Roche's Evrysdi, an oral treatment for Spinal Muscular Atrophy, receives EMA approval in Europe
Background and Clinical Significance of EMA Approval
The European Medicines Agency (EMA) has officially approved Evrysdi (risdiplam), Roche's oral treatment for Spinal Muscular Atrophy (SMA). This approval is based on the results of the FIREFISH clinical trial, which involved infants aged 2 to 7 months with SMA, and the SUNFISH clinical trial, which involved patients aged 2 to 25 years with progressive SMA. Evrysdi works by modulating the splicing of the SMN2 gene, increasing the production of functional SMN protein. It is an oral liquid formulation that can be taken at home daily, significantly improving patient convenience.
Formation of a New Competitive Landscape with Three Major Players
This European approval is expected to significantly change the competitive landscape of the existing SMA treatment market. Biogen's Spinraza, which has been a major player in the market, is an intrathecal injection, and Novartis' Zolgensma is a high-cost, intravenous gene therapy. Evrysdi can be a great alternative for patients who have spinal deformities that make intrathecal injections difficult or who find it difficult to visit the hospital. Therefore, with its convenience and accessibility, it is expected to rapidly expand its market share in Europe and put pressure on competing products.
Financial Performance and Licensing Structure
From a financial perspective, this approval is also a major milestone. PTC Therapeutics, which has a licensing agreement with Roche, will receive a $20 million milestone payment for the first commercial sale in Europe. Evrysdi's global annual revenue is approximately 1.76 billion Swiss francs (approximately $2 billion) as of 2025, making it a blockbuster drug. Royalty Pharma has acquired all of the tiered royalties of 8% to 16% that Roche pays, maximizing capital efficiency.
Continued Growth Expected in the SMA Treatment Market
In the future, the expansion of newborn screening in the European market is expected to further increase the number of early-diagnosed patients. Early treatment, before symptoms appear, increases the preservation rate of motor neurons, making the accessibility of oral medications even more valuable. The global SMA treatment market is expected to continue to grow at a rate of more than 10% per year, reaching $5 billion to $6 billion by 2025-2026. As Evrysdi's indications continue to expand to include infants and all age groups, it is expected to become a key cash cow for Roche's biopharmaceutical division.
This European approval of Evrysdi is a significant milestone that accelerates the paradigm shift in the global Spinal Muscular Atrophy (SMA) market, which is worth approximately $5 billion to $6 billion. In the short term, Evrysdi, a convenient oral medication, will rapidly absorb the market share held by Biogen's Spinraza in Europe, leading to a market reshuffle. In the medium to long term, Roche's partner, PTC Therapeutics, will secure a $20 million milestone payment from the successful commercialization in Europe, as well as a tiered royalty of 8% to 16% on the nearly $2 billion in revenue, providing a solid financial foundation for the development of its subsequent pipeline. This will be recognized as a key case study that demonstrates the clinical success of the next-generation RNA splicing modulator platform for researchers.
Source: EMA (ema)