Gamifant was tested in 2 clinical trials for FDA approval.
Gamifant was studied in 39 children and adults. These participants had active MAS with Still’s disease including sJIA that could not be controlled by steroids or with MAS that had come back.
The results of the studies supported the approval of Gamifant to be used in patients who have active MAS with Still’s disease including sJIA who experienced 1 or more of the following:
Had another MAS episode during previous treatment
The studies lasted 8 weeks.
How Gamifant performed in the trials
Researchers looked at 8 measures to see if Gamifant worked. If a patient got better in all 8, they were said to have a complete response.*
Complete response (CR): This means that all patients experienced no MAS activity after taking Gamifant. Researchers tested 8 different measures of improvement to determine this.
No MAS activity: This means that the symptoms of MAS had resolved or were under control, according to the treating doctor.
53%
of patients got better in all 8 measures
(complete response)*
To measure complete response, researchers used statistics to see if the results were really from the new medicine and not just by chance. However, the analysis wasn't able to prove that these results were due to the treatment.
According to the treating doctor.
ALT=alanine aminotransferase; LDH=lactate dehydrogenase.
These are the individual measures researchers checked and the percentage of patients who got better in each:
MAS ACTIVITY
82% MAS was resolved or inactive†
SIGNS OF INFLAMMATION
BLOOD CELL COUNTS
LIVER FUNCTION
To measure complete response, researchers used statistics to see if the results were really from the new medicine and not just by chance. However, the analysis wasn't able to prove that these results were due to the treatment.
According to the treating doctor.
ALT=alanine aminotransferase; LDH=lactate dehydrogenase.
