Therapies for multiple sclerosis, spinal muscular atrophy, and rare diseases, among the new positive opinions of the EMA

The EMA's Committee for Human Medicinal Products supported in April a new series of treatments covering serious neurological diseases, rare genetic disorders, ocular pathologies, and breast cancer, in a package that also includes extensions of indication for new medicines already authorized.

Treatments for non-relapsing secondary progressive multiple sclerosis, spinal muscular atrophy, a rare hereditary disease affecting lipid metabolism, ocular conditions causing vision loss, and breast cancer are among the main files supported in April by the Committee for Human Medicinal Products of the European Medicines Agency.

In brief

CHMP issued positive opinions for five medicines targeting serious neurological diseases, rare genetic disorders, ocular pathologies, and breast cancer.

These include Cenrifki for progressive multiple sclerosis, Itvisma for spinal muscular atrophy, and Redemplo for familial chylomicronemia syndrome.

Rexatilux is a biosimilar for several ocular diseases, while Palbociclib Viatris is a generic for breast cancer.

CHMP also supported the extension of indications for new medicines already authorized, including Comirnaty, Opdivo, Skyrizi, and Venclyxto.

Two files were withdrawn, for Viokat and for a new use of Pluvicto, and all positive opinions are now awaiting the decision of the European Commission.

The package of positive opinions issued by CHMP in April covers a wide range of treatments, from medicines for severe neurological diseases and rare disorders to products that can broaden access to treatment through increased competition. At the center of the meeting were five new medicines recommended for authorization at the European Union level.

Cenrifki, with the active substance tolebrutinib, is intended for the treatment of non-relapsing secondary progressive multiple sclerosis, a condition of the brain and spinal cord in which inflammation destroys the protective sheath of the nerves and the nerves themselves. Itvisma, with the active substance onasemnogene abeparvovec, is a gene therapy for the treatment of spinal muscular atrophy 5q, described by EMA as a rare and severe hereditary disease that causes weakness and muscle atrophy.

Redemplo, with the active substance plozasiran, targets the treatment of familial chylomicronemia syndrome in adults, a rare hereditary disease that prevents the body from breaking down lipids. EMA emphasizes that this medicine offers a new therapeutic option for patients with high unmet medical needs. Both Itvisma and Redemplo have orphan drug designation.

Another two positive opinions concern the extension of access to treatment through products already known in other classes of use. Rexatilux, with the active substance ranibizumab, is a biosimilar medicine intended for the treatment of several ocular diseases that lead to vision impairment, including age-related wet macular degeneration and other retinopathies. Palbociclib Viatris, with the active substance palbociclib, is a generic medicine for the treatment of breast cancer.

Alongside these new products, CHMP supported the extension of therapeutic indications for new medicines already authorized in the European Union: Agamree, Aquipta, Crysvita, Comirnaty, Inaqovi, Opdivo, Privigen, Skyrizi, and Venclyxto. The announcement does not detail on this page all the new uses approved at the committee level, but shows that the files received favorable opinions and are following the procedural path to the European Commission.

The meeting also included two withdrawals. The initial authorization request for Viokat, developed for the treatment of hyperphagia in individuals with Prader-Willi syndrome, was withdrawn. The request for a new use of Pluvicto in the treatment of certain adult patients with metastatic castration-resistant prostate cancer was also withdrawn.

EMA also separately announced the completion of the evaluation for the extension of the use of Opdualag in advanced melanoma. Although the agency did not recommend the new use, it accepted that relevant data from the file be included in the product information, so that healthcare professionals have access to updated data regarding the effects of the medicine for a certain category of patients.

Overall, the April meeting outlines a picture in which European scientific decisions simultaneously cover highly complex therapies, such as gene therapy, treatments for rare and severe diseases, but also biosimilar and generic products that can contribute to expanding therapeutic options and broader market access.

CHMP plays the role of scientific evaluator in the European authorization procedure, and its opinions form the basis for subsequent decisions by the European Commission. In this case, the focus is not only on the procedural step but also on the profile of the evaluated medicines: treatments for progressive neurological diseases, rare genetic disorders, severe metabolic disorders, ocular diseases, and oncology.

https://2eu.brussels/ro/stiri/terapii-pentru-scleroza-multipla-atrofie-musculara-spinala-si-boli-rare-printre-noile-avize-pozitive-ale-ema