Regulatory Affairs

FDA approves Triptodur to treat central precocious puberty

Published 03 July 2017

Arbor Pharmaceuticals and Debiopharm International announced that the US Food and Drug Administration (FDA) has approved Triptodur for the treatment of pediatric patients 2 years and older with central precocious puberty (CPP). CPP occurs when a child shows signs of puberty sooner than normal - before age 8 in girls and 9 nine in boys.

“We are excited to be bringing Triptodur, a new treatment option for children impacted by the disruptive effects of CPP, to the U.S. market,” said Ed Schutter, President and CEO of Arbor.

“Triptodur adds to our growing portfolio of approved medications that may help to improve the lives of our patients.”

Triptodur, a gonadotropin-releasing hormone (GnRH) agonist administered through intramuscular injection (IM), is the first GnRH agonist to offer once-every six-month dosing approved for the treatment of CPP in the U.S. In a phase III clinical trial, Triptodur demonstrated a return to pre-pubertal luteinizing hormone (LH) levels in 93 percent of patients, with pre-pubertal LH suppression maintained at 12 months by 98 percent of patients. The most common adverse reactions are injection site reactions and menstrual (vaginal) bleeding.

“Early puberty in a child can pose significant physical and emotional challenges throughout their life, including shorter adult stature, social, psychological and emotional effects,” said Karen Klein, M.D., Pediatric Endocrinologist, University of California San Diego and Rady Children’s Hospital. “With treatment, hormone levels in children with CPP are returned to a normal level, slowing the clinical signs of puberty until an age appropriate time.”

“We are extremely pleased to provide this pediatric population with the option of the triptorelin six-month formulation", said Eija Lundstrom, Medical Director, Debiopharm International SA. "This will allow children to benefit from an effective and well tolerated treatment with only two injections per year."

Triptorelin was developed by Debiopharm and will be manufactured in Switzerland by Debiopharm Research & Manufacturing SA. Arbor acquired exclusive U.S. commercial rights to Triptorelin 6-month for CPP in November 2015. Triptodur™ is expected to be available in the fourth quarter of 2017.

About Central Precocious Puberty (CPP)

GnRH-dependent CPP is defined by pubertal development occurring before the age of 8 years in girls and 9 years in boys. It is characterized by early pubertal changes such as breast development and start of menses in girls and increased testicular and penile growth in boys, appearance of pubic hair, as well as acceleration of growth velocity and bone maturation and tall stature during childhood, which often results in reduced adult height due to premature fusion of the growth plates.

Reliable epidemiological data on CPP worldwide is not available. The condition is a rare disease occurring in about 1 out of every 5,000 to 10,000 children.5 Central precocious puberty is more common in girls than in boys, with a female: male ratio estimated to be between 3:1 and 23:1.3

About Triptorelin

Triptorelin is an agonist analogue of the natural gonadotropin-releasing hormone (GnRH). Debiopharm has developed three sustained-release formulations (1, 3 and 6 months) of triptorelin pamoate. The 1-, 3- and 6-month formulations have been registered in numerous countries for several indications.

Triptorelin was first registered in France in 1986 and is currently marketed in more than 80 countries for various indications including CPP.

Source: Company Press Release