This web site uses cookies. Do you accept the use of external cookies? Yes No Know more x
Survey about achondroplasia
We have designed a questionnaire with the purpose to better know the population connected to achondroplasia.
The questionnaire is optional and anonymous. The data obtained will be used for statistical purposes and to better understand the natural history of achondroplasia. Please consider answering.
If you would like to take the survey later, you can click on the link at the top "Register at BA"

15th Dec 2020

Press statement

Pfizer Doses First Participants as Part of Global Achondroplasia Phase 2 Development Program

Pfizer announced that the first participants were dosed in the global Phase 2 multiple-dose, randomized study to assesses the safety, tolerability, pharmacokinetics, and efficacy of Recifercept in children with achondroplasia. Participants received a subcutaneous injection of the biologic Recifercept at Antwerp University Hospital, Belgium, under the care of Professor Geert Mortier, and Hospital Vithas San José, Spain under Dr. Josep De Bergua. 1

“With our clinical program underway, starting simultaneously in Belgium and Spain, we look forward to advancing a potential novel differentiated medicine for children with achondroplasia who need it the most,” said Seng Cheng, Senior Vice President and Chief Scientific Officer of Pfizer’s Rare Disease Research Unit. “The initiation of the Phase 2 trial is a major step forward in our progress toward providing a potential therapy to address the varied functional, respiratory, and neurological conditions associated with achondroplasia. We look forward to generating additional data as the program progresses.” This Phase 2 study will enroll up to 63 children between 3 months and 11 years old with achondroplasia. Potential participants for this Phase 2 study will come from a subset of sites in an ongoing natural history study led by Pfizer, which seeks to understand the clinical and anthropometric characteristics of children with achondroplasia (; NCT03794609).
In addition to evaluating safety and tolerability, the phase 2 study will evaluate measurements of the increase in height (growth above expected in reference population), assessments of the potential impact on achondroplasia-related complications, and changes in health-related quality of life. 1

Participants will receive the study drug for 12 months. More information about the trial and participating sites may be found here ( NCT04638153). Further sites (hospitals/clinic research centers) in addition to Antwerp Hospital and Hospital Vithas San José will soon begin enrolling in Europe, North America, and Australia. 1

As one of the first centers for the phase 2 clinical trial is in Spain, a national organization was invited to give a statement “We are pleased to partner with Pfizer to move this clinical study forward,” said Carmen Alonso, Director, Fundación ALPE Achondroplasia. “Children with achondroplasia lack options to address medical complications associated with their condition, and as a result, this research has the potential to be lifechanging.” 1

What is Pharmacokinetics?
Is the study of "what the body does to the drug". It includes:
•  the rate and extent to which drugs are absorbed into the body and distributed to the body tissues;
•  the rate and pathways by which drugs are eliminated from the body by metabolism and excretion;
•  the relationship between time and plasma drug concentration

1. Pfizer  
To view this site you need Internet Explorer 8 (or a higher version)
or another internet browser.

Thank You.