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FDA Upgrades Xinnate's TCP-25 Trial for Epidermolysis Bullosa to Registrational Status

Rare Disease
  • Following a successful Type C meeting with the FDA, Xinnate's upcoming STEP trial for TCP-25 in Epidermolysis Bullosa patients has been upgraded to serve as a registrational trial.
  • TCP-25, an immunomodulatory peptide with dual-action capabilities targeting both inflammation and bacterial infection, previously received Orphan Drug Designation from the FDA for EB treatment.
  • The STEP trial, planned to begin in 2025, is designed as an international multicenter, randomized, double-blind, placebo-controlled study to assess TCP-25's efficacy and safety in dystrophic and junctional EB patients.

Real-World Data Confirms Trikafta's Long-Term Efficacy as Gold Standard in Cystic Fibrosis Treatment

Real World EvidenceRare Disease
  • New real-world evidence presented at ATS 2025 validates Trikafta (elexacaftor/tezacaftor/ivacaftor) as an effective long-term therapy for cystic fibrosis patients with at least one F508del mutation.
  • The study by Sutharsan and colleagues provides valuable insights into laboratory markers while demonstrating sustained clinical benefits across various outcomes in this patient population.
  • These findings further establish Trikafta's position as the gold standard treatment in long-term cystic fibrosis care, reinforcing its therapeutic value for patients.

Be Biopharma Initiates First-in-Human Trial of CRISPR-Based Gene Therapy for Haemophilia B

Orphan DrugRare Disease
  • Be Biopharma has dosed the first patient in the BeCoMe-9 Phase I/II trial of BE-101, marking the first clinical application of B Cell Medicines technology for haemophilia B treatment.
  • BE-101 uses CRISPR/Cas9 gene editing to insert the human Factor IX gene into patients' own B cells, designed to continuously secrete Factor IX without requiring preconditioning or immunosuppression.
  • The two-part trial will enroll up to 24 subjects with moderately severe to severe haemophilia B, with participants monitored for 52 weeks to evaluate safety and clinical activity.
  • The therapy has received FDA fast-track and orphan drug designations, positioning it as a potentially transformative treatment option for this rare bleeding disorder.

BeCoMe-9: A Clinical Study of BE-101 for the Treatment of Adults With Moderately Severe or Severe Hemophilia B

NCT06611436RecruitingPhase 1
Be Biopharma
Posted 12/4/2024

FDA Grants Orphan Drug Designation to Repair Biotechnologies' mRNA Therapy for Rare Cholesterol Disorder

Rare Disease
  • The FDA has granted Orphan Drug Designation to REP-0003, a novel mRNA therapy developed by Repair Biotechnologies for treating homozygous familial hypercholesterolemia (HoFH), a rare condition causing accelerated atherosclerosis.
  • REP-0003 works by selectively clearing harmful excess free cholesterol inside cells while preserving essential cholesterol, showing promising results in preclinical studies including plaque regression and improved liver function.
  • The designation provides Repair Biotechnologies with seven years of market exclusivity upon approval, tax credits for clinical trials, waived FDA fees, and potential fast-track pathways as they prepare for first human trials in 2026.

Prime Medicine Reports Breakthrough Clinical Data for First Prime Editing Therapy in Chronic Granulomatous Disease

Rare Disease
  • Prime Medicine's PM359, the first Prime Editing therapy administered to humans, demonstrated rapid restoration of NADPH oxidase activity in a patient with Chronic Granulomatous Disease, exceeding therapeutic thresholds.
  • A single infusion of PM359 achieved 66% DHR positivity by Day 30, significantly above the 20% threshold believed to be potentially curative, with faster engraftment than existing gene editing technologies.
  • The therapy showed an encouraging safety profile with no serious adverse events related to PM359, marking a significant milestone for Prime Editing technology as a potential one-time curative treatment for genetic diseases.

Ruxoprubart Shows Promising Phase II Results as Novel Monotherapy for Paroxysmal Nocturnal Hemoglobinuria

Rare Disease
  • NovelMed's Ruxoprubart demonstrated significant efficacy in a Phase II trial for treatment-naïve PNH patients, meeting all primary endpoints including complete transfusion avoidance and increased hemoglobin levels.
  • The drug's selective inhibition of the Alternative Pathway without affecting the Classical Pathway offers a potentially safer profile than existing treatments, which often carry Black Box warnings for infection risk.
  • With FDA Orphan Drug Designation already secured and plans to file for Breakthrough Therapy Designation, Ruxoprubart is positioned as a potential best-in-class therapy for PNH and other complement-mediated disorders.

EMA Designates Allopurinol as First Orphan Drug for Marfan Syndrome

Orphan DrugRare Disease
  • The European Medicines Agency has designated allopurinol as the first orphan drug for Marfan syndrome, a rare connective tissue disease affecting approximately 7 in 100,000 people in the European Union.
  • Researchers from the University of Barcelona, IDIBAPS, and CIBERER have demonstrated allopurinol's potential to halt and prevent aortic aneurysms in animal models, with international clinical trials in patients planned for the future.
  • This repurposing of allopurinol, currently used for gout treatment, represents a significant advancement for Marfan syndrome patients who currently have no curative options beyond limited palliative treatments and high-risk surgical interventions.

Taiwan Approves Chugai's PiaSky as First Subcutaneous Treatment for Paroxysmal Nocturnal Hemoglobinuria

Rare DiseaseMonoclonal Antibody
  • Taiwan FDA has granted orphan drug approval for PiaSky, making it the first subcutaneous treatment for paroxysmal nocturnal hemoglobinuria (PNH) available in Taiwan for patients 13 years and older.
  • PiaSky, developed with Chugai's proprietary Recycling Antibody technology, allows for convenient subcutaneous administration every 4 weeks, significantly reducing treatment burden compared to existing biweekly intravenous options.
  • The approval was based on positive results from three Phase III clinical trials, including COMMODORE 2, which demonstrated efficacy in transfusion avoidance and control of hemolysis compared to eculizumab.

AKANTIOR® Receives UK Marketing Authorization as First Approved Treatment for Acanthamoeba Keratitis

Orphan DrugDrug ApprovalRare Disease
  • SIFI's AKANTIOR® (polihexanide 0.08%) has received both Marketing Authorization and Promising Innovative Medicine designation from the UK's MHRA, marking it as the first approved treatment for Acanthamoeba keratitis.
  • The approval confirms AKANTIOR's Orphan Drug Designation and New Active Substance status, recognizing its efficacy against an ultra-rare corneal infection that can lead to blindness if untreated.
  • Following its European approval in August 2024, this UK authorization represents a significant advancement for patients with this devastating eye infection, with SIFI planning to file for NICE reimbursement by June 2025.

FDA Approves 18 New Personalized Medicines in 2024, Marking Significant Shift in Treatment Paradigm

Drug ApprovalOncologyRare DiseaseAIReal World EvidencePrecision Medicine
  • The FDA approved 18 new personalized medicines in 2024, representing 38% of all newly approved therapeutic molecular entities across multiple treatment areas including cancer and Alzheimer's disease.
  • Six new gene and cell-based therapies for rare genetic diseases and cancers were authorized, alongside expanded indications for 11 diagnostic testing systems and the first-ever expanded indication for an approved gene therapy.
  • Personalized medicines now constitute at least 25% of drug approvals for the past decade, a substantial increase from less than 10% ten years ago, demonstrating the healthcare system's shift away from one-size-fits-all approaches.

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