LIB Therapeutics launched Lerochol in the United States after FDA approval for lowering LDL-C in adults with hypercholesterolemia, including HeFH. The once-monthly PCSK9 inhibitor is available with a $199-per-month cash-pay option.
Ultragenyx reported Q4 2025 revenues of $207 million, up 25% year-over-year, while announcing a 10% workforce reduction and resubmission of its UX111 gene therapy application following positive long-term clinical data.
| NCT ID | Title | Status | Phase |
|---|---|---|---|
| NCT07037771 |
A Phase 3 Study of Zodasiran in Adolescent and Adult Subjects With Homozygous Familial Hypercholesterolemia (YOSEMITE) |
ACTIVE_NOT_RECRUITING | PHASE3 |
| NCT05217667 |
Study of ARO-ANG3 in Participants With Homozygous Familial Hypercholesterolemia (HOFH) |
TERMINATED | PHASE2 |
