First FDA Approval in FSGS — Major Commercial Milestone
FILSPARI received full FDA approval for FSGS (April 13), becoming the first and only approved medicine for this rare disease; company estimates >30,000 U.S. patients currently eligible in FSGS and >100,000 total U.S. patients eligible across IgA nephropathy and FSGS, supporting a modeled ~$3 billion potential peak sales opportunity.
Strong FILSPARI Commercial Performance
Record commercial demand with 993 new patient start forms in Q1 and U.S. FILSPARI net product sales of $105.2 million, an ~88% year-over-year increase despite typical Q1 insurance resets, gross-to-net dynamics, and fewer revenue shipping weeks.
Robust Company Revenue and Product Mix
Total U.S. net product sales were $124.5 million and total revenue was $127.2 million in Q1 2026; Thiola/Thiola EC contributed $19.3 million and licensing/collaboration revenue was $2.7 million.
Profitability Improvement on a Non-GAAP Basis
On a non-GAAP adjusted basis the company reported net income of $4.1 million, or $0.05 per share, versus a non-GAAP net loss of $16.9 million (−$0.19) in Q1 2025 — a material improvement in underlying profitability.
Pipeline Progress — HARMONY Phase III Restarted
Dosed the first new patient in the pivotal Phase III HARMONY study of pegtibatinase for classical homocystinuria after enrollment restart; top-line HARMONY results are expected in 2027 and the program targets an estimated 7,000–10,000 addressable HCU patients globally.
Supportive Clinical and Real-World Evidence
New publications and data (CJASN, PROTECT) reinforce FILSPARI’s efficacy: PROTECT showed patients reaching proteinuria <0.3 g/g had eGFR decline <1 mL/min/yr; DUPLEX data showed sustained proteinuria reductions to ~1.5 g/g and CJASN publication demonstrated consistent efficacy/safety in genetic FSGS. These data support strong physician adoption and persistence.
Solid Balance Sheet and Milestone Receipt
As of March 31, 2026 the company reported cash, cash equivalents, marketable securities and receivables of ~ $352 million (cash balance reported ~$264.7M with a $25M Mirum milestone received in April), supporting near-term operations and pipeline investment.