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Stoke Therapeutics and Biogen Initiate Phase 3 EMPEROR Trial for Zorevunersen in Dravet Syndrome

Orphan DrugRare Disease
  • Stoke Therapeutics and Biogen have dosed the first patient in the global Phase 3 EMPEROR study evaluating zorevunersen, an investigational antisense oligonucleotide for Dravet syndrome treatment.
  • The 52-week randomized, sham-controlled trial will enroll patients aged 2-18 with confirmed SCN1A gene variants, measuring seizure frequency reduction and cognitive improvements as primary endpoints.
  • Zorevunersen represents a potential first-in-class disease-modifying treatment targeting the underlying genetic cause of Dravet syndrome, which affects up to 38,000 people globally.
  • The drug has received FDA Breakthrough Therapy Designation and orphan drug status, with previous Phase 1/2 studies showing substantial seizure reductions and cognitive improvements.

A Double-blind Study Evaluating the Efficacy, Safety, and Tolerability of Zorevunersen in Patients With Dravet Syndrome

NCT06872125RecruitingPhase 3
Stoke Therapeutics, Inc
Posted 6/4/2025

Vicore's Buloxibutid Shows Promising Lung Function Improvement in Phase 2a IPF Trial

Rare Disease
  • Vicore Pharma's buloxibutid demonstrated significant lung function improvement in IPF patients, with an average FVC increase of 216ml over 36 weeks compared to the typical 180ml decline in untreated patients.
  • The angiotensin II type 2 receptor agonist was well-tolerated with no new drug-related adverse events reported during the 26-week treatment period, suggesting a favorable safety profile.
  • Final data from the Phase 2a AIR trial will be presented as a late-breaking presentation at the 2024 American Thoracic Society International Conference, with Vicore planning to advance to a Phase 2b ASPIRE trial.

Global Phase 3 Trial Tests Efzofitimod as Steroid-Sparing Treatment for Sarcoidosis

Rare Disease
  • Royal Papworth Hospital is participating in the world's first advanced phase global trial for sarcoidosis, testing if the protein efzofitimod can reduce steroid dependence in patients.
  • Sarcoidosis affects approximately 7,000 people in the UK, typically younger adults in their 30s and 40s, causing granulomas to form in the lungs and potentially other organs.
  • The trial involves about 200 patients across Europe, North America, and Asia, aiming to improve quality of life by reducing long-term steroid side effects like weight gain, diabetes risk, and bone weakness.

FDA Analysis Reveals Decade of Growth in Rare Pediatric Disease Drug Development Through Priority Review Voucher Program

Rare DiseaseFDA Approval
  • The FDA's Rare Pediatric Disease Priority Review Voucher program granted 569 designations between 2013-2022, targeting 245 unique rare pediatric diseases across neurology, metabolism, and oncology therapeutic areas.
  • Gene therapies comprised 28% of all designations, reflecting the genetic nature of most rare diseases, while 38% of designations were supported by clinical data indicating advanced development stages.
  • The program awarded 38 priority review vouchers worth approximately $100 million each, providing crucial non-dilutive funding to rare disease drug developers.
  • A five-fold surge in designations occurred in 2020 when the program was set to expire, highlighting the uncertainty created by periodic reauthorization requirements.

FDA Grants Orphan Drug Designation to Tigilanol Tiglate for Soft Tissue Sarcoma Treatment

Rare DiseaseOncology
  • The FDA has awarded orphan drug designation to tigilanol tiglate (Stelfonta), an intratumoral injection, for treating soft tissue sarcoma, a rare cancer affecting both adults and children.
  • The designation provides QBiotics with important regulatory incentives including tax credits, user fee exemptions, and potential seven years of market exclusivity following approval.
  • Tigilanol tiglate is currently being evaluated in a phase 2 trial at Memorial Sloan Kettering Cancer Center, with preliminary phase 1 data showing responses in 6 of 22 patients.
  • Soft tissue sarcomas comprise more than 80 subtypes with unfavorable prognosis in advanced stages, highlighting the urgent need for new treatment options in this rare disease area.

A Clinical Study to Investigate the Efficacy of Intratumoral Tigilanol Tiglate in Soft Tissue Sarcoma

NCT05755113RecruitingPhase 2
QBiotics Group Limited
Posted 4/13/2023

ADI-PEG20 Quadruples Three-Year Survival in Phase 3 Mesothelioma Trial

OncologyRare Disease
  • A phase 3 trial of ADI-PEG20 combined with chemotherapy increased median survival by 1.6 months and quadrupled three-year survival rates in 249 patients with pleural mesothelioma.
  • The drug works by depleting arginine levels in the bloodstream, starving tumor cells that cannot manufacture their own arginine due to missing ASS1 protein.
  • This represents the first successful combination therapy breakthrough for mesothelioma in 20 years, offering new hope for patients with one of the world's worst cancer survival rates.
  • The international ATOMIC-meso trial was conducted across 43 centers in five countries between 2017 and 2021, demonstrating the treatment was well tolerated with no new safety signals.

Novartis Secures $1.3 Billion Deal with Chong Kun Dang for HDAC6 Inhibitor Targeting CMT Disease

Orphan DrugRare Disease
  • Novartis has signed a $1.3 billion licensing agreement with Korean biotech Chong Kun Dang Pharmaceutical for CKD-510, an HDAC6 inhibitor with FDA orphan drug designation for Charcot-Marie-Tooth disease.
  • The deal includes an $80 million upfront payment plus $1.2 billion in potential milestone payments, with Novartis gaining exclusive global rights to develop and commercialize the drug outside Korea.
  • This acquisition follows Novartis' recent procurement of DTX-1252, another CMT1A therapeutic candidate, signaling the company's strategic expansion into rare neurological disease treatments.

UK Approves World's First CRISPR Gene Therapy for Sickle Cell Disease and β-Thalassemia

Drug ApprovalRare Disease
  • The UK has made a landmark decision to approve the world's first CRISPR-based gene therapy, developed by Vertex Pharmaceuticals, for treating sickle cell disease and β-thalassemia.
  • This one-time treatment offers potential transformation of patient lives by addressing the genetic root cause of these blood disorders, marking a historic milestone in gene editing technology.
  • Despite its therapeutic promise, concerns remain about the high cost of the treatment and questions about accessibility for patients in need, particularly in regions with high disease prevalence.

BEACON: A Study Evaluating the Safety and Efficacy of BEAM-101 in Patients With Severe Sickle Cell Disease

NCT05456880RecruitingPhase 1
Beam Therapeutics Inc.
Posted 8/30/2022

Elafibranor Shows Significant Efficacy in Phase III Trial for Primary Biliary Cholangitis

Rare Disease
  • Ipsen and GENFIT's Phase III ELATIVE trial demonstrated that elafibranor achieved a 47% placebo-adjusted difference in biochemical response rates, with 51% of patients on elafibranor versus 4% on placebo meeting the primary endpoint.
  • Only patients receiving elafibranor achieved alkaline phosphatase normalization at 52 weeks (15% vs 0% placebo), with rapid ALP reductions observed as early as week 4 and sustained throughout the study.
  • The dual PPAR α,δ agonist showed potential for improving pruritus symptoms and was well-tolerated, with data supporting regulatory submissions worldwide for this rare autoimmune liver disease.

Study of Elafibranor in Patients With Primary Biliary Cholangitis (PBC)

NCT04526665Active, Not RecruitingPhase 3
Ipsen
Posted 9/24/2020

Novartis' Ianalumab Achieves Primary Endpoint in Phase III ITP Trial, Offering Potential for Extended Treatment-Free Periods

Monoclonal AntibodyOrphan DrugRare Disease
  • Novartis announced positive Phase III results for ianalumab in immune thrombocytopenia, with the drug significantly prolonging time to treatment failure compared to placebo when combined with eltrombopag.
  • Patients receiving ianalumab experienced significantly higher rates of sustained platelet count improvements at six months, the key secondary endpoint of the VAYHIT2 study.
  • The monoclonal antibody demonstrated a consistent safety profile with no new safety signals, and could potentially offer long-term disease control through just four once-monthly doses.
  • Regulatory submissions are planned for 2027 alongside results from the ongoing first-line ITP trial, with ianalumab having received Orphan Drug Designation from both FDA and EMA.

A Study of Efficacy and Safety of Ianalumab Versus Placebo in Addition to Eltrombopag in Primary Immune Thrombocytopenia Patients Who Failed Steroids

NCT05653219Active, Not RecruitingPhase 3
Novartis Pharmaceuticals
Posted 2/2/2023

Safety, Efficacy and Tolerability of Ianalumab Versus Placebo, Combination With SoC Therapy, in Participants With Active Lupus Nephritis

NCT05126277RecruitingPhase 3
Novartis Pharmaceuticals
Posted 7/14/2022

Three-arm Study to Assess Efficacy and Safety of Ianalumab (VAY736) in Patients With Active Sjogren's Syndrome

NCT05349214Active, Not RecruitingPhase 3
Novartis Pharmaceuticals
Posted 8/4/2022

A Clinical Study to Evaluate Ianalumab in Participants With Diffuse Cutaneous Systemic Sclerosis

NCT06470048RecruitingPhase 2
Novartis Pharmaceuticals
Posted 10/9/2024

Two-arm Study to Assess Efficacy and Safety of Ianalumab (VAY736) in Patients With Active Sjogren's Syndrome

NCT05350072Active, Not RecruitingPhase 3
Novartis Pharmaceuticals
Posted 7/28/2022

Study of Ianalumab Versus Placebo in Addition to First-line Corticosteroids in Primary Immune Thrombocytopenia (ITP)

NCT05653349Active, Not RecruitingPhase 3
Novartis Pharmaceuticals
Posted 3/6/2023

Phase 3 Study to Evaluate Two Regimens of Ianalumab on Top of Standard-of-care Therapy in Patients With Systemic Lupus Erythematosus (SIRIUS-SLE 1)

NCT05639114RecruitingPhase 3
Novartis Pharmaceuticals
Posted 3/2/2023

A Study of Efficacy and Safety of Ianalumab in Previously Treated Patients With Warm Autoimmune Hemolytic Anemia

NCT05648968RecruitingPhase 3
Novartis Pharmaceuticals
Posted 12/30/2022

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