathic short stature (ISS) without ACAN-deficiency, which represents approximately 95% of children enrolled in the ISS trial, will continue as planned.

BioMarin uses its investor relations website (https://investors.biomarin.com) as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD.

Forward-Looking Statements

This Current Report on Form 8-K contains forward-looking statements about the business prospects of BioMarin Pharmaceutical Inc. (BioMarin), including without limitation, statements about: BioMarin’s decision to discontinue enrollment and dosing of VOXZOGO in children with Turner syndrome, SHOX-deficiency and Aggrecan (ACAN)-deficiency in BioMarin’s Phase 2 CANOPY clinical trials of VOXZOGO; and plans to continue the Phase 2 CANOPY trials of VOXZOGO in children with Noonan syndrome, as well as those living with Idiopathic Short Stature (ISS) without ACAN-deficiency. These forward-looking statements are predictions and involve risks and uncertainties such that actual results may differ materially from these statements. These risks and uncertainties include, among others, the results and timing of current and planned preclinical studies and clinical trials and the release of data from those trials; the content and timing of decisions by regulatory authorities; and those factors detailed in BioMarin’s filings with the Securities and Exchange Commission (SEC), including, without limitation, the factors contained under the caption “Risk Factors” in BioMarin’s Annual Report on Form 10-K for the year ended December 31, 2025, as such factors may be updated by any subsequent filings with the SEC. Investors are urged not to place undue reliance on forward-looking statements, which speak only as of the date hereof. BioMarin is under no obligation, and expressly disclaims any obligation to update or alter any forward-looking statement, whether as a result of new information, future events or otherwise.

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.

Source: View Original Filing on SEC EDGAR

3 documents

BioMarin Pharmaceutical Inc.,

a Delaware corporation

Date: March 16, 2026

/s/ G. Eric Davis

G. Eric Davis

Executive Vice President, Chief Legal Officer