BioMarin Pharmaceutical Inc. filed a Form 8-K with the SEC on September 15, 2026, disclosing its decision to discontinue development of VOXZOGO for children with Noonan syndrome.

Key Highlights

  • Program discontinued: BioMarin announced it will stop development of VOXZOGO specifically for children with Noonan syndrome.
  • Decision factors: According to the filing, the company cited study feasibility and the current treatment environment for children with Noonan syndrome as factors in the decision.
  • Safety and efficacy unaffected: The filing states the discontinuation "was not related to the safety or efficacy of VOXZOGO."
  • Signed by CLO: The report was signed by G. Eric Davis, Executive Vice President and Chief Legal Officer of BioMarin Pharmaceutical Inc.

BioMarin Ends VOXZOGO Noonan Syndrome Program After Feasibility Review

BioMarin Pharmaceutical Inc. (NASDAQ:BMRN), a Delaware-incorporated biopharmaceutical company headquartered in San Rafael, California, disclosed on September 15, 2026 that it has chosen to discontinue its development program for VOXZOGO in children with Noonan syndrome. The company reported the decision under Item 8.01 (Other Events) of the Form 8-K filing.

According to the filing, BioMarin reached this conclusion after assessing multiple factors, specifically including "the feasibility of the study and the current treatment landscape for children with Noonan syndrome." The company made clear that the discontinuation was not driven by concerns about the product's safety or efficacy profile. VOXZOGO is an existing BioMarin product; this filing addresses only the termination of one specific development program for an additional patient population, children with Noonan syndrome, and does not indicate any broader changes to the product's approved uses or ongoing programs as described in this disclosure.

The 8-K was authorized and signed by G. Eric Davis, the company's Executive Vice President and Chief Legal Officer, on behalf of BioMarin Pharmaceutical Inc. No financial metrics, clinical data, or revised guidance were included in the filing. The company's common stock carries a par value of $0.001 per share and is listed on The Nasdaq Global Select Market under the ticker symbol BMRN.

What Was Disclosed

BioMarin Pharmaceutical Inc. filed an 8-K on September 15, 2026 reporting the discontinuation of VOXZOGO development for children with Noonan syndrome, citing study feasibility and treatment environment considerations, and specifying that safety and efficacy were not factors in the decision.