• Source : Press Release
  • Date : 2020-11-03
  • Event type : Approved
  • Companies : Roche

Regulator OKs Roche's Oral Treatment for Spinal Muscular Atrophy

Roche Korea said Monday that the Ministry of Food and Drug Safety has approved Evrysdi (Ingredient: Risdiplam), the world's first oral drug for treating spinal muscular atrophy (SMA).

The Ministry of Food and Drug Safety approved Roche Korea's spinal muscular atrophy, Evrysdi. (Roche Korea)

The ministry gave its nod based on Roche Korea's results from various clinical trials, such as Firefish and Sunfish, involving SMA patients in a wide range of ages and types, the company said.

According to Roche Korea, FIREFISH Part 2 clinical results showed that after 12 months of Evrysdi treatment, 29 percent of patients met the criteria for sitting without help in the Bailey Infant Development Test (BSID-III). Also, 93 percent of the patients survived, and 85 percent could live without a permanent breathing device.

In contrast, untreated infancy type 1 patients were unable to stand without help. After 14 months of age, the survival rate without a permanent respiratory device was expected to be only 25 percent.

For Sunfish Part 2 clinical trials, in the Evrysdi-treated group at 12 months of treatment, the Motor Function Measure - 32 items (MFM-32) score improved by an average of 1.55 points compared to the placebo group, showing a significant effect.

MFM-32 is an evaluation criterion that includes all movements necessary for survival, such as breathing and swallowing, the company said.

Also, as a result of comparing the total score of the Revised Upper Limb Module (RULM) from the baseline 12 months after treatment, the company observed a difference of 1.59 points compared to placebo, while confirming a clinically significant improvement in the patient's symptoms.

The main drug-related adverse reactions for both trials were diarrhea and rash. However, the side effects wore out when the company continued treatment.

“We are pleased to be able to provide treatment options to a wider range of patients in the field of rare diseases where drug development is difficult,” Roche Korea CEO Nic Horridge said. “Starting with the approval of Evrysdi, Roche will continue to supply innovative medicines in the field of neuroscience in the future to contribute to the improvement of public health.”

SMA is a rare hereditary neuromuscular disease in which the whole body's muscles gradually weaken due to the loss of motor nerves due to the deficiency of the survival motor neuron (SMN) protein essential for motor function.

The first symptoms appear in infancy, but there are cases in adulthood, and various muscles, including motor function, gradually degenerate. Since these symptoms can affect the whole body, systematic disease management, and systemic action of drugs are important.

To this end, Evrysdi acts as a mechanism to increase and maintain the concentration of SMN protein by binding to the immature messenger RNA (pre-mRNA) of the SMN2 gene to compensate for defects caused by gene mutations.

Based on such a mechanism, Evrysdi can cross the blood-brain barrier and is distributed evenly in all parts of the body, including the central nervous system, to increases the SMN protein throughout the body.