• The researchers who discovered and developed the spinal muscular atrophy (SMA) treatment Evrysdi (risdiplam) have received two awards for their efforts.
• Evrysdi is a first-in-class oral treatment for SMA.

The researchers who discovered Evrysdi, the first oral treatment for SMA, have been honored with two prestigious awards for their efforts. Evrysdi was given the 2021 Drug Discovery Prize by the Society for Medicines Research (SMR) and the British Pharmacological Society’s 2022 Drug Discovery of the Year award.

Approved by the U.S. Food and Drug Administration (FDA) in 2020, Evrysdi was discovered at PTC Therapeutics, and then continued through development and testing with Roche, Genentech, and the SMA Foundation. The drug is approved in more than 60 counries worldwide. It’s currently indicated by the FDA for treating SMA in adults, children, and babies two months and older. In January, the agency announced it was giving priority review to an application to treat pre-symptomatic babies under two months of age.

“We are honored to have Evrysdi recognized by these awards. More importantly, we are most thrilled by the transformative impact this oral therapy has had on people living with SMA,” said Dr. Stuart W. Peltz, CEO of PTC Therapeutics, in a press release.

How Does Evrysdi Work?

SMA is caused by defects in the genes for survival motor neuron 1 and 2 (SMN1 and SMN2). These defects mean that the body cannot make the SMN1 and SMN2 proteins properly, which are required for motor neuron function. Without these proteins, motor neurons become weak and waste away.

In order for the cells to make proteins, they must receive instructions copied from the DNA on how to make them. These instructions are known as messenger RNA (mRNA) molecules. In SMA, the instructions from mRNA are not formed properly, so they cannot make the SMN protein.

Evrysdi was developed to help modify the mRNA molecules so that they can send instructions to make SMN proteins, thereby treating SMA. Evrysdi is technically known as a small molecule mRNA splicing modifier. It’s the first medication of its kind to be developed through a clinic.

The drug has proven beneficial in clinical trials:

• In one clinical trial, 41 percent of infants with SMA treated with Evrysidi over 12 months were able to sit without support for more than 5 seconds.
• In a second trial, people with SMA treated with the drug over 12 months, on average, improved muscle function. Those who took a placebo (“sugar pill”) continued to experience muscle deterioration.
• A third trial found that 85 percent of infants with SMA who’d been treated with the drug were able to survive without permanent ventilation, compared to 42 percent who were not treated with the drug.

Also notably, Evrysdi is an oral medication that is taken at home — this is different from other SMA treatments such as nusinersen (Spinraza) or onasemnogene abeparvovec-xioi (Zolgensma), which need to be injected into the spinal cord in a clinical setting.

Awards for Evrysdi

The Society for Medicines Research awards its Research Drug Discovery prize every three years to a single approved treatment with a novel approach to medicine. The society recognizes outstanding contributions and achievements to the world of drug discovery and development.

The British Pharmacological Society awarded Evrysdi out of 150 other medicines because it is a first-in-class treatment for SMA that can be given at home. “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,” said Steve Rees, elected trustee from the British Pharmacological Society, in a press release.