With no Food and Drug Administration-approved therapies, achondroplasia has several experimental candidates going through clinical testing. This deal announced Tuesday will intensify the race to an approved treatment.
It appears Pfizer saw something they liked in the Phase 1 data enough to bring the compound into its own rare disease labs.
“Pfizer’s existing research programs for pediatric growth disorders provide a complementary setting for this potential breakthrough therapy,” said Seng Cheng, chief scientific officer of Pfizer’s rare disease unit, in a May 8 statement.
BioMarin is farthest along in development for an achondroplasia treatment with vosoritide. That biotech expects to read out Phase 3 results by year’s end.
The 52-week treatment arm for that study has completed enrollment, BioMarin CEO Jean-Jacques Bienaime said on last month’s first quarter earnings call. He estimated an addressable market for the therapy of about 25,000 children with achondroplasia.
Ascendis also has a potentially competing therapy, dubbed TransCon CNP, expected to begin Phase 2 testing in the third quarter of this year.
The rare genetic disorder is marked by problems in converting cartilage to bone. This is particularly challenging for longer bones, such as the arms and legs. The problem stems from mutations to the FGFR3 gene, which guides proteins involved in building and maintaining bone and brain tissue, according to the National Institutes of Health.
Achondroplasia tends to carry a range of potential health complications, including breathing difficulties, obesity and recurrent ear infections.
For Therachon, the Pfizer buyout bookends its five-year history. Luca Santarelli has led the company as CEO, leaving Roche after a dozen years to co-found the biotech.
“With its rare disease expertise and worldwide reach, Pfizer is well positioned to accelerate the development of TA-46 and fulfill Therachon’s vision of addressing the complications suffered by children with achondroplasia by targeting the molecular root causes of this condition,” Santarelli said in a statement.
