Core News Summary - The European Commission (EC) has extended the approval of Evkeeza (evinacumab) for treating children aged 6-months and older with homozygous familial hypercholesterolemia (HoFH), making it the first and only medicine approved in the EU for this age group [1][2] - Evkeeza is an ANGPTL3 inhibitor that reduces LDL-C levels and is now indicated for pediatric patients as young as 6-months old, addressing a critical unmet need in managing HoFH [1][5] Efficacy and Safety - Model-based extrapolation analysis predicts that pediatric patients aged 6-months to less than 5 years will experience a similar or higher magnitude of LDL-C reduction at week 24 compared to adults when receiving a 15 mg/kg dose every 4 weeks [2] - Compassionate use data for five patients aged 1-4 years showed clinically meaningful LDL-C reductions consistent with older patients, with no new safety concerns identified [2] Market and Availability - Evkeeza is now reimbursed and commercially available in the UK, US, Canada, Italy, Japan, the Netherlands, Spain, and Luxembourg, with early access schemes in 13 additional countries including Austria and France [2] - The treatment was initially approved for adults and adolescents aged 12+ in June 2021, with the indication extended to children aged 5-11 in December 2023 [2] Disease Background - HoFH is an ultrarare genetic disorder affecting 1 in 300,000 people globally and approximately 1,600 in the EU, characterized by dangerously high LDL-C levels (>400 mg/dL) and early cardiovascular risks [3][4] Product Mechanism - Evkeeza works by blocking the ANGPTL3 protein, which regulates cholesterol levels, and is administered via monthly infusions [5] Development and Commercialization - Evkeeza was discovered and developed by Regeneron Pharmaceuticals, Inc, with Ultragenyx responsible for its development and commercialization outside the US [7] Company Overview - Ultragenyx is a biopharmaceutical company focused on developing novel therapies for rare and ultrarare genetic diseases, with a diverse portfolio addressing high unmet medical needs [12][13]

Ultragenyx Pharmaceutical (RARE) announced that it has submitted a biologics license application (BLA) to the FDA for its UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA) under the accelerated approval pathway.Earlier this year, the FDA aligned with Ultragenyx, after a thorough evaluation, on the use of cerebral spinal fluid (CSF) heparan sulfate (HS) as a well-characterized biomarker to support an accelerated approval pathway for mucopolysaccharidoses ...

LONDON, Dec. 20, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialisation of novel therapies for rare and ultrarare genetic diseases, today announced that NHS England has implemented the commissioning of Evkeeza (evinacumab) following the National Institute for Health and Care Excellence (NICE) final guidance in September. The use of Evkeeza in eligible people aged 12 years and older will be routinely commissioned by ...

Company on track to initiate the Aurora study to evaluate GTX-102 in other Angelman syndrome genotypes and in other age groups in 2025NOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), today announced that the first patient has been dosed in the pivotal Phase 3 Aspire study (NCT06617429) evaluating the efficacy and safety of GTX-102, its investigational antisense oligonucleotide (ASO) for Angelman syndrome. "Initiation of patient dosing in our Phase 3 Aspire stu ...

If approved, UX111 would be the first approved therapy in the U.S. for Sanfilippo Syndrome Type ANOVATO, Calif., Dec. 19, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE) today announced the submission of a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA or the Agency) seeking accelerated approval for UX111 (ABO-102) AAV gene therapy as a treatment for patients with Sanfilippo syndrome type A (MPS IIIA). “The path to get a treatment to the point of a B ...

PLYMOUTH MEETING, Pa., Dec. 3, 2024 /PRNewswire/ -- Harmony Biosciences Holdings, Inc. (Nasdaq: HRMY), today announced that Harmony's management team will participate in a fireside chat at the upcoming Oppenheimer Movers in Rare Disease Summit in New York, NY on Thursday, December 12, 2024, at 9:00 a.m. ET.A webcast of the fireside chat will be available on the investor page of Harmony's website at https://ir.harmonybiosciences.com/.About Harmony BiosciencesHarmony Biosciences is a pharmaceutical company de ...

NOVATO, Calif., Nov. 26, 2024 (GLOBE NEWSWIRE) -- Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE), a biopharmaceutical company focused on the development and commercialization of novel therapies for serious rare and ultrarare genetic diseases, today announced participation in three upcoming investor conferences. Citi’s 2024 Global Healthcare Conference (Miami, FL) Tuesday, December 3, 2024, Emil Kakkis, M.D., Ph.D., CEO and President and Howard Horn, Chief Financial Officer, will participate in a fireside cha ...

MITIGATE Phase 3 Study Results Reinforce Promise of UPLIZNA® as the First Potential Treatment tor IgG4-RDPhase 4 AGILE Data Support Shortening KRYSTEXXA® Infusion TimeTHOUSAND OAKS, Calif., Nov. 14, 2024 /PRNewswire/ -- Amgen (NASDAQ:AMGN) today announced the presentation of new data across its rare disease portfolio and pipeline at the annual American College of Rheumatology (ACR) Convergence 2024 conference in Washington, D.C., Nov. 14-19, 2024. New data showcase reduction in disease activity by UPLIZNA® ...

Thank you for reading my research on Seeking Alpha. If you want to learn even more about my method and how I discover these investment opportunities, please check out my subscription marketplace service, Compounding Healthcare , and sign up for a free trial.It has been years since I have considered restarting a position in Ultragenyx Pharmaceutical Inc. (NASDAQ: RARE ) after having a string of big wins in gene therapy tickers following Biogen Inc.'s ( BIIB )He is the leader of the investing group Compoundin ...

Ultragenyx Pharmaceutical (RARE) incurred a third-quarter 2024 loss of $1.40 per share, narrower than the Zacks Consensus Estimate of a loss of $1.45. The company had reported a loss of $2.23 per share in the year-ago quarter.      Ultragenyx’s total revenues amounted to $139.5 million in the reported quarter, up 42% year over year. The top line surpassed the Zacks Consensus Estimate of $134 million on the back of higher product revenues.The company markets four drugs, namely Crysvita, Mepsevii, Dojolvi and ...