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.
US pharma giant Pfizer’s (NYSE: PFE) Besponsa (inotuzumab ozogamicin) received a positive opinion from the Committee for the treatment of acute lymphoblastic leukemia. Besponsa has an orphan designation. If approved, Besponsawill be the first antibody drug conjugate available for patients with this type of leukemia. Around 10,000 new adult cases of ALL are diagnosed in Europe each year, and there is an urgent unmet need for patients with relapsed or refractory adult ALL.
The CHMP granted a positive opinion for French pharma major Sanofi’s (Euronext: SAN) Kevzara (sarilumab) for the treatment of rheumatoid arthritis. Kevzara has been shown to inhibit the progression of joint damage in patients with rheumatoid arthritis.
Skilarence (dimethyl fumarate), from Spain’s largest drugmaker Almirall (ALM: MC) received a positive opinion from the Committee. It is a first-line induction and long-term treatment for adults with moderate-to-severe chronic plaque psoriasis.
One hybrid application from GMP-Orphan, 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 biosimilars cleared for marketing
Three biosimilar medicines from Sandoz, a subsidiary of Swiss pharma giant Novartis (NOVN: VX), 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 pediatric 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 leukemia. A biosimilar medicine is a biological medicine that is highly similar to another biological medicine that is already authorized for use.
The CHMP’s positive opinions are now referred to the European Commission, which grants the final marketing authorization for centrally authorized medicines in the EU. A decision from the EC is expected in the next two months or so.