The Inflation Reduction Act (IRA) is introducing landmark policy changes to the healthcare industry, raising important questions about launch prices, pipeline strategy, and evidence requirements. In the IRA Question of the Week series, Avalere answers the pressing questions shaping healthcare stakeholders’ strategic decision-making as the law is implemented. In this installment, Avalere experts discuss how orphan drug investment…| Avalere
FDA approved Qalsody (tofersen) to treat patients with amyotrophic lateral sclerosis (ALS) associated with a mutation in the superoxide dismutase 1 (SOD1) gene (SOD1-ALS). Qalsody is an antisense oligonucleotide that targets SOD1 mRNA to reduce the synthesis of SOD1 protein. The approval was based o| U.S. Food and Drug Administration