Ultragenyx to Sell Rare Pediatric Disease Voucher for $210M

Shots: Ultragenyx has entered into a definitive agreement to sell a Rare Pediatric Disease PRV for $210M, provide Ultragenyx with significant non-dilutive capital to advance therapies for rare & ultra-rare diseases Rare Pediatric Disease PRV was received following FDA approval of Genglycos (pariglasgene brecaparvovec-opnr), the first treatment targeting the underlying cause of glycogen storage disease…

ByRidhi Rastogi2 days ago

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The US FDA Approves Ultragenyx’s Fayuvi for Sanfilippo Syndrome Type A

Shots: The FDA has granted full approval to Fayuvi (rebisufligene etisparvovec-hopf) for pediatric pts with Sanfilippo syndrome Type A, plus a Priority Review Voucher; commercial product will be available for shipment to QTCs within 30-60 days Approval was supported by pivotal Transpher A & long-term follow-up data, where Fayuvi-treated pts from the mITT population (n=17)…

ByRidhi RastogiSeptember 18, 2026

NEWS

Ultragenyx Receives FDA Accelerated Approval for GENGLYCOS Gene Therapy to Treat the Underlying Cause of GSDIa

Shots: Ultragenyx reported the US FDA accelerated approval to GENGLYCOS (pariglasgene brecaparvovec-opnr; DTX401) for adults and pediatric patients (≥8yrs., n=46) with glycogen storage disease type Ia (GSDIa) The approval was supported by the P-III (GlucoGene) study, in which GENGLYCOS (n=20) demonstrated a statistically significant reduction in cornstarch requirements at 48wks. vs PBO (n=24) (p<0.001). The…

ByPrince GiriAugust 20, 2026

NEWS

The US FDA Accepts Ultragenyx’s Resubmitted BLA for UX111 in Sanfilippo Syndrome Type A

Shots: The US FDA has accepted the resubmitted BLA of UX111 (rebisufligene etisparvovec) for accelerated approval in Sanfilippo syndrome Type A (MPS IIIA), with a PDUFA action date of Sep 19, 2026 UX111 BLA is supported by up to 8yrs. of follow-up data showing durable clinical benefit & acceptable safety. Also, during prior late-cycle review,…

ByRidhi RastogiApril 6, 2026

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Ultragenyx Reports the P-III (Enh3ance) Trial for DTX301 AAV8 Gene Therapy in OTC Deficiency

Shots: Ultragenyx has reported the P-III (Enh3ance) study data assessing DTX301 AAV8 gene therapy vs PBO for the treatment of ornithine transcarbamylase (OTC) deficiency DTX301 reduced 24hr. plasma ammonia by 18% vs PBO at Wk. 36, with ammonia levels maintained in the normal range, despite a 27% mean decrease in scavenger drugs & ~13% increase…

ByRidhi RastogiMarch 13, 2026

NEWS

Ultragenyx Completes FDA Rolling BLA Submission for DTX401 in Glycogen Storage Disease Type Ia (GSDIa)

Shots: Ultragenyx has completed rolling submission of a BLA to the FDA seeking approval of DTX401 (pariglasgene brecaparvovec) for the treatment of GSDIa BLA is supported by data from 52 pts with 6yrs. of follow-up. Previously reported P-III (GlucoGene) trial results showed significant reductions in both quantity & frequency of daily cornstarch intake, improved euglycemia…

ByRidhi RastogiDecember 31, 2025

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Regeneron’s Evkeeza Receives the US FDA’s Approval to Treat Homozygous Familial Hypercholesterolemia in Younger Patients

Shots: The US FDA has approved label expansion to Evkeeza (evinacumab-dgnb) as an adj. to diet, exercise & other lipid-lowering therapies for the treatment of children (1 to <5yrs.) with homozygous familial hypercholesterolemia (HoFH) Approval was supported by efficacy & safety results in 6 children with HoFH (incl. PK data from 4) who participated in…

ByRidhi RastogiSeptember 29, 2025

NEWS

Ultragenyx Completes Patient Enrolment in P-III (Aspire) Trial of GTX-102 for Angelman Syndrome

Shots: Ultragenyx has completed enrolment in its P-III (Aspire) trial to assess GTX-102 (apazunersen) in Angelman syndrome pts with confirmation of full maternal UBE3A gene deletion; Aurora study in more ages & genotypes to begin in H2'25 Trial will evaluate GTX-102 (intrathecal, 8mg QM for 3mos., then up to 14mg Q4M) vs a sham treatment…

ByRidhi RastogiAugust 1, 2025

NEWS

Ultragenyx Reports the Health Canada’s Approval of Evkeeza (evinacumab) to Treat Children with Homozygous Familial Hypercholesterolemia (HoFH)

Shots: Health Canada has approved label expansion of Evkeeza, as an adjunct to diet & other lipid-lowering therapies, to treat children (≥6mos.) with HoFH Approval was based on model-based analysis that predicted similar or greater LDL-C reductions with Evkeeza (15mg/kg, Q4W) in children (6mos. to 5yrs.) at 24wks. Data from compassionate use in 5 children…

ByDipanshu DixitJuly 15, 2025

NEWS

Ultragenyx Reports the EC’s Approval of Evkeeza (evinacumab) to Treat 6-Months Old Children with Homozygous Familial Hypercholesterolemia (HoFH)

Shots: The EC has approved Evkeeza (an angiopoietin-like 3 inhibitor), as an adjunct to diet & other lipid-lowering therapies, to treat children (6mos. to 5yrs.) with HoFH Approval was based on model-based analysis that predicted similar or greater LDL-C reductions with Evkeeza (15mg/kg, Q4W) in children (6mos. to 5yrs.) at 24wks. Data from compassionate…

BySenior EditorJanuary 6, 2025

Ultragenyx to Sell Rare Pediatric Disease Voucher for $210M

The US FDA Approves Ultragenyx’s Fayuvi for Sanfilippo Syndrome Type A

Ultragenyx Receives FDA Accelerated Approval for GENGLYCOS Gene Therapy to Treat the Underlying Cause of GSDIa

The US FDA Accepts Ultragenyx’s Resubmitted BLA for UX111 in Sanfilippo Syndrome Type A

Ultragenyx Reports the P-III (Enh3ance) Trial for DTX301 AAV8 Gene Therapy in OTC Deficiency

Ultragenyx Completes FDA Rolling BLA Submission for DTX401 in Glycogen Storage Disease Type Ia (GSDIa)

Regeneron’s Evkeeza Receives the US FDA’s Approval to Treat Homozygous Familial Hypercholesterolemia in Younger Patients

Ultragenyx Completes Patient Enrolment in P-III (Aspire) Trial of GTX-102 for Angelman Syndrome

Ultragenyx Reports the Health Canada’s Approval of Evkeeza (evinacumab) to Treat Children with Homozygous Familial Hypercholesterolemia (HoFH)

Ultragenyx Reports the EC’s Approval of Evkeeza (evinacumab) to Treat 6-Months Old Children with Homozygous Familial Hypercholesterolemia (HoFH)

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