TransCon HGH Gets Orphan Designation in Europe for Treatment of Pediatric Growth Hormone Deficiency

A leading biopharmaceutical company Ascendis Pharma has been granted Orphan Designation by the European Commission (EC) for its TransCon hGH, which showed great results in the Phase 3 trial made public several months ago.

Ascendis Pharma

TransCon hGH promises to be a more convenient alternative for the treatment of pediatric growth hormone deficiency (PGHD). The aim of the therapy is to reduce the frequency of treatment for patients to once a week, from daily. It employs a regulated release mechanism to enable it to work for a longer duration.

The latest Orphan Designation suggests that TransCon hGH is a step closer to becoming available.

“This designation from the EC for TransCon Growth Hormone acknowledges the need in Europe for a long-acting therapy,” Ascendis Pharma CEO Jan Mikkelsen said.

Therapies are usually granted Orphan Designation when they are intended for the diagnosis, prevention, or treatment of rare and life-threatening or chronic disorders. Such medical conditions are the kinds that affect less than five people per 10,000.

For therapy to qualify for Orphan Designation, no suitable treatment must exist for the condition it is targeted at. It must also be proven to be of considerable help to patients that will be using it.

Superior results

Ascendis Pharma made TransCon hGH to release unmodified growth hormone in a sustained manner over a week period. The type of growth hormone used is the same as that present in the daily injections that patients receive.

In addition to its main constituent, the therapy features an inert carrier and a linker. The carrier shields the unmodified growth hormone, while the linker binds the two together. After injecting, the body’s temperature and physiologic PH set off the release of the active component in a coordinated fashion.

Orphan Designation for TransCon, an abbreviated form for “transient conjugation,” came after impressive results from a Phase 3 trial. Its use was found to compare very well to daily HGH injections.

In the 52-week trial, 161 children with growth hormone deficiency received either once-weekly injections of TransCon or daily injections of Genotropin. The Ascendis Pharma product showed similar, non-substandard results as the popular daily HGH. was even better in terms of annualized height velocity (AHV).

In addition to the impressive effects, the height Trial showed that the therapy was safe and well-tolerated.

Future plans

After receiving Orphan Designation in Europe for TransCon, Ascendis will proceed to file a Biologics License Application (BLS) in the United States. It plans to do this in the first half of next year.

The biopharma company will also make a marketing application (MAA) for its once-weekly therapy in Europe in the second half of 2020.

“This is an important step towards providing a new treatment option for children with GHD, reflecting our commitment to developing therapies globally that address unmet patient needs and make a meaningful difference in patients’ lives,” Mikkelsen said.

After receiving marketing authorization, Ascendis will be able to solely market TransCon for 10 years. Market exclusivity may also be extended for another two years after that initial period.