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FUJIFILM's FF-10832 Receives FDA Orphan Drug Designation for Biliary Tract Cancer Treatment

OncologyRare Disease
  • The FDA has granted orphan drug designation to FF-10832, FUJIFILM's investigational liposomal formulation of gemcitabine, for treating biliary tract cancer.
  • Phase 1 study results presented at ASCO 2025 demonstrated that FF-10832 is well tolerated and shows anti-tumor activity in patients with advanced biliary tract cancer.
  • The novel liposomal formulation is designed to enhance anti-tumor activity by prolonging plasma half-life and improving targeted delivery to tumors.
  • Biliary tract cancer affects approximately 16,000 new patients annually in the U.S., with most presenting with unresectable or metastatic disease and poor survival rates.

A Phase 1 Dose-escalation Study of FF-10832 for Treatment of Solid Tumors Including Biliary Tract Cancer

NCT03440450Active, Not RecruitingPhase 1
Fujifilm Pharmaceuticals U.S.A., Inc.
Posted 3/22/2018

A Study to Evaluate Safety, Efficacy of FF-10832 in Combo With Pembrolizumab in Urothelial & Non-small Cell Lung Cancer

NCT05318573RecruitingPhase 2
Fujifilm Pharmaceuticals U.S.A., Inc.
Posted 6/1/2022

Bezuclastinib Achieves 87% Response Rate in Pivotal Trial for Non-Advanced Systemic Mastocytosis

Rare DiseasePrecision MedicineOncology
  • Cogent Biosciences' bezuclastinib demonstrated superior symptom improvement with a placebo-adjusted difference of 8.91 points in total symptom score at 24 weeks (p=0.0002) in the SUMMIT trial.
  • The drug showed remarkable efficacy on mast cell burden, with 87.4% of patients achieving at least 50% reduction in serum tryptase compared to 0% in the placebo group.
  • Bezuclastinib exhibited a favorable safety profile with most adverse events being low-grade, supporting its potential for chronic use in systemic mastocytosis patients.
  • The company plans to submit a New Drug Application to the FDA by the end of 2025 based on these positive results from the registration-directed trial.

(Summit) a Study to Evaluate the Efficacy and Safety of CGT9486 Versus Placebo in Patients with Indolent or Smoldering Systemic Mastocytosis

NCT05186753Active, Not RecruitingPhase 2
Cogent Biosciences, Inc.
Posted 6/27/2022

Orchard Therapeutics Completes Enrollment in Pivotal Gene Therapy Trial for Hurler Syndrome

Rare Disease
  • Orchard Therapeutics has completed enrollment in the HURCULES registrational trial for OTL-203, an investigational hematopoietic stem cell gene therapy for MPS-IH (Hurler syndrome), nearly one year ahead of schedule.
  • The randomized controlled trial compares OTL-203 to standard allogeneic stem cell transplant across four clinical sites in the U.S. and Europe, with primary analysis anticipated two years post-treatment.
  • Previous proof-of-concept data showed robust metabolic correction and improvements in cognitive, motor, skeletal, ocular and auditory health, addressing clinical manifestations not fully covered by current treatments.
  • MPS-IH affects approximately 60% of children born with MPS-I, a rare disease occurring in 1 in 100,000 live births, with patients rarely surviving past age 10 when untreated.

A Study to Investigate the Efficacy and Safety of OTL-203 in Subjects With MPS-IH Compared With Standard of Care With Allogeneic HSCT

NCT06149403Active, Not RecruitingPhase 3
Orchard Therapeutics
Posted 12/11/2023

BioCryst Pharmaceuticals Appoints Babar Ghias as CFO and Head of Corporate Development

Rare Disease
  • BioCryst Pharmaceuticals has appointed Babar Ghias as chief financial officer and head of corporate development, bringing extensive deal-making experience from rare disease companies.
  • Ghias joins from AvenCell Therapeutics where he served as CFO since 2022, and previously raised over $1 billion in capital across multiple biotechnology companies.
  • The appointment comes as BioCryst seeks to deploy capital and accelerate growth driven by commercial momentum from ORLADEYO, their oral treatment for hereditary angioedema.
  • CEO Jon Stonehouse emphasized that Ghias's expertise in capital deployment and operational experience are essential for the company's sustainable growth strategy into the next decade.

ALS Community Files Citizens' Petition Seeking FDA Approval for NurOwn Stem Cell Therapy

Rare Disease
  • A coalition of ALS patients and families has filed a 309-page Citizens' Petition with the FDA requesting approval of NurOwn, a neurotrophically-enhanced stem cell therapy developed by BrainStorm Cell Therapeutics.
  • The petition presents unprecedented survival data showing 100% five-year survival in NurOwn's Expanded Access Program participants compared to 20% in natural ALS history, with median tracheostomy-free survival of 7 years.
  • NurOwn demonstrated statistically significant changes in 23 cerebrospinal fluid biomarkers and up to 85% slowing of ALS progression, supported by real-world evidence from clinical trials and expanded access programs.
  • The petition argues NurOwn meets approval thresholds for traditional, accelerated, or conditional approval pathways based on survival data, respiratory function preservation, and biomarker evidence.

Eleva Advances Factor H Therapy CPV-104 into First-in-Human Trial for Rare Kidney Disease C3 Glomerulopathy

Rare Disease
  • Eleva has administered the first dose of CPV-104, a recombinant human complement Factor H therapy, to healthy volunteers in a Phase 1 clinical study for C3 Glomerulopathy (C3G).
  • The therapy targets the abnormal regulation of the complement system that causes C3G, a rare renal disease, and has received Orphan Drug Designation in the European Union.
  • Preclinical data published in Frontiers in Immunology demonstrated CPV-104's ability to normalize serum C3 levels and rapidly degrade C3 deposits in the kidney.
  • This milestone represents Eleva's second proprietary program to advance into clinical trials, with the company also developing CPV-104 for dry AMD as a second indication.

Amicus Therapeutics Secures Japan Approval for Pombiliti + Opfolda Combination Therapy for Late-Onset Pompe Disease

Drug ApprovalRare Disease
  • Japan's Ministry of Health, Labour and Welfare has approved Pombiliti + Opfolda for treating adult patients with late-onset Pompe disease, expanding global access to this innovative therapy.
  • The approval was based on data from the Phase 3 PROPEL study, which uniquely studied both treatment-naïve and treatment-experienced patients in a controlled setting.
  • Pombiliti + Opfolda is now approved in seven major markets including the US, EU, UK, Canada, Australia, Switzerland, and Japan.
  • The two-component therapy combines a recombinant human GAA enzyme with enhanced muscle cell uptake and an oral enzyme stabilizer designed to maintain enzyme activity in blood.

Sanofi Advances Multiple Phase 2 Trials Targeting Rare Kidney Diseases and Ulcerative Colitis

Rare Disease
  • Sanofi has initiated a Phase 2a clinical trial evaluating three experimental drugs—Frexalimab, SAR442970, and Rilzabrutinib—for treating primary focal segmental glomerulosclerosis (FSGS) and minimal change disease (MCD) in patients aged 16 to 75 years.
  • The kidney disease study employs a randomized, double-blind, placebo-controlled design with quadruple masking to assess changes in proteinuria and remission rates of nephrotic syndrome.
  • Separately, Sanofi is conducting a Phase 2 dose-ranging study of SAR441566, an oral tablet, for moderate-to-severe ulcerative colitis to evaluate clinical remission rates.
  • Both studies utilize rigorous randomized, double-blind methodologies with placebo controls and could significantly impact Sanofi's competitive position in treating these challenging conditions.

Gene Therapy for Fabry Disease Shows Promise with $3.7 Million Cost Savings in Early-Stage Trial

Rare Disease
  • A small-scale gene therapy study for Fabry disease enabled three of five male patients to discontinue costly enzyme-replacement therapy, generating $3.7 million in savings against $4 million research costs.
  • The experimental treatment uses bone marrow stem cells to deliver replacement copies of faulty genes, with patients maintaining elevated enzyme production five years post-treatment.
  • Researchers plan to expand the study to 25-30 patients including women over two to three years, as the current therapy requires bi-weekly treatments costing $300,000 annually per patient.
  • The gene therapy demonstrated a favorable safety profile with only two minor side effects related to preparatory chemotherapy rather than the gene therapy itself.

Bio-Thera's BAT4406F Achieves Early Trial Success in Rare Neurological Disorder NMOSD

Monoclonal AntibodyRare Disease
  • Bio-Thera Solutions' BAT4406F, an ADCC-enhanced anti-CD20 monoclonal antibody, demonstrated statistically significant efficacy in treating neuromyelitis optica spectrum disorder (NMOSD) during interim analysis of its pivotal Phase II/III trial.
  • The Independent Data Monitoring Committee recommended early termination of the 45-site Chinese trial due to compelling efficacy results that met pre-defined superiority criteria.
  • Bio-Thera has closed patient enrollment ahead of schedule and will begin preparing regulatory approval submissions to China's NMPA for this rare autoimmune disease affecting optic nerves and spinal cord.
  • The breakthrough addresses a critical unmet medical need for NMOSD patients, who face devastating outcomes including permanent blindness, paralysis, and progressive disability with limited current treatment options.

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