Childhood dementia Tx Xenpozyme to soon land in KOR

MFDS conducts review for final approval

Received Breakthrough Therapy designation from the FDA... the only existing treatment option

The first childhood dementia treatment is expected to be commercialized in Korea soon.

 

According to industry sources, the Ministry of Food and Drug Safety is conducting the final review to approve Sanofi Genzyme’s treatment for acid sphingomyelinase deficiency (ASMD)m ‘Xenpozyme (olipudase alfa).’ Starting with Japan in March, the drug was also approved in Europe in July and by the US FDA in August and received Breakthrough Therapy designation in the countries.

 

The drug received final review in July and September in Europe and the US, respectively.

 

The efficacy of Xenpozyme, the only existing ASMD treatment, was identified through the ASCEND and ASCEND-Peds trials.

 

The ASCEND trial evaluated the efficacy and safety of Xenpozyme in 36 adult patients with ASMD type A/B or type B.

 

The patients were randomized to receive Xenpozyme or a placebo for 52 weeks (primary analysis).

 

At Week 52, Xenpozyme improved pulmonary function from baseline by 22% in the predicted diffusing capacity of carbon monoxide (DLco).

 

Compared with the 3% improvement shown in the placebo group, the difference between the two treatment arms of 19% was statistically significant.

 

Also, at Week 52, patients treated with Xenpozyme had a mean reduction in spleen volume by 39.5% compared with the 0.5% increase in the placebo group.

 

All patients that were treated with Xenpozyme showed an improvement in one or two primary endpoints.

 

The single-arm ASCEND-Peds trial studied 20 pediatric patients younger than 12 years of age with ASMD type A/B or type B.

 

The primary objective of the trial was to evaluate the safety and tolerability of Xenpozyme for 64 weeks, and the explored efficacy endpoints of progressive lung disease, spleen, and liver enlargement, and platelet count were also explored in the trial.

 

The nine patients who could take the test for DLco in the trial showed a 33% improvement in diffusing capacity after 1 year.

 

The patients also showed a mean reduction in spleen volume of 49%.

 

Meanwhile, ASMD is caused by the lack of an enzyme needed to break down a complex lipid, called sphingomyelin, which accumulates in the liver, spleen, lung, and brain.

 

Patients with ASMD experience enlarged abdomens at 3 to 6 months of birth.

 

The most severely affected patients have profound neurologic symptoms and rarely survive beyond two to three years of age.