Pfizer to Buy Rare Disease Portfolio Pipeline for $810 Million

May 14, 2019 | M&A, Orphan disease, Pfizer, Rare Diseases

Rare Diseases

Pfizer will buy Therachon Holding, a privately held, clinical-stage biotechnology company focused on rare diseases, with assets in development for the treatment of achondroplasia and short bowel syndrome (SBS). The deal is worth up to $810 million.

Key Terms

  • $340 million upfront
  • An additional $470 million based on key milestone payments based on the development and commercialization of Therachon’s lead pipeline candidate TA-46.
  • TA-46 is used for treating achondroplasia, a genetic condition and the most common form of dwarfism

What is TA-46?

It is an investigational, soluble recombinant human fibroblast growth factor receptor 3 (FGFRS) decoy, a mechanism of action that is believed to normalize the overactive FGFR3 signaling pathways that underlie bone development abnormalities associated with achondroplasia. They are developing TA-46 as a weekly subcutaneous injection for children and adolescents living with the condition.


TA-46 has completed Phase 1 clinical trials and has received orphan drug designation from the EMA and FDA


Prior to closing, Therachon will spin-off its apraglutide development program into a separate, independent company.  A once weekly potentially best-in-class glucagon-like peptide 2 (GLP-2) analog in Phase 2 development for short bowel syndrome, Pfizer Ventures currently holds a minority stake.


Formed in 2014 in Basel, Switzerland with a research lab in Nice, France, Therachon is a global biotechnology company focused on developing medicines for rare, genetic diseases that currently have no available treatments. The company’s lead pipeline candidate, TA-46, is a novel protein therapy in development for achondroplasia, the most common form of short-limbed dwarfism.  The founders are Elvire Gouze and Luca Santarelli who spent many years at Roche.

The venture has raised $100 million, according to CrunchBase, from investors including Novo Holdings and Orbimed.


Pin It on Pinterest