EMA recommends five new medicines, including a first-in-class small interfering RNA treatment
Five new medicines are recommended for approval; a further nine medicines are recommended for extension of their therapeutic indications
The EMA’s April meeting of the Medicinal Products for Human Use Committee (CHMP) recommended five new medicines and nine existing medicines with extended therapeutic indications for approval.
The new medicines include a gene therapy for spinal muscular atrophy (SMA) - Itvisma (onasemnogene abeparvovec, Novartis), a treatment for non-relapsing secondary progressive multiple sclerosis - Cenrifki (tolebrutinib, Sanofi), and a first-in-class RNA interference (RNAi) mechanism-based medicine to help block a harmful protein produced in the liver that inhibits the breakdown of triglycerides, for the treatment of a rare inherited disease, (familial chylomicronaemia syndrome FCS) - Redemplo (plozasiran, Arrowhead Pharmaceuticals). FCS affects around 1 in 250 people across Europe.
“We are pleased with the CHMP’s recommendation, which brings us closer to potential approval of Redemplo as a new treatment option for people living with genetically or clinically confirmed FCS across Europe,” said President and CEO at Arrowhead Pharmaceuticals, Christopher Anzalone. “This milestone reflects the strength of the clinical data and the ability of our Targeted RNAi Molecule platform (TRiM™) [...] we are working with regulators and the healthcare community to bring this therapy to more patients as quickly as possible.”
“Patients with FCS face a substantial burden of disease and are at life-long risk of acute pancreatitis, with few effective treatment options available,” said Professor of Cardio-metabolic Medicine, University of Western Australia, Gerald F Watts. “[The] data highlight the potential of targeted RNA interference approaches in addressing conditions like FCS that have so far been difficult to treat. Plozasiran is a major advance in the care of FCS and a significant step toward expanding treatment options for patients across Europe.”
A biosimilar medicine - Rexatilux (ranibizumab, Intas Third Party Sales 2005 S.L.) - for the treatment of macular degeneration and risual impairment, as well as a generic - Palbociclib Viatris (palbociclib, Viatris) - for the treatment of breast cancer, were also approved.
Nine medicines approved for new indications
In addition to the new medicines, nine existing medicines were recommended for new indications. Agamree (vamorolone, Santhera Pharmaceuticals) for Duchenne muscular dystrophy (DMD) now recommended for children of 2 years and older; Aquipta (atogepant, AbbVie) for acute migraine; Comirnaty (tozinameran, BioNTech/Pfizer) the COVID-19 vaccine is extended from 5 years to infants of 6 months or older; Inaqovi (decitabine / cedazuridine, Otsuka Pharmaceutical); Crysvita (burosumab, Kyowa Kirin); Opdivo (nivolumab, Bristol Myers Squibb); Privigen (human normal immunoglobulin, CSL Behring); Skyrizi (risankizumab, AbbVie); Venclyxto (venetoclax, AbbVie).
For full results of the CHMP, click here.