Tuesday, March 3, 2026

BioMarin withdraws hemophilia gene therapy Roctavian

BioMarin is discontinuing Roctavian, a one-time gene therapy for hemophilia A, and is withdrawing the product from the market. The company said the decision follows an attempt to divest the therapy that did not result in a qualified buyer.

Roctavian was approved in 2023 as the first one-time gene therapy for hemophilia A. Over the past months, BioMarin had already scaled down operations and concentrated commercial activity on the U.S., Germany and Italy, where reimbursement frameworks were in place.

Charges and recent sales

BioMarin expects charges of about $240 million in the fourth quarter of 2025 tied to the withdrawal. The company said this includes a $119 million inventory write-off and $118 million in asset impairments.

Roctavian generated $36 million in sales in 2025, compared with $26 million in 2024.

Limited uptake amid competition and access hurdles

Despite some use late in its lifecycle, broad uptake remained challenging. In hemophilia A, Roctavian faced competition from established and newer therapies, including Roche’s Hemlibra, Sanofi’s Altuviiio and Novo Nordisk’s Alhemo. BioMarin also pointed to common gene therapy barriers such as patient eligibility constraints and reimbursement complexity.

BioMarin’s next chapter

As Roctavian exits, BioMarin is preparing for a major portfolio expansion through its planned $4.8 billion acquisition of Amicus Therapeutics, announced late last year and expected to close in the second quarter of this year. The deal would add Galafold for Fabry disease and the Pombiliti-Opfolda combination for Pompe disease.

BioMarin’s 2026 guidance calls for $3.3 billion to $3.4 billion in full-year sales, excluding any contribution from the Amicus products. For 2025, BioMarin reported $3.2 billion in revenue, up 13% year over year. The company attributed growth to gains across its enzyme therapy portfolio and Voxzogo for achondroplasia, which posted $927 million in 2025 sales.

Analysts have highlighted increasing competitive pressure in achondroplasia, including BridgeBio’s oral candidate infigratinib, which reported a statistically significant improvement in annualized height velocity from baseline in a phase 3 study in children. BioMarin is also advancing an early-stage achondroplasia program, BMN 333, after reporting positive phase 1 results.