Update: Phase 3 Data Published on Ganaxalone for CDD

According to a recent article in Businesswire, The Lancet has published data from the third phase of the Marigold trial. This clinical trial evaluated ganaxalone – marketed as ZTALMY – as a treatment for seizures caused by CDKL5 deficiency disorder (CDD). Want to read the full paper? You can find it here. If not, Patient Worthy is here to give you a quick summary.

About CDD

First, let’s talk about what CDD is. This rare, genetic form of epilepsy is extremely rare, with around 1,000 cases reported throughout the world. Symptoms begin during infancy, and they are characterized by treatment-resistant seizures and severe developmental delay. Patients experience intellectual disabilities, limited or no speech, a delay in the development of gross motor skills, issues with fine motor skills, teeth grinding, speech disruptions, GI issues, repetitive hand movements, feeding difficulties, irregular breathing, and distinctive facial features. Scoliosis, microcephaly, and tapered fingers are other possible symptoms as well.

All of these symptoms are the result of a mutated CDKL5 gene, which holds the instructions for a protein needed for brain development and function. More research is necessary to gain a better understanding of this gene and its relation to the specific symptoms, but medical professionals know that it is inherited in an X-linked dominant pattern. Because of this, females account for about 90% of cases. It’s important to mention that this mutation can occur sporadically as well.

About the Phase 3 Marigold Trial

The paper, which is titled “Efficacy and safety of ganaxolone in patients with CDKL5 deficiency disorder: a randomized, double-blind, placebo-controlled, Phase 3 trial,” dives into the data from the phase 3 Marigold trial. 101 CDD patients were randomized to receive either ganaxalone or a placebo. Some highlights include:

  • The frequency of 28-day major motor seizures was reduced by a median of 30.7%
    • In the placebo group, the mean reduction was 6.9%
    • This data met the study’s primary endpoint
  • Consistent safety profile
  • Ganaxalone was well-tolerated
    • Somnolence was the most common adverse event (AE)

Not only are these date extremely positive, but they led to the FDA approval of ganaxalone for CDD in patients two years and older. Now that it’s approved, patients can expect access in July of this year. When they are able to take it, they’ll do so three times a day.

Want to read more about ganaxalone’s FDA approval? You can find a Patient Worthy article on it here!

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