Eleven new medicines have taken a giant leap towards approval in Europe after winning backing from the European Medicines Agency’s Committee for Medicinal Product for Human Use (CHMP), including two that treat rare neurodegenerative conditions in children.

Biogen Idec’s Spinraza (nusinersen) has been recommended for approval to treat patients with spinal muscular atrophy (SMA), a rare and often fatal genetic disease that causes muscle weakness and progressive loss of movement.

The decision is based on clinical trial showing that 51 percent of patients responded to treatment with the drug - which is administered by lumbar puncture injection every four months - compared to none receiving the mock injection (controls).

Sixteen patients (22 percent) achieved full head control, six patients (8 percent) achieved independent sitting, and one patient (1 percent) achieved standing with support, whereas no subjects in the control group achieved any of these milestones. Also, the risk of death or permanent ventilation was 47 percent less in patients treated with the drug.

BioMarin’s Brineura (cerliponase alfa) was put forward for the treatment of a very rare, fatal neurodegenerative condition in children called neuronal ceroid lipofuscinosis type 2 (CLN2) disease, characterised by low levels of the enzyme tripeptidyl-peptidase 1 and for which there are currently no approved treatments.

The enzyme replacement therapy is expected to improve some of the symptoms experienced by the young patients; in one clinical trial, 20 of the 23 patients treated (and who could take part in the efficacy analyses) experienced either a slower than expected progression of the disease, a stabilisation of the progression of the disease or some improvement in their motor and language abilities, which was considered a significant therapeutic effect.

Elsewhere, Pfizer’s orphan drug Besponsa (inotuzumab ozogamicin) received a positive opinion from the Committee for the treatment of acute lymphoblastic leukaemia. The drug has been shown to increase the proportion of patients who have complete remission and molecular remission and to delay the progression of disease. The most common side effects are thrombocytopenia, neutropenia, anaemia, leucopenia, infection, haemorrhage and venoocclusive liver disease/sinusoidal obstruction syndrome.

The CHMP also granted a positive opinion for Sanofi-Aventis' Kevzara (sarilumab) for the treatment of rheumatoid arthritis. According to the Agency, the drug is able to reduce the signs and symptoms of the disease, improve physical function, and inhibit the progression of joint damage in patients. The most common side effects were found to be neutropenia, increased ALT, injection site erythema, upper respiratory-tract infections, and urinary tract infections.

Almirall’s Skilarence (dimethyl fumarate) was backed for the treatment of psoriasis in patients needing systemic therapy, on the back of data showing its ability to reduce signs and symptoms of the disease. The most common side effects are gastrointestinal events, flushing and lymphopaenia.

A hybrid application for GMP-Orphan’s Cuprior (trientine tetrahydrochloride) resulted in a recommendation for the treatment of Wilson’s disease, a rare autosomal recessive inherited disorder. Hybrid applications are comprised of pre-clinical and clinical data for a reference product and in part on new data. The drug can decrease serum copper levels in patients with the illness, and thus helps to prevent neurological abnormalities and hepatic dysfunction.

Three biosimilars were also backed for approval: Sandoz’ Erelzi (etanercept) for the treatment of rheumatoid arthritis, juvenile idiopathic arthritis, psoriatic arthritis, axial spondyloarthritis, plaque psoriasis and paediatric plaque psoriasis; and Sandoz’ 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.

Mylan’s generic Febuxostat Mylan (febuxostat) was backed for the prevention and treatment of hyperuricaemia, and Lucane Pharma’s generic Ucedane (carglumic acid) was given a nod for the treatment of hyperammonaemia due to N-acetylglutamate synthase primary deficiency.