Motor Neurone Disease: investigational drug granted accelerated approval

Trapped inside a body that no longer works, unable to speak, move or, eventually, breathe. People with motor neurone disease (MND) know this is how their life will end, and with no treatment or cure available, it is a terrifying fate.

Tofersen (brand name Qalsody) is an investigational drug developed by the company Biogen to treat MND caused by a mutation in the SOD1 gene and was granted accelerated conditional approval by the Food & Drug Administration (FDA) in the United States in April 2023.

The SOD1 gene mutations were discovered in 1993, and account for about 15-30% of familial and 2% of sporadic MND cases in people of European descent. In people of Asian descent, SOD1 gene mutations are the most common genetic cause of MND.

Tofersen works by targeting the SOD1 RNA to reduce the amount of SOD1 protein being made.  It is only effective in SOD1-type MND and cannot be used in other types of MND. The medication is given once a month via injection into the spinal fluid.

Biogen has announced an early access program that patients diagnosed with MND caused by a SOD1 gene mutation can apply to.

The Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) has recently recommended approval be granted for marketing under exceptional circumstances.

MND NZ is encouraged by the positive opinion of the CHMP and what this means for people with SOD1-type MND. We are in dialogue with Pharmac and international MND associations and will monitor developments to inform the best access pathway to Tofersen in New Zealand.

Read the full statement, here.