Amylyx ALS drug failure raises questions and concerns

Ultragenyx said Thursday afternoon that its gene therapy for a rare liver ailment, glycogen disease type 1A, succeeded in a Phase 3 trial, setting up a potential approval.

Patients with GSD1a, as the condition is often known, have a genetic mutation that prevents them from adequately maintaining blood sugar levels. Once considered fatal, GSD1a can now be controlled with regular doses of cornstarch. But if patients miss a dose, there can be significant, even life-threatening, complications.

advertisement

In the 49-person, randomized study, patients who received the Ultragenyx drug were able to take 41% less cornstarch after 48 weeks than they did at the start. Patients on placebo took only 10% less. The difference was statistically significant. Five patients were not included in the analysis, as three dropped out and the company didn’t have 48-week data from two.

STAT+ Exclusive Story

Already have an account? Log in

This article is exclusive to STAT+ subscribers

Unlock this article — plus daily coverage and analysis of the biotech sector — by subscribing to STAT+.

Already have an account? Log in

Already have an account? Log in

Monthly

$39

Totals $468 per year

Totals $468 per year

Starter

$30

for 3 months, then $39/month

Then $39/month

Annual

$399

Save 15%

Save 15%

11+ Users

Custom

Savings start at 25%!

Savings start at 25%!

2-10 Users

$300

Annually per user

$300 Annually per user

Get unlimited access to award-winning journalism and exclusive events.