Kim Moran, SVP, head of U.S. Rare Diseases at UCB, discusses Zilbrysq and the Phase 3 study that led to its approval.
Transcription:
We have the US FDA approval for ZILBRYSQ, which is based on our pivotal study, which is a Phase 3 study under the acronym of RAISE. It is a typical development study in which we evaluated ZILBRYSQ to look at its efficacy against myasthenia gravis specific efficacy endpoints and then compared it to placebo.
In this study, it’s a 12-week study. Differences between ZILBRYSQ and placebo were seen as early as one week. That’s very meaningful for patients if you think they need very quick therapy to address their symptoms. It also provides a broad patient population for the acetylcholine receptor positive population, which is the majority of generalized myasthenia gravis patients.
In the study, ZILBRYSQ was self-administered each day and continuous across that 12-week study. We were lucky in that we had the study published in May of this year in Lancet Neurology, which looked at both the safety and efficacy of the entire Phase 3 clinical trial. In the Phase 3 study, it’s a typical design, so randomized, double-blind, placebo-controlled, and looked at both the efficacy and the safety, as well as tolerability of ZILBRYSQ in adult patients with ACHR-positive myasthenia gravis.
Patients were randomized, one to one, so either received placebo for 12 weeks or a 0.3 milligram per kg daily injection of ZILBRYSQ. The study demonstrated that ZILBRYSQ delivered rapid, consistent, and statistically significant benefits in patient and clinician reported outcomes at 12 weeks, with the primary endpoint being the MGADL.
Maybe I can get into what the MGADL is. The MGADL, which is assessed both at the start of the study being baseline and at the end being at the 12-week endpoint. The ADL for myasthenia gravis goes across eight items, but it’s really important to patients. These are the things like breathing, talking, swallowing, being able to get up out of a chair, all the muscle weakness categories that we’re trying to address with these new targeted therapies.
Each item is scored, and it is this primary endpoint that delivers statistical significance. Now on the other side of things, on the safety side, the most common adverse events that were looked at in the study that were greater than 10% occurrence were injection site reactions, upper respiratory tract infections, and diarrhea.
To learn more about Myasthenia Gravis: https://checkrare.com/diseases/
