Ascendis Pharma Presents Results from Long-Term enliGHten Trial of TransCon™ hGH in Pediatric Growth Hormone Deficiency
- The majority of children and adolescents treated once weekly with TransCon hGH (lonapegsomatropin) met or exceeded average parental height SDS at time of treatment completion or last visit
- Trial demonstrated the long-term safety of TransCon hGH in patients treated up to 6 years
The enliGHten trial enrolled 298 participants (mean age 10.3 years) from the Phase 3 heiGHt Trial of treatment-naïve pediatric GHD patients and the Phase 3 fliGHt Trial of pediatric GHD patients switching from daily somatropin treatment. Patients in these trials received a total of up to 6 years of treatment with TransCon hGH.
At the time of the enliGHten Trial conclusion, 81 participants were designated as treatment completers, based on their physician’s determination that treatment for pediatric GHD was no longer required. Of these treatment completers, 59% met or exceeded their average parental height SDS, with mean TransCon hGH treatment duration of 3.2 years. Treatment completers’ baseline mean height standard deviation score (SDS) at the beginning of the open-label extension trial was -1.6, compared to mean height SDS of -0.4 (achieving height similar to their parents’) at their final study visit. At the time of final visit, all treatment completers were Tanner stage IV or V, a categorization of physical development during puberty.
“As an investigator in the enliGHten Trial, I am pleased to see results confirming that treated children and adolescents have continued to grow well, achieving statures in line with those of their parents,” said
TransCon hGH was generally safe and well-tolerated. The most commonly reported adverse events over the course of the trial were categorized as infections, injury, and respiratory/thoracic/medical disorders. The majority of adverse events were mild in severity and unrelated to treatment. No adverse events led to discontinuation of the study treatment.
About TransCon hGH (Lonapegsomatropin)
TransCon hGH (lonapegsomatropin) is a prodrug of somatropin administered once weekly, designed to provide sustained release of active, unmodified somatropin. The unmodified, unbound somatropin released from lonapegsomatropin has the identical 191 amino-acid sequence and size (22 kDa) as endogenous growth hormone. TransCon hGH is approved and marketed as SKYTROFA® (lonapegsomatropin-tcgd) in
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Source: Ascendis Pharma