Royalty Pharma Goes All-In on Evrysdi with $300M Final Royalty Deal

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

This transaction marks the culmination of a multi-year, multi-billion-dollar strategy to consolidate one of the most valuable assets in its portfolio. Evrysdi, an oral therapy for the debilitating genetic disorder SMA, has seen remarkable market uptake since its FDA approval in 2020, treating over 21,000 patients worldwide and generating sales of approximately $1.9 billion in 2024.

Consolidating a Crown Jewel

Royalty Pharma’s path to 100% ownership has been a deliberate and systematic campaign. The firm, known as the largest buyer of biopharmaceutical royalties, first invested in Evrysdi in July 2020, acquiring a 43% stake in PTC’s royalty for $650 million. This was followed by a much larger transaction in October 2023, when it paid $1.0 billion to increase its share to 81%.

With this final acquisition, Royalty Pharma now commands the entire tiered royalty paid by Roche on Evrysdi’s worldwide net sales. Starting in the first quarter of 2026, the company will receive 8% on 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. Given that analyst consensus projects Evrysdi sales to reach $2.9 billion by 2030, the deal positions Royalty Pharma to capture significant upside from the drug's continued growth.

This acquisition perfectly aligns with the company’s core business model: identifying and securing long-term revenue from proven, high-growth biopharmaceutical products. By consolidating its stake, Royalty Pharma eliminates any shared interest and maximizes its return on an asset with patent protection extending into the early 2030s. The move has been viewed favorably by market observers, with many Wall Street analysts maintaining a “Strong Buy” rating on the company’s stock, citing its robust portfolio and strategic acumen.

Evrysdi's Enduring Market Power

The confidence displayed by Royalty Pharma is rooted in Evrysdi's powerful market position and distinct clinical advantages. As the first and only orally administered, at-home treatment for SMA, it offers a significant convenience advantage over its main competitors. Biogen’s Spinraza, the first approved SMA treatment, requires regular intrathecal injections, while Novartis’s Zolgensma is a one-time gene therapy administered intravenously in a clinical setting, primarily for infants and young children.

Evrysdi’s broad label, covering adults, children, and infants as young as two months, has fueled its rapid adoption. The drug’s 18% year-over-year sales growth in 2024 underscores its momentum in a competitive but expanding market. While its competitors are not standing still—Biogen is pursuing approval for a higher, less frequent dose of Spinraza, and Novartis is studying Zolgensma in older pediatric patients—Evrysdi’s non-invasive profile gives it a durable edge in patient and physician preference, particularly for long-term maintenance therapy.

A Strategic Pivot for PTC Therapeutics

For PTC Therapeutics, the sale of its final royalty stake represents a strategic pivot. The infusion of $240 million in non-dilutive capital provides immediate financial flexibility and strengthens its balance sheet. The company has stated the proceeds will be used to retire outstanding debt obligations with Blackstone Life Sciences and to fund its planned operations.

This divestment allows PTC to shift its focus and resources toward its internal pipeline and commercial-stage assets. By monetizing the passive royalty stream from Evrysdi, the company can now invest more aggressively in its own research and development programs, including its efforts around the Duchenne muscular dystrophy drug Translarna. Analysts note that the deal removes a “significant overhang” on PTC’s stock, clarifying its go-forward strategy and allowing investors to value the company based on the potential of its proprietary pipeline rather than its ties to a Roche-marketed product.

The Evolving Landscape of SMA Treatment

The spinal muscular atrophy treatment market has been revolutionized over the past decade. Once a disease with no therapeutic options, it now has three powerful drugs that have dramatically improved patient outcomes. The global market, valued at over $5 billion in 2025, is projected to grow at a compound annual growth rate of nearly 15% over the next decade, potentially reaching over $25 billion by 2035.

This growth is fueled not only by existing therapies but also by a dynamic research pipeline. Emerging treatments are exploring novel mechanisms, including neuroprotection and muscle function improvement. Biogen’s next-generation antisense oligonucleotide, BIIB115, is in early-stage trials with the promise of extended dosing intervals. Furthermore, researchers are actively exploring combination therapies, using multiple drugs in concert to achieve optimal results for patients. This continuous innovation ensures that while Royalty Pharma has secured a powerful revenue stream with Evrysdi, the push to find even better solutions for those living with SMA continues to accelerate.