Paediatric endocrinologist welcomes first six-monthly treatment for early onset puberty

Pharma News: Australian children diagnosed with unusually early onset of puberty, central precocious puberty (CPP), now have access to Australia’s first six-­monthly CPP treatment regimen following the Pharmaceutical Benefits Scheme (PBS) listing of Diphereline (triptorelin embonate) 22.5mg.

The gonadotropin-­releasing hormone agonist (GnRHa), is now listed on the PBS for the treatment of children aged two years and older, who have been diagnosed with CPP, defined as puberty <8 years in girls and <9 years in boys. Around 800 children each year are expected to benefit with the PBS listing.

According to paediatric endocrinologist, Associate Professor Tony Huynh, Brisbane, QLD, the PBS listing of Diphereline offers a new treatment option to manage the condition and reduce the number of consultations for children.

“Ongoing prognosis and treatment of CPP require regular in-­clinic physical assessments prior to, and during treatment cycles, which can often be uncomfortable for children.

“The new PBS listing for Diphereline is a welcome treatment addition offering not only a potential improvement in treatment but also the potential to increase comfort for children with CPP through reduced injection frequency and reduced consultations,” said Dr Huynh.

“Many patients are also co-­shared with GPs who work in tandem throughout the treatment period and this may further help to assist the delicate management of these very young patients.”

While growth rates in children have been increasing and the age of first menstruation has been decreasing over the last 100 years, in recent decades the age of breast development and sperm maturity have also been shown to be decreasing. Environmental and chemical changes, including global migration, nutrition and sunlight have all been attributed to recent earlier onset of puberty.

Ipsen Head of Asia Pacific (excluding China), Peter Koetsier, said the company is delighted to have received PBS listing of Australia’s first six-month GnRHa CPP treatment, which follows swiftly on the heels of its recent Therapeutic Goods Administration (TGA) approval.

“Ipsen is proud to bring this new treatment advance to children in Australia and we are very pleased to have received a PBS listing to help to support the improvement of their lives and health outcomes.

“CPP is a condition that can cause physical, emotional, behavioural and social problems and the PBS listing of Diphereline 22.5mg for CPP may help avoid these outcomes. This new availability means children can potentially receive treatment with only two injections per year for the first time,” Koetsier added.

GnRHa treatment aims to delay puberty to synchronise with age-­related development. Advanced bone maturation is also a key feature of CPP and slowing this premature maturation is an ultimate treatment goal of GnRHa’s, given the risk of shorter adult height from premature growth plate fusion.

The PBS listing of Diphereline 22.5mg six-­monthly for CPP is based on a non-­comparative Phase III study involving 39 treatment naïve girls aged 2-­8 years and five treatment naïve boys aged 2-­9 years with at least one year bone age advancement over chronological age. It is the first study to assess
suppression of the pituitary-­gonadal axis with a triptorelin 22.5mg six-­month formulation, when given as consecutive injections six months apart. Study outcomes include an achievement of pre-­pubertal LH levels at each time point during treatment in 93% of children, increasing to 97.7% at 12 months. Oestradiol concentrations were suppressed in 79.5-­92.3% of girls between months 1-12 and testosterone levels were suppressed in 80-­100% of boys between months 1-12.

Diphereline was listed on the Australian Register of Therapeutic Goods (ARTG) on September 1 for children with CPP and the PBS listing comes within 61 days of TGA registration, demonstrating a commitment to the treatment of rare diseases in Australia.