The European Medicines Agency (EMA), SMA Europe, and TREAT-NMD network will lead a one-day workshop to discuss, support, and advance development of new therapies to treat spinal muscular atrophy (SMA), a neuromuscular disease.
The “Spinal Muscular Atrophy Workshop” will be held 9 a.m. – 7 p.m. at EMA’s headquarters in London on Nov. 11.
SMA Europe and TREAT-NMD are non-profit organizations that bring together patients and academics from Europe and other parts of the world to advance SMA research and facilitate the development of new therapies.
The day-long event for stakeholders, including patients, medical professionals, industry representatives, researchers, and policy makers, will feature discussions about new drug possibilities and provide updates on the latest scientific developments in SMA research.
Discussion topics will include an overview of the disease, the pharmacology of the molecules under investigation, natural history data, clinical outcome measures, and potential uses of biomarkers in drug development.
Recently, EMA validated a marketing authorization application (MAA) for nusinersen, an investigational drug for SMA. Nusinersen is a novel potential disease-modifying therapy that works as an antisense oligonucleotide (ASO). Nusinersen was created to alter the splicing of SMN2, a gene that is nearly identical to SMN1, to increase production of fully functional SMN protein.
ASOs are short synthetic strings of nucleotides designed to selectively bind to target ribonucleic acid (RNA) and regulate gene expression. With the technology, nusinersen can potentially increase the amount of functional SMN protein in children and infants with SMA.
If nusinersen is approved, it will become the first therapy for SMA. EMA’s Committee for Medicinal Products for Human Use (CHMP), which previously granted nusinersen accelerated assessment status, has conditionally accepted the market name ‘Spinraza’ for the new drug.
With the FDA’s priority review and CHMP’s assessment status, accelerated regulatory review for nusinersen is now on track in the U.S. and the European Union.