Europe's drug regulator has firmly decided against renewing the marketing authorization for Translarna, a treatment developed by PTC Therapeutics for Duchenne muscular dystrophy (DMD). The European Medicines Agency (EMA) initially granted authorization in 2014, contingent upon PTC providing further data demonstrating the drug's efficacy in specific DMD cases. However, following four reviews of the available evidence, the EMA concluded that Translarna's effectiveness remains unconfirmed.
Translarna's EMA Rejection
The EMA's decision marks a significant setback for PTC Therapeutics, as Translarna was intended to address a rare muscle-wasting disease with limited treatment options. Duchenne muscular dystrophy affects primarily males and is characterized by progressive muscle degeneration and weakness. The agency will now forward its verdict to the European Commission for a final decision on the drug's status within EU countries.
Novartis's Oncology Expansion
Novartis is expanding its oncology portfolio by licensing a small molecule cancer drug from Chengdu Baiyu Pharmaceutical, a China-based company, for an upfront payment of $70 million. The agreement includes potential milestone payments of up to $1.1 billion. While the specific biological or disease targets for the drug were not disclosed, Chengdu Baiyu noted its focus on researching the core pharmacological components of Ginkgo biloba and developing "botanical" drugs. The company's pipeline includes six cancer-related experimental drugs.
Leqembi's Regulatory Hurdles
Eisai and Biogen's Leqembi, a closely watched Alzheimer's disease drug, has faced a setback in Australia. The Therapeutic Goods Administration (TGA) decided not to approve the drug, citing that its safety risks outweigh its potential benefits. Eisai plans to request a reconsideration of this decision. Leqembi is approved in the U.S. for individuals in the early stages of Alzheimer's, a neurodegenerative disease affecting over 400,000 people in Australia. The drug has also encountered resistance in Europe.
