Chugai and BioMarin ink exclusive sublicense agreement

Japanese drug major Chugai Pharmaceutical (TYO: 4519) has concluded an exclusive sublicense agreement with US rare disease drug developer BioMarin Pharmaceutical (Nasdaq: BMRN) on the patent of Chugai's C-type natriuretic peptide (CNP) intended for treatment of achondroplasia in the regions of the USA and Japan.

CNP is an endogenous peptide discovered in Japan. It induces bone growth by inhibiting the fibroblast growth factor receptor 3, which inhibits the growth of the cartilage that controls bone growth. This peptide is expected to be beneficial for treatment of patients with achondroplasia, the intractable disease, in whom bone growth is systemically impaired, said Chugai, which is majority-owned by Swiss pharma giant Roche (ROG: SIX).

In the USA, European Union and Australia, BioMarin is currently conducting a Phase II study of a CNP derivative, vosoritide (formerly called BMN 111), in children with achondroplasia, the most common form of short limb dwarfism. The interim analysis of this study demonstrated favorable safety and efficacy of vosoritide subcutaneously administered once daily. The Phase II study added a fourth cohort and increased the dose. The company is also planning to initiate a pivotal phase III study, as well as another study in children under the age of five.