EMA Approves Alnylam's Onpattro, the World's First RNAi Therapy
Commercialization of the World's First RNA Interference (RNAi) Therapy in Europe
The European Medicines Agency (EMA) has granted final approval for Onpattro (patisiran) from Alnylam Pharmaceuticals (ALNY) as a treatment for hereditary transthyretin-mediated amyloidosis (hATTR). This followed the European Commission's (EC) official approval on August 27, 2018, and the positive opinion from the EMA's Committee for Medicinal Products for Human Use (CHMP). This approval marks a significant milestone, as it represents the first commercialization of RNA interference (RNAi) technology, which inhibits gene expression. It signifies a new paradigm that goes beyond simply alleviating symptoms and aims to eliminate the root cause of the disease at the genetic level.
Overwhelming Efficacy Demonstrated in the APOLLO Phase 3 Clinical Trial
This approval is based on the results of the global Phase 3 APOLLO study, which involved 225 patients with hATTR. In the Onpattro group, patients received 0.3 mg/kg intravenously (IV) every three weeks for 18 months, resulting in an average reduction of 6.0 points in the modified Neuropathy Impairment Score +7 (mNIS+7), a measure of polyneuropathy, compared to baseline, effectively reversing disease progression. In contrast, the placebo group showed an increase of 28.0 points, resulting in a least-squares mean difference of -34.0 points (p < 0.001) compared to the control group, demonstrating remarkable statistical significance. This provides strong clinical evidence that it can potentially restore fundamental physical functions in patients suffering from nerve damage.
Clinical Success of Lipid Nanoparticle (LNP) Delivery Technology
The success of Onpattro also holds significant technological importance, as it demonstrates the effectiveness and safety of lipid nanoparticles (LNPs), a key technology for stably delivering RNA substances to target cells in the body. The LNP platform, which protects unstable single-stranded RNA and efficiently delivers it into liver cells, has become a cornerstone for the development of various gene therapies. It has also established safety standards for subsequent messenger RNA (mRNA) and small interfering RNA (siRNA)-based pipelines, including COVID-19 vaccines, at the regulatory level. As a result, a full-scale reevaluation of the value of global biotechnology companies with platform technology capabilities is expected.
Changes in the hATTR Market Landscape and Global Competitive Structure
The hATTR treatment market is projected to grow at an annual rate of over 11%, reaching a maximum of $8.95 billion by 2025. With the launch of Onpattro, a fierce competitive landscape has emerged with Pfizer's existing standard treatment, Vyndaqel (tafamidis), and Ionis's competing drug, Tegsedi (inotersen). With Onpattro recording global sales of $475 million in 2021 and successfully establishing itself in the market, Alnylam is accelerating patient conversion to its next-generation subcutaneous (SC) formulation, Amvuttra (burosiran), to maximize market share.
Alnylam's Onpattro received final approval from the European Commission in 2018, marking its entry into the hATTR treatment market, which is valued at approximately $8.9 billion, as the world's first RNA interference (RNAi) therapy. It demonstrated strong market competitiveness by achieving remarkable data in the APOLLO Phase 3 clinical trial, with a 34-point improvement in the Neuropathy Impairment Score +7 (mNIS+7) compared to the control group, thereby threatening Pfizer's existing standard treatment, Vyndaqel, and Ionis's Tegsedi. In the short term, it is accelerating patient conversion from Onpattro, with annual sales of $475 million, to Amvuttra, its next-generation subcutaneous drug, to improve the convenience of intravenous administration. In the medium to long term, as the first successful case of an LNP-based platform, it will provide guidelines and regulatory standards for new drug approvals to researchers and venture capitalists developing gene therapies, thereby promoting investment in the bio sector.
Source: EMA (ema)