Royalty Pharma acquired the final portion of PTC Therapeutics' royalty on Roche's Evrysdi for $240 million upfront plus up to $60 million in milestones, giving it 100% ownership of the 8-16% tiered royalty. Evrysdi generated $1.9 billion in 2024 sales with projected growth to $2.9 billion by 2030. RPRX stock is up 53% year-to-date but down slightly on Wednesday. The company also recently acquired royalty interests in Nuvalent's cancer drugs.

Royalty Pharma has acquired the final portion of PTC Therapeutics' remaining royalty on Roche's Evrysdi for $240 million upfront plus up to $60 million in sales-based milestones. Following the transaction, Royalty Pharma will own 100% of the tiered 8% to 16% royalty on worldwide net sales of Evrysdi, an FDA-approved spinal muscular atrophy treatment that generated approximately $1.9 billion in sales in 2024 with projected growth to $2.9 billion by 2030.

PTC Therapeutics' CEO Matthew Klein highlighted the commercial potential of Sephience, a drug for treating phenylketonuria (PKU), projecting significant market opportunities in the U.S. and internationally. The company expects to reach cash flow breakeven with growing sales and believes most patients will respond positively to the treatment.

Novartis reported strong Q2 2025 financial performance with 11% sales growth, double-digit core operating income increase, and raised full-year guidance. Key growth drivers included oncology and specialty medicine brands like Kisqali, Entresto, and Pluvicto.

Pomerantz LLP is investigating claims on behalf of investors of Capricor Therapeutics, Inc. (CAPR) after the company announced that the FDA will hold an advisory committee meeting for its Biologics License Application, causing the stock price to drop.

Pomerantz LLP is investigating claims on behalf of investors of PTC Therapeutics, Inc. (PTCT) due to concerns about the company's securities fraud or other unlawful business practices. The investigation is related to the Phase 2 PIVOT-HD study of PTC518 (votoplam) in Huntington's disease patients.

The RNA Targeting Small Molecule Drug Discovery Market is expected to grow significantly, driven by advancements in RNA biology and AI-driven drug discovery. However, the complexity of RNA structures and target specificity pose challenges for the industry.

The mitochondrial disease market is witnessing steady growth, driven by increasing awareness, improved genetic diagnostics, and advancements in personalized medicine. The article provides insights into the market landscape for Leigh Syndrome, Leber's Hereditary Optic Neuropathy, MELAS Syndrome, and Thymidine Kinase 2 Deficiency.

PTC Therapeutics, Inc. (PTCT) faces a potential securities fraud investigation after the European Medicines Agency issued a negative opinion on the renewal of the conditional marketing authorization for its drug Translarna.

PTC Therapeutics, Inc. (PTCT) faces a potential investigation after the European Medicines Agency's Committee for Medicinal Products for Human Use issued a negative opinion on the renewal of the conditional marketing authorization of Translarna™ (ataluren) for the treatment of nmDMD, leading to a drop in the company's stock price.

PTC Therapeutics (PTCT) falls as the EC's advisory committee adopts a negative opinion regarding the renewal of the conditional marketing approval of Translarna to treat nmDMD in the EU.

PTC Therapeutics (PTCT) witnessed a jump in share price last session on above-average trading volume. The latest trend in earnings estimate revisions for the stock suggests that there could be more strength down the road.