Ascendis Pharma A/S's growth hormone therapy for pediatric patients with growth hormone deficiency was granted orphan designation by the European Commission.
The Danish biopharmaceutical company's TransCon Growth Hormone is in development as a once-weekly treatment for pediatric growth hormone deficiency. It provides sustained release of the growth hormone at a calculated rate over one week.
On March 4, the company disclosed results from a late-stage study showing that TransCon Growth Hormone therapy encouraged more growth in pediatric patients with growth hormone deficiency than Pfizer Inc.'s daily Genotropin medicine.
Ascendis said in an Oct. 21 news release that it plans to file for a U.S. Biologics License Application in the first half of 2020 and a marketing application in Europe in the second half of 2020 for TransCon Growth Hormone.
There are no approved long-acting growth hormone treatments in Europe currently.
The European Commission's orphan designation is granted to therapies aimed at the treatment, prevention or diagnosis of a life-threatening disease that affects no more than five in 10,000 people in the EU and for which satisfactory treatment is unavailable. Drugs with the orphan tag have 10 years of market exclusivity after they receive market authorization in the EU and the tag supports future reimbursement and access to new therapies.