Ractigen Therapeutics' RAG-21 Receives FDA Orphan Drug Designation for Treating FUS-ALS

Ractigen Therapeutics, a clinical-stage pharmaceutical company dedicated to developing innovative therapies, today announced that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) to RAG-21, its novel siRNA therapy targeting the FUS gene, for the treatment of amyotrophic lateral sclerosis (ALS). RAG-21 is an siRNA therapy specifically designed to target FUS-ALS, one of the most aggressive subtypes of amyotrophic lateral sclerosis (ALS).

Publish Date: 19-11-2024 Source: Ractigen Therapeutics

Amyotrophic lateral sclerosis (ALS), also known as motor neuron disease (MND), is a degenerative disorder of the nervous system that manifests as progressive muscle wasting and weakness. It is both debilitating and fatal, typically occurring later in life. The disease advances swiftly, with a median survival period of three to five years, and only 20% of patients survive five to ten years after symptom onset. Respiratory failure is the most common cause of death in ALS patients. Despite the generally grim prognosis, there is significant variability in survival rates. As many as 10% of individuals with ALS manage to live more than eight years following the emergence of initial symptoms. Genetic risk factors include gene mutations such as SOD1, TARDBP, FUS, C9orf72, TBK1, and NEK1. Most of these risk factors have been uncovered within the past decade. Currently, the U.S. Food and Drug Administration (FDA) has approved seven drugs for the treatment of ALS and its associated symptoms. These medications include Qalsody, RELYVRIO, Radicava, Rilutek, Tiglutik, Exservan, and Nuedexta.

ALS prevalence in the United States ranges between 5.2 and 6.5 cases per 100,000 population, whereas incidence cases range between o.6 and 2.8 cases per 100,000 population.

However, the current Amyotrophic lateral sclerosis (ALS) treatment market share, market uptake, and attribute analysis concerning the most potential emerging therapies (NP001, Dazucorilant, VRG50635, etc..) has been provided under the market outlook section of the study covering 8 MM countries; The United States, EU5 (Germany, Spain, France, Italy, UK) Japan and China.

In terms of pharmacologic therapies, several pharmaceutical products are being approved and under different phases of development for the Amyotrophic lateral sclerosis (ALS) treatment. The key companies in the advanced development stage are Neuvivo, Corcept Therapeutics, Verge Genomics, etc..

Based on solid domain and business knowledge, Thelansis Knowledge Partners has published the market outlook forecast report on Amyotrophic Lateral Sclerosis (ALS) to provide a clear understanding of disease area background, epidemiology, current and future competitions, the country-specific standard of care, and the complete market forecast for 2023 to 2033.

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