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Recifercept is the most advanced rare disease candidate to become discontinued. The molecule is designed to bind to fibroblast growth factor isoforms and thereby act as a decoy that reduces FGFR3 signaling. Seeing that FGFR3 mutations drive inhibitory signaling in the rare disease achondroplasia, Pfizer made the condition its focus indication for recifercept development. Last week, Pfizer decided to discontinue development of Recifercept for the treatment of achondroplasia. 

Read the press release:

"Pfizer shared its decision to discontinue the development of recifercept for the treatment of achondroplasia. The decision comes following completion of a planned interim analysis of its Phase II clinical study of recifercept, which showed the study would not meet its primary endpoint. It is important to note that the study was not discontinued for safety reasons. In addition, the achondroplasia natural history study will be terminated. For more information, please visit


We anticipate that this news will deeply disappoint the many families living with achondroplasia who directly participated in the studies, as well as all of the families living with achondroplasia hoping for therapies that will be impactful. In due course, Pfizer intends to share data from the phase 2 clinical study with the scientific community in order to contribute to the body of knowledge, which may help the development of new therapies for patients. The achondroplasia community has provided an unwavering commitment to these studies and to Pfizer’s research and development efforts. We thank the patients, families, advocacy organizations, and investigators that participated in this trial, without whom this meaningful research would not have been possible."

Katherine Beaverson, MS
Exc Director; Head of Patient Advocacy and External Engagement
Pfizer Worldwide Research Development and Medical
Rare Disease Research Unit

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