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BridgeBio Secures $300 Million Royalty Financing to Support ATTR-CM Drug Launch

Drug ApprovalRare Disease
  • BridgeBio Pharma secured $300 million in upfront financing by selling 60% of European royalties for BEYONTTRA to HealthCare Royalty and Blue Owl Capital.
  • The transaction monetizes royalties on the first $500 million of annual BEYONTTRA net sales in Europe with a 1.45x cap on total investor payments.
  • Acoramidis demonstrated rapid clinical benefits in Phase 3 trials, showing 42% reduction in cardiovascular events and 50% reduction in cardiovascular hospitalizations.
  • The drug is approved as Attruby in the US and BEYONTTRA in Europe for treating transthyretin amyloidosis with cardiomyopathy (ATTR-CM).

Investor Challenges Avadel Board Over Alleged Lumryz Launch Mismanagement

Drug Approval
  • ASL Strategic Value Fund, holding $15 million in Avadel shares, plans to push for board removal citing mismanagement of Lumryz drug launch.
  • The investment firm alleges the mishandled launch cost Avadel hundreds of millions in potential revenue for the narcolepsy treatment.
  • ASL is calling for Avadel to hire an investment bank and explore strategic alternatives including a potential sale.
  • Lumryz received FDA approval last year for treating narcolepsy symptoms in children aged 7 and older, targeting a disorder affecting sleep-wake cycle regulation.

FDA Approves Voranigo (Vorasidenib) as First Targeted Therapy for IDH-Mutant Glioma

Drug ApprovalTargeted Therapy
  • The FDA approved Voranigo (vorasidenib) on August 6, 2024, as the first targeted treatment for Grade 2 IDH-mutant astrocytoma or oligodendroglioma in patients 12 years and older.
  • Phase 3 INDIGO trial results showed Voranigo significantly extended progression-free survival to 27.7 months compared to 11.1 months with placebo.
  • The drug works by blocking mutant IDH1 and IDH2 enzymes, reducing tumor activity and crossing the blood-brain barrier to effectively treat brain tumors.
  • Common side effects include tiredness (37%), COVID-19 (33%), and muscle or joint pain (26%), with liver function monitoring required during treatment.

AD HOC Trial: Artificial Intelligence-Based Drug Dosing In Hepatocellular Carcinoma

NCT05669339SuspendedPhase 2
University of Florida
Posted 12/2/2024

Vorasidenib in Combination With Temozolomide (TMZ) in IDH-mutant Glioma

NCT06478212RecruitingPhase 1
Institut de Recherches Internationales Servier
Posted 1/22/2025

Study of AG-120 and AG-881 in Subjects With Low Grade Glioma

NCT03343197CompletedPhase 1
Institut de Recherches Internationales Servier
Posted 3/20/2018

Study of Vorasidenib (AG-881) in Participants With Residual or Recurrent Grade 2 Glioma With an IDH1 or IDH2 Mutation (INDIGO)

NCT04164901Active, Not RecruitingPhase 3
Institut de Recherches Internationales Servier
Posted 1/5/2020

ViCToRy: Vorasidenib in Combination With Tumor Specific Peptide Vaccine for Recurrent IDH1 Mutant Lower Grade Gliomas

NCT05609994Not Yet RecruitingPhase 1
Katy Peters, MD, PhD
Posted 6/1/2025

Study of Orally Administered AG-881 in Patients With Advanced Solid Tumors, Including Gliomas, With an IDH1 and/or IDH2 Mutation

NCT02481154CompletedPhase 1
Institut de Recherches Internationales Servier
Posted 6/1/2015

FDA Approves Gamifant as First Treatment for Macrophage Activation Syndrome in Still's Disease

Drug ApprovalMonoclonal AntibodyRare Disease
  • The FDA has approved Gamifant (emapalumab-lzsg) as the first-ever treatment for adults and children with macrophage activation syndrome in Still's disease, marking a significant breakthrough for patients with this life-threatening condition.
  • Clinical trials demonstrated that 54% of patients achieved complete response at week 8, with 82% reaching clinical MAS remission, providing new hope for managing severe hyperinflammation.
  • This approval addresses a critical unmet medical need for patients with MAS, offering an alternative to high-dose glucocorticoids and potentially reducing reliance on conventional therapies with significant side effects.

Phase 3 Efficacy and Safety Study of GTX-102 in Pediatric Subjects With AS

NCT06617429RecruitingPhase 3
Ultragenyx Pharmaceutical Inc
Posted 12/3/2024

Evaluate Efficacy, Safety and Tolerability, PK and PD of Emapalumab in Children and Adults With MAS in Still's or SLE

NCT05001737CompletedPhase 3
Swedish Orphan Biovitrum
Posted 12/15/2021

A Study to Investigate the Safety and Efficacy of Emapalumab, an Anti-IFN-gamma mAb in Patients With Systemic Juvenile Idiopathic Arthritis (sJIA) or Adult-onset Still's Disease (AOSD) Developing Macrophage Activation Syndrome/Secondary HLH (MAS/sHLH)

NCT03311854CompletedPhase 2
Swedish Orphan Biovitrum
Posted 2/20/2018

FDA-Approved Tovorafenib Offers New Treatment Option for Pediatric Low-Grade Glioma with BRAF V600 Mutations

Targeted TherapyDrug Approval
  • Tovorafenib (Ojemda) is approved for treating low-grade glioma that has returned after previous treatments failed, specifically targeting tumors with BRAF V600 mutations.
  • The oral medication is available for adults and children as young as 6 months old, administered once weekly as either tablets or oral suspension.
  • Treatment requires genetic testing to confirm BRAF V600 mutations before initiation and regular monitoring for potential side effects including bleeding, liver problems, and growth impacts in children.
  • Common side effects include fatigue, gastrointestinal symptoms, cold-like symptoms, and skin reactions, with serious adverse events requiring immediate medical attention.

Study for Patients With Hairy Cell Leukemia to Test the Targeted Agent Tovorafenib in Combination With Rituximab and Compare the Combination With Current Standard Treatment

NCT06965114Not Yet RecruitingPhase 1
National Cancer Institute (NCI)
Posted 9/30/2025

A Study With Tovorafenib (DAY101) as a Treatment Option for Progressive, Relapsed, or Refractory Langerhans Cell Histiocytosis

NCT05828069RecruitingPhase 2
National Cancer Institute (NCI)
Posted 3/28/2024

FDA Maintains Record-Breaking Approval Pace Despite Organizational Upheaval, Highlighting Three Breakthrough Therapies

Drug ApprovalOncology
  • The FDA has approved 84 drugs in 2025 so far, marking the second-highest total for this period despite major organizational changes including 3,500 staff layoffs.
  • Gilead's Yeztugo represents a potential game-changer for HIV prevention with 100% efficacy in phase 3 trials and twice-yearly dosing, though priced at $28,218 annually.
  • Daiichi Sankyo's ADC portfolio expansion continues with Datroway's lung cancer approval, while GSK's Blujepa becomes the first new urinary tract infection antibiotic in three decades.

Swissmedic Launches Fast-Track Clinical Trial Review to Accelerate Patient Access to Life-Saving Treatments

Drug Approval
  • Swissmedic introduces a pilot fast-track procedure for clinical trial applications, reducing review times from 30 to 20 days for known investigational drugs and from 60 to 40 days for first-in-human studies.
  • The accelerated process prioritizes studies addressing high medical need where no approved treatment options exist in Switzerland, positioning the country ahead of EU standards.
  • The pilot program runs from July 2025 through 2026, with potential permanent implementation by 2027, while maintaining all safety requirements and regulatory standards.
  • This initiative aims to strengthen Switzerland's position as a leading clinical research location and provide patients faster access to innovative, potentially life-saving therapies.

FDA Approves Streamlined Access for Bristol Myers Squibb CAR T Cell Therapies

CAR-TDrug Approval
  • The FDA has approved label updates for Bristol Myers Squibb's CAR T cell therapies Breyanzi and Abecma, reducing patient monitoring requirements and removing Risk Evaluation and Mitigation Strategy (REMS) programs.
  • Key changes include reducing driving restrictions from 8 weeks to 2 weeks post-treatment and decreasing the requirement to stay near healthcare facilities from 4 weeks to 2 weeks following infusion.
  • These updates reflect growing real-world evidence from over 30,000 patients treated with CAR T cell therapy, with studies showing most serious adverse events occur within the first two weeks of infusion.
  • Currently only about 2 in 10 eligible patients receive cell therapy treatment due to logistical and geographic barriers, which these label changes aim to address.

Study Evaluating Safety and Efficacy of JCAR017 in Subjects With Relapsed or Refractory Chronic Lymphocytic Leukemia (CLL) or Small Lymphocytic Lymphoma (SLL)

NCT03331198RecruitingPhase 1
Juno Therapeutics, a Subsidiary of Celgene
Posted 11/27/2017

A Study to Evaluate the Efficacy and Safety of JCAR017 in Adult Subjects With Relapsed or Refractory Indolent B-cell Non-Hodgkin Lymphoma (NHL)

NCT04245839RecruitingPhase 2
Celgene
Posted 7/14/2020

A Study to Compare the Efficacy and Safety of JCAR017 to Standard of Care in Adult Subjects With High-risk, Transplant-eligible Relapsed or Refractory Aggressive B-cell Non-Hodgkin Lymphomas

NCT03575351CompletedPhase 3
Celgene
Posted 10/23/2018

Lisocabtagene Maraleucel (JCAR017) as Second-Line Therapy (TRANSCEND-PILOT-017006)

NCT03483103CompletedPhase 2
Juno Therapeutics, a Subsidiary of Celgene
Posted 7/27/2018

FDA Rejects Accelerated Approval for Edgewise's Sevasemten in Becker Muscular Dystrophy

Drug Approval
  • The FDA declined to consider Edgewise Therapeutics' sevasemten for accelerated approval in Becker muscular dystrophy, citing insufficient data from the Phase II CANYON trial.
  • The agency affirmed that North Star Ambulatory Assessment scores can serve as a clinically meaningful endpoint for traditional approval, providing a clear regulatory pathway forward.
  • Edgewise must now rely on its ongoing Phase III GRAND CANYON trial, which is expected to read out in Q4 2026 and is designed to demonstrate statistical significance versus placebo.
  • The company's stock dropped 10% following the announcement, though analysts noted the decision was not unexpected given the FDA's tightened stance on accelerated approval requirements.

First European Patient Receives Breakthrough Treatment for Ultra-Rare APDS Immune Disorder

Rare DiseaseTargeted TherapyDrug ApprovalOrphan Drug
  • Mary Catchpole, 19, becomes the first European patient to receive leniolisib (Joenja), a newly approved targeted treatment for activated PI3-kinase delta syndrome (APDS), a rare inherited immune disorder.
  • The drug works by inhibiting an overactive enzyme that disrupts immune function, offering a potential cure for patients who previously faced lifelong infections and invasive treatments.
  • APDS was discovered by Cambridge researchers in 2013 with help from Catchpole's family, who lost four members to the condition before this breakthrough treatment became available.
  • The NHS approved leniolisib at a list price of £352,000 per year with a confidential discount, potentially benefiting up to 50 patients over age 12 in England.
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