BioMarin Pharmaceutical (NASDAQ: BMRN) Disclosed Wednesday August 26, 2026 Additional Detail on the Previously-Announced Alesta Therapeutics Acquisition (Definitive Agreement Announced August 21): The Transaction Value Is $275 Million and Alesta's Lead Asset ALE1 Is an Oral Hypophosphatasia (HPP) Therapy That Would Challenge the Currently-Approved AstraZeneca / Alexion Enzyme Replacement Strensiq (Asfotase Alfa, a Recombinant Tissue-Nonspecific Alkaline Phosphatase-Fc Fusion Protein Administered as Subcutaneous Injection) With a Smaller-Molecule Oral Format That Could Expand Access to Adult HPP Patients Where the Injectable Enzyme Replacement Has Been Less-Broadly Reimbursed; The Acquisition Continues BioMarin's Rare-Disease Portfolio Expansion Following the Amicus Integration Alongside Voxzogo (Vosoritide, C-Type Natriuretic Peptide Analog for Achondroplasia)
BioMarin Pharmaceutical (NASDAQ: BMRN) disclosed Wednesday August 26, 2026 additional detail on the previously-announced Alesta Therapeutics acquisition (definitive agreement announced August 21). Transaction value: $275 million. Alesta's lead asset ALE1: an oral hypophosphatasia (HPP) therapy in clinical development. Hypophosphatasia is a rare inherited metabolic disease caused by loss-of-function mutations in the ALPL gene encoding tissue-nonspecific alkaline phosphatase (TNSALP), an enzyme required for bone mineralization and other biological processes; without adequate TNSALP activity, patients develop rickets, osteomalacia, seizures (in the severe perinatal form), and other complications. Commercial context: the currently-approved treatment is AstraZeneca / Alexion's Strensiq (asfotase alfa), a recombinant TNSALP-Fc fusion protein administered as three-times-weekly subcutaneous injection at a list price of approximately $1.5-2.0 million per adult patient per year. ALE1 would compete as a smaller-molecule oral format that could expand access to adult HPP patients where the injectable enzyme replacement has been less-broadly reimbursed by payers due to cost and administration burden concerns. The acquisition continues BioMarin's rare-disease portfolio expansion following the Amicus Therapeutics integration earlier in 2026 (which added GALAFOLD for Fabry disease and POMBILITI + OPFOLDA for late-onset Pompe disease) alongside Voxzogo (vosoritide, C-type natriuretic peptide analog for achondroplasia, on track for $1+ billion annual sales in 2026) and the broader enzyme-replacement franchise.