EMA backs authorisation of biologics

The EMA’s Committee for Medicinal Products for Human Use (CHMP) recommended authorisation for three orphan medicines to treat rare neurodegenerative conditions in children: Biogen Idec’s Spinraza (nusinersen) to treat patients with spinal muscular atrophy (SMA) and BioMarin International’s Brineura (cerliponase alfa) to treat neuronal ceroid lipofuscinosis type 2 (CLN2) disease. Spinraza is an antisense oligonucleotide, licenced from Ionis Pharmaceuticals, that increases the expression of the SMN protein, thus improving neuronal survival. The active substance of the enzyme replacement therapy Brineura is cerliponase alfa, which works by replacing the missing enzyme tripeptidyl peptidase-1 in patients with the lysosomal storage disorder CLN2, reducing build-up of lipofuscins. Additionally, gmp-orphan SA received a positive opinion for its hybrid application to treat the rare autosomal recessively inherited Wilson’s disease with Cuprior (trientine tetrahydrochloride). Hybrid applications rely in part on the results of pre-clinical tests and clinical trials for a reference product and in part on new data. Additionally, the CHMP backed market authorisation of Pfizer’s Besponsa (inotuzumab ozogamicin) for the treatment of acute lymphoblastic leukaemia. Besponsa has also been granted EU orphan designation.

Three biosimilar medicines made by Sandoz were recommended for approval by the Committee: Erelzi (etanercept) for the treatment of rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, axial spondyloarthritis, plaque psoriasis and paediatric plaque psoriasis; and Rixathon and Riximyo, both containing rituximab, for the treatment of non-Hodgkin’s lymphoma, rheumatoid arthritis, granulomatosis with polyangiitis and microscopic polyangiitis. Rixathon is also intended for the treatment of chronic lymphocytic leukaemia.

Furthermore, the CHMP granted a positive opinion for Sanofi’s IL-6 receptor alpha blocker Kevzara (sarilumab) for the treatment of rheumatoid arthritis. Almirall S.A.’s Skilarence (dimethyl fumarate) received a positive opinion from the Committee for the treatment of psoriasis. 

An MAA for Solithromycin Triskel EU Services (solithromycin) has been withdrawn. This medicine designed for the treatment of community-acquired pneumonia, inhaled anthrax and inhaled tularaemia. The CHMP was concerned that not enough data had been provided to support use in inhaled anthrax and inhaled tularaemia. There were also concerns that solithromycin could be harmful to the liver. In addition, it had concerns about the manufacturing process of the active substance, which did not exclude the presence of impurities, and the test for ensuring sterility of the product for infusion was not considered valid.