Drug Discovery of the Year Award 2022

The winner of the British Pharmacological Society’s 2022 Drug Discovery of the Year award is the team of scientists who discovered and developed Evrysdi® (risdiplam) – an oral treatment for Spinal Muscular Atrophy (SMA), which is now approved in more than 60 countries worldwide.

Evrysdi was developed by Roche in collaboration with PTC Therapeutics and the SMA Foundation. The Society welcomes and celebrates this collaborative approach to drug discovery.

SMA is a serious, debilitating genetic condition. It often presents early in life and is the most common genetic cause of death in infants and toddlers. People living with SMA experience muscle atrophy and weakness, leading to problems with movement.

People with SMA have insufficient levels of the survival motor neuron (SMN) protein, a protein critical for motor neuron health. The function of motor neurons is essential for transmitting signals from the brain and spinal cord to the muscles, and therefore for people with SMA, the brain and spinal cord are less able to communicate effectively with the muscles.

Evrysdi was selected as the 2022 winner of the British Pharmacological Society’s Drug Discovery of the Year award because it is a First-in-Class medicine for the treatment of SMA. Evrysdi is a small molecule mRNA splicing modifier which can be administered at home and is designed to increase and sustain SMN protein throughout the central nervous system and in peripheral tissues. This is the first time that a small molecule mRNA splicing modifier has been developed successfully through the clinic. Evrysdi may pave the way for a new era of oral medicines targeting mRNA for many other diseases.

The team will present their work during Pharmacology 2022, taking place in Liverpool, UK, from 13-14 September 2022.

William Pao, Head of Roche Pharmaceutical Research and Early Development

The development story of Evrysdi, the first at home administered small molecule mRNA splicing modifier, shows what the power of innovation, collaboration and passion can do for patients. By thinking out-of-the-box, partnering with PTC Therapeutics and the SMA Foundation, and working across the entire organisation, our colleagues have been able to deliver a life-transforming medicine to SMA patients who desperately need it.

Traditional small molecule medicines act to inhibit or activate the protein target to mediate their therapeutic effects. Evrysdi is the first example of an approved small molecule medicine that targets RNA rather than protein to mediate therapeutic efficacy.

There is huge interest in the creation of medicines to target RNA, opening new avenues to treat disease and making previously undruggable targets tractable. This is an area of science that will explode in the coming years.