THE WIRE · № 62100
BioMarin Announces Five-Year Phase 3 Data for POMBILITI® (cipaglucosidase alfa-atga) + OPFOLDA® (miglustat) at World Muscle Society Annual Congress
01 THE MACHINE READ
WHY IT MATTERS.
Long-term Phase 3 data support durability of an approved Pompe therapy, but no new approval or revenue guidance is disclosed.
Supports existing Pompe franchise, not a new catalyst
Regulatory or payer decisions on label expansion
No new efficacy or sales figures disclosed
FULL DISPATCH
Long-term data reinforce durable efficacy and safety of POMBILITI + OPFOLDA in late-onset Pompe disease SAN RAFAEL, Calif., Oct. 2, 2026 /PRNewswire/ -- BioMarin Pharmaceutical Inc. (Nasdaq: BMRN) today announced new data evaluating the efficacy and safety of POMBILITI® (cipaglucosidase...
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STANDALONE ITEM — NOT YET FOLDED INTO A DEVELOPING STORY.
03 WHO'S INVOLVED
ENTITIES ON FILE.
04 RECEIPTS
THE SOURCE RECORD.
1 SOURCES CONFIRMED05 PROPAGATION
WHAT THIS TOUCHES.
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New Phase 3 trial: A Study of JNJ-99245354 (ITI-1284) as an Adjunctive Treatment in Participants With Generalized Anxiety Disorder (GAD) Who Have an Inadequate Response to GAD Treatment — Intra-Cellular Therapies, Inc.2026-10-02 12:00 UTC
Ascletis' ASC36_35FDC Demonstrated Potential for Once-Monthly to Once-Quarterly Dosing and Robust Weight-Loss in Preclinical Studies Presented at EASD 20262026-10-02 09:00 UTC
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