BioMarin scraps BMN 401, ending its $270 million Inozyme bet

10 August 2026

US biotech BioMarin Pharmaceutical (Nasdaq: BMRN) has pulled the plug on BMN 401, discontinuing the enzyme replacement therapy across all indications just over a year after it paid around $270 million to buy the asset along with its developer, Inozyme Pharma.

The decision, disclosed alongside second-quarter results on August 6, follows the pivotal Phase III ENERGY 3 trial in children aged one to 12 with ENPP1 deficiency, which in May met only one of its two co-primary endpoints.

This article is accessible to registered users, to continue reading please register for free. A free trial will give you access to exclusive features, interviews, round-ups and commentary from the sharpest minds in the pharmaceutical and biotechnology space for a week. If you are already a registered user please login. If your trial has come to an end, you can subscribe here.

Try before you buy

Free

7 day trial access

Take a Free Trial

All the news that moves the needle in pharma and biotech
Exclusive features, podcasts, interviews, data analyses and commentary from our global network of life sciences reporters.
Receive The Pharma Letter daily news bulletin, free forever.

Become a subscriber

£820

Or £77 per month

Subscribe Now

Unfettered access to industry-leading news, commentary and analysis in pharma and biotech.
Updates from clinical trials, conferences, M&A, licensing, financing, regulation, patents & legal, executive appointments, commercial strategy and financial results.
Daily roundup of key events in pharma and biotech.
Monthly in-depth briefings on Boardroom appointments and M&A news.
Choose from a cost-effective annual package or a flexible monthly subscription

The Pharma Letter is an extremely useful and valuable Life Sciences service that brings together a daily update on performance people and products. It’s part of the key information for keeping me informed

Chairman, Sanofi Aventis UK