Efficacy and safety of garetosmab, an activin A-blocking antibody, in fibrodysplasia ossificans progressiva (OPTIMA): a randomised, double-blind, placebo-controlled, phase 3 trial

Riccardo Papa, Susan Rhee, Patricia Delai, Keqin Zhang, Ignacio Briceño Balcázar, Kavitha Rethanavelu, Toshifumi Fujiwara, Kenichi Mishima, Jacek Tabarkiewicz, Thomas Funck-Brentano, Raphaella Stander, Javier Bachiller-Corral, Kathryn M Dahir, E Marelise W Eekhoff, Matti Hero, Evelyn Kuong, Naoto Uemura, Meijian Zhou, Dinko Gonzalez Trotter, Jing Gu, Robert J Sanchez, Donal Óg O’Donovan, Aris N Economides, Robert Pordy, Boaz Hirshberg, George D Yancopoulos, Richard Keen, Patricia Delai, Keqin Zhang, Ignacio Briceño Balcázar, Erik Iván Quevedo Langenegger, Kavitha Rethanavelu, Toshifumi Fujiwara, Kenichi Mishima, Jacek Tabarkiewicz, Thomas Funck-Bretano, Raphaella Stander, Javier Bachiller-Corral, Kathryn M Dahir, E Marelise W Eekhoff, Matti Hero, Evelyn Kuong, Naoto Uemura, Richard Keen, Carla Toledo Neira, Xiaoya Zhang, Risto Lapatto, Caterina Matucci Cerinic, Masaki Matsushita, Neeltje Rosenberg, Malgorzata Szcezepanek, Lauren Murray, Verónica Garcia-Garcia, Judith Bubbear, LaToya M Hannah

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DOI: 10.1016/s0140-6736(26)01599-0
Published: 2026-10
Container: The Lancet
Publisher: Elsevier BV
Open access: unknown

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Cite this work

BibTeX

@article{allodium:10.1016/s0140-6736-26-01599-0,
  title = {Efficacy and safety of garetosmab, an activin A-blocking antibody, in fibrodysplasia ossificans progressiva (OPTIMA): a randomised, double-blind, placebo-controlled, phase 3 trial},
  author = {Riccardo Papa and Susan Rhee and Patricia Delai and Keqin Zhang and Ignacio Briceño Balcázar and Kavitha Rethanavelu and Toshifumi Fujiwara and Kenichi Mishima and Jacek Tabarkiewicz and Thomas Funck-Brentano and Raphaella Stander and Javier Bachiller-Corral and Kathryn M Dahir and E Marelise W Eekhoff and Matti Hero and Evelyn Kuong and Naoto Uemura and Meijian Zhou and Dinko Gonzalez Trotter and Jing Gu and Robert J Sanchez and Donal Óg O’Donovan and Aris N Economides and Robert Pordy and Boaz Hirshberg and George D Yancopoulos and Richard Keen and Patricia Delai and Keqin Zhang and Ignacio Briceño Balcázar and Erik Iván Quevedo Langenegger and Kavitha Rethanavelu and Toshifumi Fujiwara and Kenichi Mishima and Jacek Tabarkiewicz and Thomas Funck-Bretano and Raphaella Stander and Javier Bachiller-Corral and Kathryn M Dahir and E Marelise W Eekhoff and Matti Hero and Evelyn Kuong and Naoto Uemura and Richard Keen and Carla Toledo Neira and Xiaoya Zhang and Risto Lapatto and Caterina Matucci Cerinic and Masaki Matsushita and Neeltje Rosenberg and Malgorzata Szcezepanek and Lauren Murray and Verónica Garcia-Garcia and Judith Bubbear and LaToya M Hannah},
  year = {2026},
  journal = {The Lancet},
  doi = {10.1016/s0140-6736(26)01599-0},
  url = {https://doi.org/10.1016/s0140-6736(26)01599-0}
}

RIS

TY  - JOUR
TI  - Efficacy and safety of garetosmab, an activin A-blocking antibody, in fibrodysplasia ossificans progressiva (OPTIMA): a randomised, double-blind, placebo-controlled, phase 3 trial
AU  - Riccardo Papa
AU  - Susan Rhee
AU  - Patricia Delai
AU  - Keqin Zhang
AU  - Ignacio Briceño Balcázar
AU  - Kavitha Rethanavelu
AU  - Toshifumi Fujiwara
AU  - Kenichi Mishima
AU  - Jacek Tabarkiewicz
AU  - Thomas Funck-Brentano
AU  - Raphaella Stander
AU  - Javier Bachiller-Corral
AU  - Kathryn M Dahir
AU  - E Marelise W Eekhoff
AU  - Matti Hero
AU  - Evelyn Kuong
AU  - Naoto Uemura
AU  - Meijian Zhou
AU  - Dinko Gonzalez Trotter
AU  - Jing Gu
AU  - Robert J Sanchez
AU  - Donal Óg O’Donovan
AU  - Aris N Economides
AU  - Robert Pordy
AU  - Boaz Hirshberg
AU  - George D Yancopoulos
AU  - Richard Keen
AU  - Patricia Delai
AU  - Keqin Zhang
AU  - Ignacio Briceño Balcázar
AU  - Erik Iván Quevedo Langenegger
AU  - Kavitha Rethanavelu
AU  - Toshifumi Fujiwara
AU  - Kenichi Mishima
AU  - Jacek Tabarkiewicz
AU  - Thomas Funck-Bretano
AU  - Raphaella Stander
AU  - Javier Bachiller-Corral
AU  - Kathryn M Dahir
AU  - E Marelise W Eekhoff
AU  - Matti Hero
AU  - Evelyn Kuong
AU  - Naoto Uemura
AU  - Richard Keen
AU  - Carla Toledo Neira
AU  - Xiaoya Zhang
AU  - Risto Lapatto
AU  - Caterina Matucci Cerinic
AU  - Masaki Matsushita
AU  - Neeltje Rosenberg
AU  - Malgorzata Szcezepanek
AU  - Lauren Murray
AU  - Verónica Garcia-Garcia
AU  - Judith Bubbear
AU  - LaToya M Hannah
PY  - 2026
JO  - The Lancet
DO  - 10.1016/s0140-6736(26)01599-0
UR  - https://doi.org/10.1016/s0140-6736(26)01599-0
ER  -

APA

Papa, R., Rhee, S., Delai, P., Zhang, K., Balcázar, I. B., Rethanavelu, K., Fujiwara, T., Mishima, K., Tabarkiewicz, J., Funck-Brentano, T., Stander, R., Bachiller-Corral, J., Dahir, K. M., Eekhoff, E. M. W., Hero, M., Kuong, E., Uemura, N., Zhou, M., Trotter, D. G., Gu, J., Sanchez, R. J., O’Donovan, D. Ó., Economides, A. N., Pordy, R., Hirshberg, B., Yancopoulos, G. D., Keen, R., Delai, P., Zhang, K., Balcázar, I. B., Langenegger, E. I. Q., Rethanavelu, K., Fujiwara, T., Mishima, K., Tabarkiewicz, J., Funck-Bretano, T., Stander, R., Bachiller-Corral, J., Dahir, K. M., Eekhoff, E. M. W., Hero, M., Kuong, E., Uemura, N., Keen, R., Neira, C. T., Zhang, X., Lapatto, R., Cerinic, C. M., Matsushita, M., Rosenberg, N., Szcezepanek, M., Murray, L., Garcia-Garcia, V., Bubbear, J., & Hannah, L. M. (2026). Efficacy and safety of garetosmab, an activin A-blocking antibody, in fibrodysplasia ossificans progressiva (OPTIMA): a randomised, double-blind, placebo-controlled, phase 3 trial. The Lancet. https://doi.org/10.1016/s0140-6736(26)01599-0

Source records

crossref · retrieved 2026-11-05T20:52:18.859Z