EC approves Spinraza, Brineura

The European Commission approved a pair of new drugs for rare diseases: Spinraza nusinersen from Biogen Inc. (NASDAQ:BIIB) to treat spinal muscular atrophy (SMA); and Brineura cerliponase alfa from BioMarin Pharmaceutical Inc. (NASDAQ:BMRN) to treat neuronal ceroid lipofuscinosis type 2 (CLN2), a form of Batten disease.

Spinraza is the first SMA drug approved in the EU. It is an antisense oligonucleotide that modulates splicing of survival of motor neuron 2 centromeric (SMN2) mRNA. Biogen has rights to Spinraza from Ionis Pharmaceuticals Inc. (NASDAQ:IONS). The approval triggered a $50 million milestone payment to Ionis, which is also eligible for tiered royalties...