Yeji

$RARE

Ultragenyx Pharmaceutical Inc.

Runway:3 mo
NasdaqMarket cap: —CIK 0001515673

Upcoming catalysts

8 surfaced
PDUFAAug 23, 2026· in 78d
$RAREUltragenyx Pharmaceutical Inc.
pariglasgene brecaparvovec · Glycogen Storage Disease Type Ia (GSDIa)
“The FDA granted the BLA Priority Review and assigned a Prescription Drug User Fee Act (PDUFA) action date of August 23, 2026.”
PDUFASep 19, 2026· in 105d
$RAREUltragenyx Pharmaceutical Inc.
ux111 · Sanfilippo syndrome Type A (MPS IIIA)
“The FDA set a Prescription Drug User Fee Act (PDUFA) action date of September 19, 2026.”
NDA/BLA filedDate TBD~ low confidence
$RAREUltragenyx Pharmaceutical Inc.
“On December 30, 2025, Ultragenyx Pharmaceutical Inc. (the "Company") issued a press release announcing that that it has completed the rolling submission of its Biologics License Application ("BLA") to the U.S. Food and Drug Administration (the "FDA") seeking approval for DTX401 AAV gene therapy (pariglasgene brecaparvovec) as a treatment for Glycogen Storage Disease Type Ia ("GSDIa").”
NDA/BLA filedDate TBD
$RAREUltragenyx Pharmaceutical Inc.
ux111 · Sanfilippo syndrome Type A (MPS IIIA)
“On December 19, 2024, Ultragenyx Pharmaceutical Inc. (the ”
PDUFADate TBD
$RAREUltragenyx Pharmaceutical Inc.
ux111 · Sanfilippo syndrome Type A (MPS IIIA)
“BLA resubmitted to FDA with PDUFA date expected in third quarter 2026. These longer-term data were included in the resubmitted Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for UX111. The Company anticipates up to a six-month review period from the date of resubmission per FDA regulations, with a PDUFA date expected in the third quarter of 2026.”
NDA/BLA filedDate TBD
$RAREUltragenyx Pharmaceutical Inc.
ux111 · Sanfilippo syndrome Type A (MPS IIIA)
“On January 30, 2026, Ultragenyx Pharmaceutical Inc. (the "Company") issued a press release announcing that that it has resubmitted its Biologics License Application ("BLA") seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA) to the U.S. Food and Drug Administration (the "FDA" or the "Agency").”
PDUFADate TBD
$RAREUltragenyx Pharmaceutical Inc.
ux111 · Sanfilippo syndrome Type A (MPS IIIA)
“A Prescription Drug User Fee Act (PDUFA) action date is expected to be assigned within a month of resubmission. The Company anticipates up to a six-month review period from the date of resubmission per FDA regulations, with a PDUFA date expected in the third quarter of 2026.”
NDA/BLA filedDate TBD~ low confidence
$RAREUltragenyx Pharmaceutical Inc.
ux111 · Sanfilippo syndrome Type A (MPS IIIA)
“These longer-term data were included in the resubmitted Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) seeking accelerated approval for UX111.”

Drug pipeline

ux111
Sanfilippo syndrome Type A (MPS IIIA)
pariglasgene brecaparvovec
Glycogen Storage Disease Type Ia (GSDIa)
No Intervention
Mucopolysaccharidosis VII
Bisphosphonate
Osteogenesis Imperfecta
Standard of Care (SOC)
Wilson Disease
Setrusumab
Osteogenesis Imperfecta
GTX-102
Angelman Syndrome
Oral prednisolone
Glycogen Storage Disease Type IA
Sialic Acid Extended Release (SA-ER)
GNE Myopathy
UX053
Glycogen Storage Disease Type III
Triheptanoin
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
UX003
Sly Syndrome
BPS804 20mg/Kg
Osteopenia
Reactive Corticosteroid Taper Regimen
Ornithine Transcarbamylase (OTC) Deficiency
SA-ER 500 mg
GNE Myopathy
Adjuvant Immunomodulatory (IM) Therapy
Mucopolysaccharidosis IIIA
BPS804
Osteogenesis Imperfecta
UX007
Long-chain Fatty Acid Oxidation Disorders (LC-FAOD)
DTX101
Hemophilia B
Sialic Acid Extended Release (SA-ER) Tablets
Hereditary Inclusion Body Myopathy (HIBM)
Aceneuramic Acid Extended-Release Tablets
Hereditary Inclusion Body Myopathy
ABO-102
MPS IIIA
Mepsevii
MPS VII
Aceneuramic Acid Extended-Release
Hereditary Inclusion Body Myopathy
UX016
GNE Myopathy
steroid regimen
GSD1
Oral Corticosteroids
OTC Deficiency
aceneuramic acid extended-release (Ace-ER)
Hereditary Inclusion Body Myopathy

Recent & active trials

NCTDrugPhaseStatusPrimary completion
NCT06636383No Intervention—Recruiting2036-12-01
NCT04632953No Intervention—Active Not Recruiting2035-12-01
NCT03604835No Intervention—Recruiting2033-05-01
NCT03651505No Intervention—Active Not Recruiting2032-12-01
NCT04783428No Intervention—Active Not Recruiting2032-02-28
NCT07157254GTX-102Phase 2Recruiting2030-01-01
NCT03636438No Intervention—Active Not Recruiting2029-12-01
NCT04884815Standard of Care (SOC)Phase 2Active Not Recruiting2029-03-01
NCT06415344GTX-102Phase 3Enrolling By Invitation2029-02-01
NCT07511556UX016Phase 2Not Yet Recruiting2028-12-01
NCT05345171Oral CorticosteroidsPhase 3Active Not Recruiting2027-09-01
NCT05933200TriheptanoinPhase 3Active Not Recruiting2027-08-01
NCT04360265Adjuvant Immunomodulatory (IM) TherapyPhase 3Enrolling By Invitation2027-08-01
NCT02716246ux111Phase 3Recruiting2027-07-01
NCT06636071SetrusumabPhase 3Active Not Recruiting2027-01-01
NCT06617429GTX-102Phase 3Active Not Recruiting2026-07-01
NCT05768854BisphosphonatePhase 3Active Not Recruiting2025-10-23
NCT05125809SetrusumabPhase 3Active Not Recruiting2025-10-20
NCT03970278No Intervention—Completed2025-02-25
NCT04259281GTX-102Phase 2Completed2025-01-08

Recent catalysts

NDA/BLA filedApr 2, 2026· 65d ago
$RAREUltragenyx Pharmaceutical Inc.
ux111 · Sanfilippo syndrome Type A (MPS IIIA)
“the U.S. Food and Drug Administration (the "FDA") has accepted for review the Company's resubmitted Biologics License Application seeking accelerated approval for UX111 (rebisufligene etisparvovec) AAV9 gene therapy as a treatment for patients with Sanfilippo syndrome Type A (MPS IIIA).”
NDA/BLA filedFeb 23, 2026· 103d ago
$RAREUltragenyx Pharmaceutical Inc.
pariglasgene brecaparvovec · Glycogen Storage Disease Type Ia (GSDIa)
“the U.S. Food and Drug Administration (the "FDA") has accepted for review the Biologics License Application (the "BLA") seeking approval of DTX401 AAV gene therapy (pariglasgene brecaparvovec) for the treatment of Glycogen Storage Disease Type Ia (GSDIa).”
CRLJul 11, 2025· 330d ago
$RAREUltragenyx Pharmaceutical Inc.
ux111 · Sanfilippo syndrome Type A (MPS IIIA)
“On July 11, 2025, Ultragenyx Pharmaceutical Inc. (the ”
CRLJul 1, 2025· 340d ago~ low confidence
$RAREUltragenyx Pharmaceutical Inc.
ux111 · Sanfilippo syndrome Type A (MPS IIIA)
“The resubmitted BLA includes comprehensive responses to chemistry, manufacturing, and controls (CMC)-related observations outlined in a Complete Response Letter (the "CRL") issued in July 2025”