Eleven medicines recommended for approval, including four orphans
The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended eleven medicines for approval at its April meeting.
The CHMP recommended granting marketing authorisations for two orphan medicines to treat rare neurodegenerative conditions in children: Spinraza (nusinersen) to treat patients with spinal muscular atrophy (SMA) and Brineura (cerliponase alfa) to treat neuronal ceroid lipofuscinosis type 2 (CLN2) disease. Both medicines were reviewed under EMA’s accelerated assessment programme. For more information on these medicines, please see the press releases in the grid below.
Besponsa (inotuzumab ozogamicin) received a positive opinion from the Committee for the treatment of acute lymphoblastic leukaemia. Besponsa has an orphan designation.
The CHMP granted a positive opinion for Kevzara (sarilumab) for the treatment of rheumatoid arthritis.
Skilarence (dimethyl fumarate) received a positive opinion from the Committee for the treatment of psoriasis.
One hybrid application, Cuprior (trientine tetrahydrochloride), received a positive opinion for the treatment of Wilson’s disease, a rare autosomal recessive inherited disorder. 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. Cuprior has an orphan designation.
Three biosimilar medicines 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. A biosimilar medicine is a biological medicine that is highly similar to another biological medicine that is already authorised for use.
Two generic medicines received a positive opinion from the CHMP: Febuxostat Mylan (febuxostat) for the prevention and treatment of hyperuricaemia and Ucedane (carglumic acid) for the treatment of hyperammonaemia due to N-acetylglutamate synthase primary deficiency.
Three recommendations on extensions of therapeutic indications
The Committee recommended extensions of indications for Avastin, Celsentri and Opdivo.