On March 27, 2020, the European Commission received a positive recommendation from the Committee for Advanced Therapies (CAT) and the Committee for Medicinal Products for Human Use (CHMP) to authorise the Onasemnogen abeparvovec (U.S. trade name: Zolgensma). The authorisation, which can subsequently be granted by the European Commission, is intended to apply to the treatment of certain forms of spinal muscular atrophy (SMA) in babies and young children. In Germany, one in 10,000 children is affected by spinal muscular atrophy, a spinal cord disease that causes muscle wasting and, in severe cases without treatment, leads to death in the first years of life. The approval of the current compassionate use progamme in Germany expires in the event of a positive authorisation decision by the European Commission and the provision of Onasemnogen abeparvovec.
The application for EU approval via the centralised procedure was evaluated in CAT and CHMP at the European Medicines Agency (EMA). The EMA's recommendation for authorisation is based on the preliminary results of an ongoing clinical trial and a clinical development programme, including three supportive studies in patients with SMA at different stages of the disease. This also included genetically diagnosed and presymptomatic patients.
Experts from the Paul-Ehrlich-Institut, Federal Institute for Vaccines and Biomedicines, have led the process in the function of the co-rapporteur in the CAT at the EMA. CAT and CHMP have proposed to the European Commission a conditional approval, according to which the marketing authorisation holder has to meet a number of additional conditions and take measures after the authorisation. This is to ensure that the safety and effectiveness of the gene therapy medicine is observed in everyday use.
The scientific recommendation of both committees is now being forwarded to the European Commission for approval of a legally binding marketing authorisation.
In the event of a positive authorisation decision by the European Commission and the provision of the gene therapy for the treatment, the conditional approval programme in Germany, implicitly approved by the Paul-Ehrlich-Institut on 3 February 2020, will no longer apply. Parents of affected children can contact the treating doctor or health insurance regarding treatment and cost coverage.
Onasemnogen abeparvovec is a gene therapy medicine that mediates the expression of human survival motoneuron (SMN) protein in affected body cells. It is a non-reproducible, genetically engineered vector (gene ferry), derived from serotype 9 of the adeno-associated virus (AAV). The AAV vector introduces the building instructions for the SMN protein in the form of a cDNA into human body cells. The treatment included in the authorisation is intended for patients with spinal muscle atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA type 1 or patients with SMA with a bi-allelic mutation in the SMN1 gene and up benefit from three copies of the SMN2 gene. The Treatment in the form of a single intravenous infusion is to be monitored in selected clinical centers by a doctor who has gained experience in the treatment of patients with SMA.
Updated: 27.03.2020
