Evinacumab

Ultragenyx Launches Evkeeza® (evinacumab for injection) in Canada for the Treatment of Homozygous Familial Hypercholesterolemia (HoFH)

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Monday, September 25, 2023

Evkeeza® was approved by Health Canada as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with homozygous familial hypercholesterolemia (HoFH) and will be commercially available in Canada effective 25-November-2023.

Key Points: 
  • Evkeeza® was approved by Health Canada as an adjunct to diet and other low-density lipoprotein cholesterol (LDL-C) lowering therapies for the treatment of adult and pediatric patients aged 5 years and older with homozygous familial hypercholesterolemia (HoFH) and will be commercially available in Canada effective 25-November-2023.
  • Evkeeza is a first-of-its-kind treatment for HoFH and is the first monoclonal antibody inhibiting the angiopoietin-like 3 protein (ANGPTL3).
  • The treatment is now available to prescribe for patients with HoFH in Canada.
  • “The approval and launch of Evkeeza in Canada for the treatment of homozygous familial hypercholesterolemia exemplifies our commitment to bring innovative therapies for people living with rare and ultrarare genetic diseases,” said Monty Keast, Vice President and General Manager at Ultragenyx Canada.

FDA Approves First-in-class Evkeeza® (evinacumab-dgnb) for Young Children with Ultra-rare Form of High Cholesterol

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Wednesday, March 22, 2023

TARRYTOWN, N.Y., March 22, 2023 (GLOBE NEWSWIRE) -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the U.S. Food and Drug Administration (FDA) has extended the approval of Evkeeza® (evinacumab-dgnb) as an adjunct to other lipid-lowering therapies to treat children aged 5 to 11 with homozygous familial hypercholesterolemia (HoFH). Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for children as young as 5 years old to control dangerously high levels of low-density lipoprotein cholesterol (LDL-C) caused by HoFH. Evkeeza was initially approved as an adjunct to other lipid-lowering therapies in those aged 12 years and older with HoFH in February 2021.

Key Points: 
  • Evkeeza is the first angiopoietin-like 3 (ANGPTL3) inhibitor treatment indicated for children as young as 5 years old to control dangerously high levels of low-density lipoprotein cholesterol (LDL-C) caused by HoFH.
  • With this FDA approval, the HoFH community now has a much-needed treatment for young children, potentially making it possible for many to achieve recommended LDL-C levels much earlier in the course of this rare disease.
  • “Guidelines recommend screening all children at high risk for homozygous familial hypercholesterolemia starting at age 2.
  • Significant reductions were also observed in other key secondary endpoints including levels of apolipoprotein B (ApoB), non-high-density lipoprotein cholesterol (non-HDL-C) and total cholesterol.

Evkeeza® (evinacumab-dgnb) sBLA for Children with Ultra-rare Inherited Form of High Cholesterol Accepted for FDA Priority Review

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Wednesday, November 30, 2022

TARRYTOWN, N.Y., Nov. 30, 2022 /PRNewswire/ -- Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced the U.S. Food and Drug Administration (FDA) has accepted for Priority Review the supplemental Biologics License Application (sBLA) for Evkeeza® (evinacumab-dgnb) as an adjunct to other lipid-lowering therapies to treat children aged 5 to 11 years with homozygous familial hypercholesterolemia (HoFH). The FDA target action date is March 30, 2023.

Key Points: 
  • HoFH is an ultra-rare inherited condition that affects approximately 1,300 patients in the U.S. and is the most severe form of familial hypercholesterolemia (FH).
  • The sBLA is supported by data from a three-part trial evaluating Evkeeza in children aged 5 to 11 years with HoFH.
  • The trial met its primary endpoint, showing children who added Evkeeza to other lipid-lowering therapies reduced their LDL-C by 48% at week 24 on average.
  • Part A (n=6) was a Phase 1b trial designed to assess the pharmacokinetics (PK), safety and tolerability of Evkeeza.

Ultragenyx Reports First Quarter 2022 Financial Results and Corporate Update

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Thursday, May 5, 2022

NOVATO, Calif., May 05, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter ended March 31, 2022 and reaffirmed its financial guidance for the year.

Key Points: 
  • Ultragenyx received rights to commercialize and distribute Evkeeza in countries outside the U.S. in January 2022.
  • A dosing update on the Phase 2 portion of the Orbit study and transition to Phase 3 is expected in the second half of 2022.
  • ET to discuss the first quarter 2022 financial results and provide a corporate update.
  • Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyxs Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/mycompany/).

Ultragenyx Reports Fourth Quarter and Full Year 2021 Financial Results and Corporate Update

Retrieved on: 
Thursday, February 10, 2022

NOVATO, Calif., Feb. 10, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today reported its financial results for the quarter and full year ended December 31, 2021 and reaffirmed its financial guidance for 2022.

Key Points: 
  • The first patients have been dosed in the Phase 3 study of DTX401 following an approximate 4- to 8-week baseline screening period.
  • Ultragenyx expects to initiate the Phase 3 eNH3ance study of DTX301 in patients with OTC in the first half of 2022.
  • ET to discuss the fourth quarter and year 2021 financial results and provide a corporate update.
  • Financial and other information about Ultragenyx is routinely posted and is accessible on Ultragenyxs Investor Relations website (https://ir.ultragenyx.com/) and LinkedIn website (https://www.linkedin.com/company/ultragenyx-pharmaceutical-inc-/mycompany/).

Ultragenyx Reports Preliminary 2021 Revenue and 2022 Revenue Guidance for Crysvita® in Ultragenyx Territories* And Dojolvi® Globally; Provides Pipeline Updates and 2022 Milestones

Retrieved on: 
Monday, January 10, 2022

NOVATO, Calif., Jan. 10, 2022 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel products for serious rare and ultra-rare genetic diseases, today reported preliminary unaudited 2021 Crysvita® revenue in Ultragenyx territories and Dojolvi® global revenue, cash and investments at year end 2021, and provided 2022 revenue guidance for Crysvita in Ultragenyx territories and Dojolvi globally.

Key Points: 
  • Crysvita revenue in Ultragenyx territories* for the year ended December 31, 2021 is approximately $191 million to $193 million.
  • For 2022, Crysvita revenue in the Ultragenyx territories is estimated to be between $250 million and $260 million, representing growth of 33% year-over-year at the mid-point of our guidance.
  • Dojolvi revenue for the year ended December 31, 2021 is approximately $38 million to $40 million.
  • The preliminary revenue results are based on management's initial analysis of operations for the quarter and year ended December 31, 2021.