Debiopharm Announces First Patient Enrolled in Phase III Study for the Rare Disease Central Precocious Puberty
Published: Aug 27, 2012
LAUSANNE, Switzerland, August 27, 2012 /PRNewswire/ --
Debiopharm Group (Debiopharm), a Swiss-based global biopharmaceutical group of companies with a focus on the development of prescription drugs that target unmet medical needs and companion diagnostics, today announced that the first patient has been enrolled in an open-label phase III study to investigate the efficacy, safety, and pharmacokinetics of the sustained release triptorelin pamoate 22.5 mg 6-month-formulation in children with central precocious puberty (CPP), a rare disease.
The multicentre, open-label, non-randomised study will involve 44 children from the United States, Chile and Mexico. Its primary objective is to evaluate the efficacy and safety of triptorelin pamoate (embonate) 22.5 mg 6-month formulation in decreasing luteinizing hormone (LH) to prepubertal levels at Month 6 (Day 169) in children with CPP.
"We are excited to be part of this study, which is the first Debiopharm-sponsored clinical trial using triptorelin for the treatment of CPP," said Rolland-Yves Mauvernay, President and founder of Debiopharm Group. "Our aim is to show that a gonadotropin-releasing hormone (GnRH) agonist therapy prevents the social and psychological difficulties associated with premature sexual development, and has a positive impact on compromised adult height. The 6-month-formulation would entail an improved compliance in a paediatric indication for which no other 6-month GnRH agonist formulation is currently approved, whilst reducing the frequency of injections in children."
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 characterised 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, which often results in reduced adult height and disproportioned body appearance due to premature fusion of the growth plates.
Reliable epidemiological data on CPP worldwide are not available. The condition occurs in about 1 out of every 5,000 to 10,000 children (leading to an estimated prevalence of 10,000 to 20,000 children in the USA). These numbers show that CPP is a rare disease. 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.
Triptorelin is a synthetic decapeptide agonist analogue that was first registered in France in 1986 and is currently marketed in more than 80 countries in various indications including advanced prostate cancer and CPP. Chronic administration of triptorelin causes down regulation of the pituitary GnRH receptors and suppresses gonadotropin (LH and FSH) secretion and finally the release of gonadal sex-hormones. Triptorelin shows a safety profile similar to other GnRH agonists. Except for injection site reactions or rare immunoallergic reactions, the side effects of triptorelin are mostly due to the initial increase in testosterone/oestrogen levels (e.g. vaginal bleeding in girls) followed by almost complete suppression of testosterone/oestrogen (e.g. hot flushes and headaches).
However, numerous published studies over more than 25 years involving large series of children ( 20 studies) around the world have reported efficacy and safety results with different triptorelin acetate and pamoate (embonate) 1- and 3-month formulations consistent with those of other approved GnRH agonists in this indication. None of these studies has revealed any safety issues.
About Debiopharm Group
Debiopharm Group (Debiopharm) is a Swiss-based global biopharmaceutical group of companies with a focus on the development of prescription drugs that target unmet medical needs. The group in-licenses, develops and/or co-develops promising biological and small molecule drug candidates having reached clinical development phases I, II or III, as well as earlier stage candidates. It develops its products for global registration and maximum commercial potential. The products are out-licensed to pharmaceutical partners for sales and marketing. Debiopharm is also active in the field of companion diagnostics with a view to progressing in the area of personalised medicine. Debiopharm independently funds the worldwide development of all of its products while providing expertise in pre-clinical and clinical trials, manufacturing, drug delivery and formulation, and regulatory affairs.
For more information on Debiopharm Group, please visit: http://www.debiopharm.com.
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SOURCE The Debiopharm Group