Ascendis Pharma reports positive results from heiGHt trial

26th March 2019 (Last Updated March 26th, 2019 00:00)

Ascendis Pharma has reported positive results from its pivotal Phase III heiGHt trial evaluating TransCon Growth Hormone (hGH) to treat children with pediatric growth hormone deficiency (GHD).

Ascendis Pharma has reported positive results from its pivotal Phase III heiGHt trial evaluating TransCon Growth Hormone (hGH) to treat children with pediatric growth hormone deficiency (GHD).

It was found that hGH, when administered once-weekly to children with GHD, had comparable safety and tolerability to daily Genotropin, with an increase in annualised height velocity over the one-year study period.

The heiGHt trial evaluated 161 treatment-naïve children with GHD randomised in a 2:1 ratio to receive either once-weekly TransCon hGH or daily Genotropin for a period of 52 weeks.

Top-line results showed that once-weekly TransCon hGH was superior to once-daily hGH on the primary endpoint of annualised height velocity (AHV) at 52 weeks.

"We are grateful for Ascendis’ commitment to developing this new treatment option for pediatric GHD and making it a reality for patients."

MAGIC Foundation CEO and co-founder Mary Andrews said: “Children have a short time to grow and a lifetime to live, which is why it is so important to help those with GHD have the best chance possible of growing up to achieve normal adult height and experience both good physical and mental health given the substantial psychosocial impact of the disease.

“We are grateful for Ascendis’ commitment to developing this new treatment option for pediatric GHD and making it a reality for patients and their families in the coming years.”

No serious adverse events related to study drug and no treatment-emergent adverse events leading to discontinuation of study drug were observed in either arm in the trial.

The serious orphan disease GHD is characterised by short stature and metabolic abnormalities that affect overall physical and mental health.

The pituitary gland in GHD does not produce sufficient growth hormone, which is important for height as well as for the development of optimal bone, heart, muscle and brain.

Last month, the company submitted an investigational new drug (IND) application for approval from the US Food and Drug Administration (FDA) to conduct a Phase II clinical trial of TransCon PTH to treat hypoparathyroidism (HP).