New Medicines

Treatment of growth hormone deficiency in children and adults


New molecular entity
Ascendis Pharma
Ascendis Pharma

Development and Regulatory status

Pre-registration (Filed)
Pre-registration (Filed)
Pre-registration (Filed)
Sep 20Filed in EU for treatment of pediatric patients who are diagnosed with GHD [6].
Jun 20Ascendis Pharma announce filing of BLA in US with orphan drug designation for TransCon hGH treatment for pediatric growth hormone deficiency [7].
Oct 19Granted orphan drug designation in EU for treatment of paediatric growth hormone deficiency. Ascendis plan to submit a NDA in EU H2 2020 [5].
Mar 19PIII program ongoing which includes heiGHht, fliGHt and enliGHten trials.[4]
Mar 19Company announced plans to submit for Biologics License Application (BLA) to the US FDA in H1 2020.[4]


Sustained-release prodrug of somatropin (human growth hormone) for weekly use - consists of a polyethylene glycol (PEG) moiety linked to somatropin via a hydrolysable linker.
Adult GH deficiency has been estimated to affect 1 in 100,000 people annually, while its incidence rate is approximately 2 cases per 100,000 population when childhood-onset GH deficiency patients are considered. About 15-20 % of the cases represent the transition of childhood GH deficiency into adulthood [1].
Treatment of growth hormone deficiency in children and adults
Subcutaneous injection

Trial or other data

Mar 19Positive results announced from PIII height trial in 161 children with pediatric growth hormone deficiency (GHD) who were randomly split in a 2:1 ratio to either receive the once-weekly ACP001 or daily Genotropin for 52 weeks. The trial met its primary objective, showing that ACP001 was non-inferior to Genotropin as well as superior to the daily hGH in terms of annualised height velocity (AHV) at 52 weeks.The AHV was higher for ACP001 than the daily hGH at each visit. At 26 weeks and after, the treatment demonstrated statistical significance. Poor responder incidence was 4% and 11% in the ACP001 and daily hGH arms, respectively. No serious side effects were observed in either arm of the trial. One serious adverse event was observed in each arm but was determined to be unrelated to the study drug.[4]
Nov 18Enrollment of PIII fliGHt open label trial (n=150, NCT03305016) completed.[2]
Aug 18Safety profile of ACP001 was found, in the PIII heiGt trial, to be consistent with the published safety profile of genotropin.[2]
Mar 18PIII heiGHt trial recruited and randomised 161 pts in the US, Italy, Greece and Poland.[2]
Dec 17PIII enliGHten long-term extension open label trial of once-weekly ACP 001 initiated in children with growth hormone deficiency (GHD) who previously participated in a PIII trial (NCT03344458). The trial wil enroll ~400 pts.[2]
Nov 17PIII fliGHt open label trial (NCT03305016) in New Zealand was initiated to assess the safety, tolerability and efficacy of ACP 001 given over 26 weeks to children aged 6 months to 17 years with growth hormone deficiency. [2]
Aug 16Ascendis Pharma initiated a 12-month, parallel PIII heiGHt trial (NCT02781727) in to investigate the safety and efficacy of a single-dose, once-weekly, s.c. injection of ACP 001 (0.24 mg/kg) in children with growth hormone deficiency vs. s.c. genotropin (34 µg/kg/daily). The primary endpoint of the trial is annualised height velocity at 52 weeks.[2]
Jul 15Data from a PII trial showed that ACP 001 released unmodified growth hormone at peak and overall exposure levels that were comparable with daily growth hormone therapies (NCT01947907).[2]
Mar 11Ascendis Pharma completed a randomised, open-label PII trial of ACP 001 (NCT01247675) which assessed the tolerability, pharmacokinetics and pharmacodynamics of three doses of once-weekly ACP 001 vs. daily growth hormone treatment over 4 weeks in 37 pts with growth hormone deficiency in Europe. The trial met all its primary and secondary endpoints which included annualised height velocity at 26 weeks.[2,3]