Nine new medicines recommended for approval by EMA/CHMP, including four orphans

The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended a total of nine new medicines for approval at its April meeting.

The CHMP recommended granting marketing authorisations for two orphan medicines to treat rare neurodegenerative conditions in children. The first, US biotech firm Biogen’s (Nasdaq: BIIB) Spinraza (nusinersen) is to treat patients with spinal muscular atrophy (SMA) It was under an accelerated assessment program. There is currently no approved therapy in the EU for the treatment of spinal muscular atrophy.

The second, from BioMarin Pharmaceuticals (Nasdaq: BMRN), is Brineura (cerliponase alfa) to treat neuronal ceroid lipofuscinosis type 2 (CLN2) disease. This was also reviewed under EMA’s accelerated assessment program. There are currently no medicines approved for treatment of CLN2 disease. Current options only treat the symptoms of the disease, a form of Batten disease, which is also known as tripeptidyl peptidase 1 (TPP1) deficiency.