Roche's Evrysdi is about to be approved for listing in the EU and has been under review in China

Roche recently announced that the European Medicines Agency (EMA) Committee for Medicinal Products for Human Use (CHMP) has issued a positive review suggesting that Evrysdi (risdiplam) should be approved for the treatment of age ≥ 2 months and a clinical diagnosis of 1 Type 2 type/3 SMA or type 5q spinal muscular atrophy (SMA) with 1-4 copies of SMN2.

In August 2020, Evrysdi was approved by the US FDA for the treatment of children and adults with SMA patients 2 months and older.

Evrysdi is a liquid preparation that can be administered orally or via a feeding tube at home, once a day.

It is worth mentioning that Evrysdi is the first oral therapy for SMA and the first SMA therapy that can be administered at home.

In 2 clinical trials, Evrysdi showed clinically meaningful improvements in motor function in SMA patients of different ages and disease severity (including type 1, type 2, and type 3).

In the European Union, CHMP’s positive review opinions are based on data from 2 clinical studies that represent a wide range of real-world SMA populations: The FIREFISH study was carried out in 2-7 months of symptomatic infants with type 1 SMA, SUNFISH The study was conducted in children and adults with symptomatic type 2 and type 3 SMA patients aged 2-25 years.

——In the FIREFISH study, the median age of infants entering the group was 5.

——In the SUNFISH study, the median age of patients enrolled in the group was 9 years: (1) The total score of the motor function measurement 32 (MFM-32) was measured, compared with the placebo group, and the children who received Evrysdi treatment There were clinically significant and statistically significant improvements in motor function in adults and adults at 12 months (respectively: 1.

In 2 studies, Evrysdi has good efficacy and safety.

Evrysdi is an oral liquid.

As part of the collaboration with the SMA Foundation and PTC Therapeutics, Genentech led the clinical development of Evrysdi.

Currently, Roche is conducting 4 global multi-center clinical studies (SUNFISH[NCT02908685], FIREFISH[NCT02913482], JEWELFISH[NCT03032172], RAINBOWFISH[NCT03779334]) to evaluate all types of Evrysdi treatment (type 1, type 2, type 3) The efficacy and safety of SMA and presymptomatic SMA in neonates.

Spinraza: The world's first SMA treatment drug, approved in China

SMA is a motor neuron disease that can cause muscle weakness and atrophy.

In December 2016, Spinraza (nusinersen), a drug developed by Biogen and its partner Ionis, was approved and became the world's first drug for the treatment of SMA.

In May 2019, the gene therapy Zolgensma (onasemnogene abeparvovec) from Novartis was approved, becoming the world's first gene therapy to treat SMA.

In the Chinese market, Spinraza was approved at the end of February 2019 for the treatment of patients with 5q spinal muscular atrophy (5q-SMA).

Original source: Roche receives positive CHMP opinion for Evrysdi, the first and only at home spinal muscular atrophy (SMA) treatment with proven efficacy in adults, children and infants two months and older