Royalty Pharma plc has acquired a royalty interest in Alnylam's breakthrough RNAi therapeutic AMVUTTRA from funds managed by Blackstone Life Sciences for $310 million. The transaction represents a significant vote of confidence in the commercial trajectory of this FDA-approved treatment for ATTR amyloidosis, a progressive, degenerative and fatal disease caused by misfolded proteins that accumulate in the nerves, heart and GI tract.
The royalty interest being acquired stems from Blackstone's 2020 financing collaboration with Alnylam, in which Blackstone invested to support AMVUTTRA's pivotal Phase 3 HELIOS-B trial. Pablo Legorreta, founder and Chief Executive Officer of Royalty Pharma, emphasized the therapeutic's clinical impact, stating that "AMVUTTRA is a breakthrough RNAi therapeutic that delivers compelling benefits to patients, including a meaningful reduction in all-cause mortality for ATTR cardiomyopathy patients."
Strong Commercial Performance and Market Opportunity
AMVUTTRA has demonstrated impressive commercial success since its approvals, reaching approximately $1 billion in sales in 2024, which represented 74% year-over-year growth. The drug received FDA approval for the treatment of TTR amyloidosis with cardiomyopathy (ATTR-CM) in 2025 and for hereditary TTR amyloidosis with polyneuropathy (hATTR-PN) in 2022.
The market opportunity for AMVUTTRA is substantial, with approximately 30,000 hATTR-PN patients globally and more than 300,000 ATTR-CM patients globally. Notably, only 20% of ATTR-CM patients are currently diagnosed, indicating significant potential for market expansion as diagnosis rates improve. ATTR-CM represents a fast-growing category driven by new therapeutic options and improving diagnosis rates.
Analyst consensus projects AMVUTTRA sales to exceed $6 billion by 2028, underscoring the significant long-term commercial potential of this RNAi therapeutic.
Transaction Details and Strategic Rationale
Under the terms of the agreement, Royalty Pharma has acquired Blackstone's 1% royalty on worldwide net sales of AMVUTTRA in exchange for an upfront payment of $310 million. The royalty duration extends through March 2035, and the transaction covers AMVUTTRA sales beginning on October 1, 2025.
The transaction excludes the fixed payments paid to Blackstone as part of the original transaction in which Blackstone invested $70 million. Royalty Pharma expects the transaction to deliver returns consistent with its stated targets for approved products under a range of scenarios that factor in significant potential competition from Alnylam's follow-on product, nucresiran.
Legorreta noted that AMVUTTRA's "impressive commercial trajectory underscores the significant market opportunity for the product and positions it as a highly attractive contributor to Royalty Pharma's portfolio."
Industry Context and Company Backgrounds
Founded in 1996, Royalty Pharma is the largest buyer of biopharmaceutical royalties and a leading funder of innovation across the biopharmaceutical industry. The company has assembled a portfolio of royalties on more than 35 commercial products, including Vertex's Trikafta, GSK's Trelegy, Roche's Evrysdi, Johnson & Johnson's Tremfya, Biogen's Tysabri and Spinraza, and 17 development-stage product candidates.
Blackstone Life Sciences is an industry-leading private investment platform with capabilities to invest across the life cycle of companies and products within the key life science sectors. The platform currently has $12 billion in assets under management and focuses on bringing promising new medicines and medical technologies to market that improve patients' lives.
