Gene Therapy for Muscular Atrophy
Gene therapies offer the chance to treat genetic disorders. The Paul-Ehrlich-Institut supports the development of urgently needed gene therapies towards marketing authorisation – and thus brings them to patients.
It became known as “the world’s most expensive drug”: since May 2020, the gene therapy medicinal product Onasemnogene abeparvovec (Zolgensma) has been authorised for the treatment of certain forms of spinal muscular atrophy. One in 10,000 children is affected by this genetically caused muscle wasting which, if left untreated, can have a fatal outcome during childhood.
In February 2020, the pharmaceutical company Novartis made this medicinal product, which had already been authorised in the USA, available in advance to 100 affected children worldwide. The Paul-Ehrlich-Institut approved a compassionate use programme for Zolgensma for the benefit of affected children in Germany. A compassionate use programme is applicable prior to marketing authorisation for serious or life-threatening diseases for which there is no suitable authorised therapy.
Through our involvement in the CAT, we contribute the specific expertise of the Paul-Ehrlich-Institut towards fast and safe marketing authorisation – for the benefit of patients in need of innovative treatments.Dr Martina Schüßler-Lenz , Chair of the Committee for Advanced Therapies (CAT)
In the meantime, the marketing authorisation procedure was being conducted at the European Medicines Agency (EMA). The Committee for Advanced Therapies (CAT) chaired by Dr Martina Schüßler-Lenz assessed this Advanced Therapy Medicinal Product (ATMP). Dr Egbert Flory, acting as co-rapporteur for the CAT, led the assessment by the experts of the Paul-Ehrlich-Institut. On 27 March 2020, the Committee for Medicinal Products for Human Use (CHMP) recommended the authorisation of the gene therapy. On 18 May 2020, the European Commission granted this marketing authorisation.
Background: Viral vectors for survival
Viral vectors deliver DNA coding for the human survival motor neuron (SMN) protein into the motor neurons in the spinal cord, thus extending the survival of the motor neurons. Motor neurons transmit impulses from the brain to numerous muscles throughout the body. If these impulses are absent, the muscles cannot develop correctly. The vectors are genetically engineered vectors derived from the adeno-associated virus (AAV). Zolgensma is administered once intravenously and the results have been encouraging.
- Dr Martina Schüßler-Lenz (Chair), Dr Jan Müller-Berghaus, Dr Egbert Flory; Committee for Advanced Therapies (CAT) – EMA
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