Enzyme Replacement Therapy Deal Benchmarks

Enzyme replacement therapies remain the backbone of lysosomal storage disorder treatment, generating $8B+ annually across Fabry, Gaucher, Pompe, and MPS indications. Phase 2 ERT deals carry a median total deal value of $3.1B, with upfront payments of $573M. The recurring revenue model (lifelong treatment at $200K-$500K per patient annually) and orphan drug protections drive strong licensing economics.

ERT deal structures reflect the predictable revenue model of chronic therapy. Development milestones average $565M, with regulatory milestones of $642M and commercial milestones of $1.4B. The 18/82 upfront/milestone split balances clinical risk with the established commercial precedent in the enzyme replacement space.

Royalty rates for ERT licensing range from 7.8% to 15.6%, reflecting the high per-patient revenue and limited biosimilar competition. Next-generation ERTs with improved half-life, tissue penetration, or oral bioavailability command premium terms. The competitive threat from gene therapy approaches is increasingly factored into ERT deal valuations, with licensees seeking assets that offer differentiated patient convenience or efficacy advantages.