The Australian treasurer, Josh Frydenberg used his budget speech yesterday to make several announcements in relation to new pharmaceuticals to be listed in the Australian Pharmaceutical Benefits Scheme (PBS). In what has been described as a monumental step forward for the spinal muscular atrophy, SMA, community, Novartis’ Zolgensma®, has become Australia’s first gene therapy pharmaceutical to be PBS listed.
SMA is a rare inherited genetic muscle wasting disease characterised by a loss of motor neurons. It causes progressive muscle weakness and wasting and can cause paralysis and death.
This ground-breaking SMA gene therapy treatment comprises administration of an adeno-associated virus vector that delivers a functional copy of the human survival motor neuron (SMN) gene into targeted motor neuron cells. This results in improved muscle function and survival of children with SMA.
Significantly, Zolgensma® is considered to be the most expensive drug in the world. However, the PBS listing of Zolgensma® will save consumers in the order of $2.5 million per treatment.
Novartis appears to leading the way in relation to both approved gene therapy and cell therapy pharmaceuticals in Australia. For example, Novartis’ Luxturna®, a gene therapy for retinal dystrophy caused by pathological biallelic RPE65 mutations, became the first gene therapy listed in the Australian Register of Therapeutic Good in August 2020. Similar to Zolgensma®, Luxturna® uses an adeno-associated virus vector to deliver a functional copy of the gene encoding retinal pigment epithelium-specific 65 kDa protein.
Novartis is also one of the major players in relation to CAR-T cell therapy, which is a personalised cell-based immunotherapy that has revolutionised the treatment of certain cancers, in particular haematological malignancies. The first CAR-T cell therapy approved in Australia in December 2018 was Novartis’ Kymriah® for the treatment of B cell lymphoma. However, the cost of this treatment is in the order of $460,000 per patient.
The era of gene and cell therapy has arrived and holds great promise for the treatment of diseases that were not so long ago considered incurable. However, as seen for Zolgensma® and Kymriah®, the costs of these gene therapy and cell therapy treatments are expensive. Accordingly, the challenge for the immediate future will be how these treatments can made available by listing on the Government subsidised PBS.