The acquisition would add a BLA-stage, one-time AAV gene therapy to PTC’s rare disease portfolio, with a rolling US submission expected to complete in the fourth quarter of 2026.

The differing reimbursement positions highlight how health technology assessment decisions can diverge across UK nations, affecting the timing of patient access to new medicines.

The agreement gives Sentynl an exclusive pathway to commercialise the Phase III-ready rare lung disease therapy in the US while providing global manufacturing rights and funding support for late-stage development.

The transaction would give Sobi full ownership of a specialist commercialisation platform spanning seven Latin American markets, building on its $105 million investment in Pint Pharma in 2025.

The 56-week observational findings add longer-term clinical evidence for the eculizumab biosimilar in both complement-inhibitor-naive patients and those previously treated with reference eculizumab.