Royalty Pharma plc (RPRX) has had quite the year, with shares climbing roughly 53% year to date. And this week, the company made another strategic move that shows exactly why investors have been paying attention.

On Monday, Royalty Pharma acquired the final piece of PTC Therapeutics Inc.'s (PTCT) remaining royalty interest in Roche Holdings AG's (RHHBY) Evrysdi. The price tag? A cool $240 million upfront, plus up to $60 million in sales-based milestones down the road.

For those unfamiliar, Evrysdi is an orally administered survival motor neuron-2 (SMN2) splicing modifier used to treat spinal muscular atrophy (SMA). It's marketed by Roche and has become a genuine blockbuster in the rare disease space.

Why This Deal Matters

The FDA approved Evrysdi back in 2020, and since then the drug has treated over 21,000 patients worldwide. That's not just a footnote—it represents substantial commercial traction for a rare disease therapy.

The financials tell an even more compelling story. In 2024, Evrysdi generated sales of approximately 1.6 billion Swiss francs (about $1.9 billion), representing 18% year-over-year growth at constant exchange rates. Analyst consensus projects the drug will reach 2.3 billion Swiss francs ($2.9 billion) in annual sales by 2030.

With this latest acquisition, Royalty Pharma will now own 100% of the tiered 8% to 16% royalty paid by Roche on worldwide net sales of Evrysdi. The company will start receiving the increased royalty beginning in the first quarter of 2026, based on Evrysdi product sales from the fourth quarter of 2025.

The Competitive Dynamics in SMA

The spinal muscular atrophy treatment landscape has become increasingly competitive, which makes Evrysdi's sustained growth all the more impressive.

Back in September, Biogen Inc. (BIIB) unveiled plans to test its Spinraza therapy in patients who've already been treated with Evrysdi. That's a direct competitive challenge, essentially trying to win back market share from patients who switched away.

More recently, in March 2025, Novartis AG (NVS) shared safety and efficacy results from its Phase 3 program for investigational intrathecal onasemnogene abeparvovec (OAV101 IT) in patients aged two to less than 18 years with spinal muscular atrophy.

In the Phase 3b STRENGTH study, treatment with OAV101 IT in patients who had discontinued treatment with either Biogen's Spinraza or Evrysdi demonstrated stabilization of motor function over 52 weeks of follow-up. The competition isn't going away, but Evrysdi continues to hold its own.

Royalty Pharma's Broader Strategy

The Evrysdi deal isn't happening in isolation. Royalty Pharma has been actively building its portfolio of royalty interests across therapeutic areas.

In December, the company acquired a pre-existing royalty interest in Nuvalent Inc.'s (NUVL) neladalkib and zidesamtinib from an undisclosed third party for up to $315 million. These are both promising candidates in the non-small cell lung cancer space.

Neladalkib is in development for patients with ALK mutation-positive non-small cell lung cancer (NSCLC), while zidesamtinib is being developed for ROS1 mutation-positive NSCLC. In November 2025, Nuvalent reported positive pivotal results for neladalkib in TKI pre-treated patients, which demonstrated durable activity and a generally well-tolerated safety profile.

Neladalkib is also being evaluated in an ongoing Phase 3 study in TKI-naïve patients. Meanwhile, zidesamtinib is currently under review by the U.S. Food and Drug Administration with an action date of September 18, 2026, for TKI pre-treated patients. The drug is also being assessed in an ongoing Phase 1/2 study in TKI-naïve patients.

Existing analyst consensus projects neladalkib sales will reach approximately $3.5 billion and zidesamtinib sales will hit approximately $1.9 billion by 2035. Those are the kinds of numbers that make royalty investments attractive.

Royalty Pharma shares were down 0.35% at $38.85 at the time of publication on Wednesday. But with the year's gains already in the books and a pipeline of royalty streams flowing in, investors seem to be betting that the company's strategy of acquiring stakes in proven therapies will continue paying dividends.