Royalty Pharma Cements Blockbuster Bet, Acquiring Full Royalty on SMA Drug Evrysdi

NEW YORK, NY – December 29, 2025 – In a significant strategic maneuver that underscores its confidence in a leading rare disease therapy, Royalty Pharma plc announced it has acquired the final remaining royalty interest in Roche’s spinal muscular atrophy (SMA) treatment, Evrysdi, from PTC Therapeutics. The deal, valued at $240 million upfront with up to $60 million in potential sales-based milestones, gives Royalty Pharma 100% ownership of a lucrative, tiered royalty stream on the blockbuster drug’s global sales.

This transaction is more than a simple acquisition; it is a consolidation of power over a high-growth asset and a testament to Royalty Pharma’s unique and dominant business model in the biopharmaceutical industry. By securing the complete royalty, the company ensures a predictable and expanding revenue source for years to come, while simultaneously providing PTC Therapeutics with a substantial infusion of non-dilutive capital to fuel its own development pipeline. The move highlights the intricate financial ecosystem that underpins modern drug development, where monetizing proven assets can unlock the next wave of innovation.

Anatomy of a Multi-Million Dollar Royalty Deal

The financial architecture of the agreement reveals the long-term value Royalty Pharma sees in Evrysdi. Effective from the first quarter of 2026, the company will receive 100% of a tiered royalty on worldwide net sales. This structure is designed to scale with the drug's success: Royalty Pharma will earn 8% on annual sales up to $500 million, 11% on sales between $500 million and $1 billion, 14% on sales between $1 billion and $2 billion, and a substantial 16% on all sales exceeding the $2 billion mark.

To understand the magnitude of this future revenue stream, one need only look at Evrysdi's current and projected performance. In 2024, the drug generated sales of approximately $1.9 billion. Had Royalty Pharma owned the full royalty then, it would have collected an estimated $221 million. Looking ahead, the potential returns are even more compelling. With analyst consensus projecting Evrysdi sales to reach nearly $2.9 billion by 2030, the annual royalty payment under this structure could surge to approximately $379 million.

This calculated investment positions Royalty Pharma to capitalize fully on Evrysdi's market trajectory. The positive investor reaction, with the company's stock gaining 1.5% in after-hours trading following the announcement, signals Wall Street's approval of a strategy that locks in a high-margin, growing annuity from a proven therapeutic powerhouse.

Evrysdi’s Dominance in a Fiercely Competitive Market

Evrysdi's commercial success is rooted in its clinical profile and its position within the competitive landscape of SMA treatments. Spinal muscular atrophy is a rare, debilitating genetic disease that affects motor neuron cells in the spinal cord, leading to progressive muscle weakness. For years, the treatment landscape was barren, but it has now become a multi-billion-dollar market contested by three major therapies.

Evrysdi (risdiplam), marketed by Swiss pharmaceutical giant Roche, has distinguished itself through its broad FDA approval for patients of all ages—from newborns to adults—and its convenient oral administration. This stands in stark contrast to its main competitors. Biogen's Spinraza, the first approved SMA treatment, requires regular and invasive intrathecal injections. While still a major product with over $1.7 billion in 2023 sales, the administration method can be a significant burden for patients and caregivers. Novartis' Zolgensma offers the paradigm-shifting promise of a one-time gene therapy, but its use is restricted to children under two, and it comes with a staggering price tag of over $2 million, which has tempered its initial blockbuster sales forecasts.

This combination of broad efficacy and patient-friendly oral delivery has propelled Evrysdi to a market-leading position, with over 21,000 patients treated worldwide. The global SMA treatment market, valued at over $4 billion in 2024, is projected to expand to over $13 billion by 2030, driven by improved newborn screening, supportive reimbursement policies, and the proven success of available therapies. By consolidating its stake in Evrysdi, Royalty Pharma is betting on the continued leadership of a drug that has fundamentally changed the standard of care for SMA patients.

The Unseen Hand of Biopharma Financing

While the deal is a clear strategic victory for Royalty Pharma, it also illuminates the critical, often unseen, role that royalty financing plays in the broader biopharmaceutical ecosystem. For PTC Therapeutics, the developer that originally out-licensed the drug, selling its remaining royalty stream provides a significant injection of immediate, non-dilutive capital. This cash can be deployed to advance its internal pipeline of rare disease therapies, fund costly clinical trials, or pursue other strategic initiatives without ceding equity or taking on debt.

This mechanism allows innovative companies to de-risk their financial future by converting a variable, long-term revenue stream into a guaranteed upfront payment. It is a symbiotic relationship: innovators like PTC gain the financial freedom to pursue the next breakthrough, while financiers like Royalty Pharma acquire de-risked, high-quality assets that provide predictable returns for their shareholders.

Royalty Pharma has built its empire on this model, acting as the industry's largest buyer of these valuable future income streams. Its portfolio contains royalties on more than 35 commercial products, making it a pivotal, if quiet, partner in the development and commercialization of many of modern medicine's most important drugs. This latest acquisition of the full Evrysdi royalty is not just an investment in a single product, but a reinforcement of a business model that fuels the entire cycle of biopharmaceutical research and development, ensuring that capital flows efficiently toward the creation of new medicines.