BioMarin ends VOXZOGO work in Noonan syndrome

BioMarin is discontinuing development of VOXZOGO in Noonan syndrome, citing feasibility and treatment landscape, not safety or efficacy.

Rhea-AI Filing Summary

BioMarin Pharmaceutical Inc. (BMRN) reported that on September 15, 2026 it decided to discontinue development of VOXZOGO for children with Noonan syndrome. The company states this decision followed an assessment of study feasibility and the current treatment landscape, and was not related to the safety or efficacy of VOXZOGO.

BioMarin also reiterates that it uses its investor relations website as a channel for disclosing material non-public information and for complying with Regulation FD.

8-K Event Classification

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.

Key Figures

Decision date: September 15, 2026 Security class: Common Stock, par value $0.001 Trading symbol: BMRN

3 metrics

Decision date September 15, 2026 Date BioMarin decided to discontinue VOXZOGO development for Noonan syndrome

Security class Common Stock, par value $0.001 Class of BioMarin shares listed on The Nasdaq Global Select Market

Trading symbol BMRN Ticker for BioMarin common stock

Key Terms

VOXZOGO, Noonan syndrome, Regulation FD, Emerging growth company

4 terms

VOXZOGO medical

"discontinue development of VOXZOGO for children with Noonan syndrome"

Noonan syndrome medical

"development of VOXZOGO for children with Noonan syndrome"

Regulation FD regulatory

"for complying with its disclosure obligations under Regulation FD"

Regulation FD is a rule that prevents company insiders, like executives, from sharing important information with some people before others get it. It matters because it helps ensure all investors have equal access to key news, making the stock market fairer and reducing chances of insider trading.

Emerging growth company regulatory

"Emerging growth company"

An emerging growth company is a recently public or smaller public firm that qualifies for temporary, lighter regulatory and disclosure rules to reduce the cost and effort of being public. For investors, it means the company may provide less historical financial detail and face fewer reporting requirements than larger firms, so it can grow more quickly but also carries higher uncertainty—like buying a promising early-stage product with fewer user reviews.

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

What did BioMarin (BMRN) announce about VOXZOGO in this 8-K?

BioMarin announced it will discontinue development of VOXZOGO for children with Noonan syndrome as of September 15, 2026, after assessing study feasibility and the current treatment landscape for these patients.

Is the discontinuation of VOXZOGO for Noonan syndrome related to safety or efficacy issues?

No. BioMarin states that the decision to discontinue VOXZOGO development for Noonan syndrome was not related to the safety or efficacy of VOXZOGO, but instead to feasibility and the current treatment landscape.

Does this 8-K affect VOXZOGO outside the Noonan syndrome program for BMRN?

The filing specifically addresses discontinuation of VOXZOGO development for children with Noonan syndrome. It does not discuss VOXZOGO in other indications.

How does BioMarin (BMRN) say it complies with Regulation FD?

BioMarin states that it uses its investor relations website as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD.

When did BioMarin decide to discontinue VOXZOGO development for Noonan syndrome?

BioMarin states that on September 15, 2026, it announced the decision to discontinue development of VOXZOGO for children with Noonan syndrome.

AI-generated analysis. How Rhea-AI works. Not financial advice.

Understanding 8-K Material Events →

See more from StockTitan in Google Search and AI answers. Adds StockTitan as a preferred source · opens Google

Add on Google Not now

Available on EDGAR 09/15/2026 - 08:32 AM Accepted by SEC EDGAR 09/15/2026 - 08:31 AM Learn about SEC filing dates

0001048477false00010484772026-09-152026-09-15

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): September 15, 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 September 15, 2026, BioMarin Pharmaceutical Inc. (BioMarin) announced its decision to discontinue development of VOXZOGO for children with Noonan syndrome. BioMarin made this decision after assessing a number of factors, including the feasibility of the study and the current treatment landscape for children with Noonan syndrome. This decision was not related to the safety or efficacy of VOXZOGO.

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.

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.

						BioMarin Pharmaceutical Inc., a Delaware corporation
Date: September 15, 2026			By:			/s/ G. Eric Davis
						G. Eric Davis
						Executive Vice President, Chief Legal Officer

Filing Exhibits & Attachments

3 documents

Other Documents

• EX-101 XBRL TAXONOMY EXTENSION SCHEMA DOCUMENT 1.8 KB
• EX-101 XBRL TAXONOMY EXTENSION LABEL LINKBASE DOCUMENT 21.4 KB
• EX-101 XBRL TAXONOMY EXTENSION PRESENTATION LINKBASE DOCUMENT 12.3 KB