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European Commission Approves First MEK Inhibitor for NF1-Associated Plexiform Neurofibromas in Adults and Children

Drug ApprovalRare DiseaseOrphan Drug
  • The European Commission has granted conditional approval for Ezmekly (mirdametinib), the first therapy approved for both adults and children with neurofibromatosis type 1-associated plexiform neurofibromas.
  • The approval is based on the ReNeu Phase 2b trial results showing objective response rates of 41% in adults and 52% in children, with median tumor volume reductions of approximately 40% in both populations.
  • Ezmekly is a selective MEK 1/2 inhibitor that blocks the RAF-MEK-ERK pathway, addressing a significant unmet need for patients with symptomatic, inoperable plexiform neurofibromas aged 2 years and above.
  • The drug demonstrated a manageable safety profile with the most common adverse reactions including dermatitis acneiform, diarrhea, and elevated blood creatine phosphokinase levels.

MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas

NCT03962543Active, Not RecruitingPhase 2
SpringWorks Therapeutics, Inc.
Posted 9/29/2019

Aptahem Advances Apta-1 to Phase II as European Pharma Company Expresses Strong Partnership Interest

Orphan Drug
  • Aptahem has received formal partnership interest from a well-reputed European pharmaceutical company for its RNA-based lead candidate Apta-1, which targets severe inflammatory conditions.
  • The company has finalized plans for a Phase II "basket trial" to evaluate Apta-1 in patients with acute urogenital, kidney, and lung disorders characterized by inflammatory and thrombotic pathologies.
  • Aptahem has initiated a strategic collaboration with Hongene Biotech to develop cost-effective and sustainable manufacturing methods for Apta-1, a critical step toward clinical advancement and commercialization.

Vicore Pharma's Buloxibutid Shows Superior Anti-Fibrotic Activity for IPF Treatment in New Data

Orphan Drug
  • Vicore Pharma presented promising Phase 2a trial data at the 2025 ATS Conference showing buloxibutid significantly improved lung function in idiopathic pulmonary fibrosis patients compared to existing treatments.
  • The novel angiotensin II type 2 receptor agonist demonstrated superior anti-fibrotic activity in laboratory studies, inhibiting key fibrosis biomarkers at clinically relevant concentrations where competitors nintedanib and nerandomilast showed limited effects.
  • Vicore's ongoing Phase 2b ASPIRE trial incorporates patient-friendly design elements based on input from IPF patients and caregivers, while their digital therapeutic Almee has shown improvements in quality of life for pulmonary fibrosis patients.

India's CDSCO Approves Two Cancer Drugs with Phase IV Trial Requirements

Drug ApprovalMonoclonal AntibodyOrphan Drug
  • India's drug regulatory authority has approved Eli Lilly's selpercatinib tablets in multiple strengths for RET fusion-positive non-small cell lung cancer treatment.
  • Intas Pharmaceuticals received approval to import and market serplulimab, a PD-1 inhibitor monoclonal antibody for cancer treatment.
  • Both approvals come with mandatory Phase IV clinical trial requirements to be submitted within three months of marketing authorization.
  • The approvals reflect India's regulatory alignment with global standards for orphan cancer drugs already approved in major markets.

Philikos Initiates Phase 1/2 Trial of T-Guard for Diffuse Cutaneous Systemic Sclerosis

Monoclonal AntibodyOrphan Drug
  • Philikos has enrolled the first patient in a Phase 1/2 trial evaluating T-Guard for diffuse cutaneous systemic sclerosis (dcSSc), a severe autoimmune disorder with limited treatment options.
  • The open-label study will assess safety and preliminary efficacy in 12 early-stage dcSSc patients whose disease remains refractory despite prior immunosuppressive therapy.
  • T-Guard, administered as four infusions over one week, aims to provide a safer alternative to hematopoietic stem cell transplantation by selectively depleting disease-associated T cells and NK cells.

FDA Greenlights Promontory Therapeutics' Phase 3 Trial Design for PT-112 in Metastatic Prostate Cancer

OncologyOrphan Drug
  • Promontory Therapeutics has successfully completed an End of Phase 2 meeting with the FDA, reaching agreement on key aspects of a registrational Phase 3 trial for PT-112 in metastatic castration-resistant prostate cancer.
  • The FDA approved the proposed dosing regimen, patient population, study comparator, and endpoints, with an interim analysis provision that could allow for drug approval before study completion.
  • Preliminary clinical outcomes from the Phase 2 trial of PT-112 will be presented at the upcoming ASCO 2025 Annual Meeting on June 2nd, following recent presentation of immune response biomarker data at AACR 2025.

PT-112 in Subjects With Thymoma and Thymic Carcinoma

NCT05104736RecruitingPhase 2
National Cancer Institute (NCI)
Posted 4/6/2022

A Study Evaluating the Safety, Pharmacokinetics, and Clinical Effects of Intravenously Administered PT-112 Injection in Subjects With Advanced Solid Tumors and Subsequent Dose Expansion Cohorts

NCT02266745Active, Not RecruitingPhase 2
Promontory Therapeutics Inc.
Posted 7/1/2014

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

Juvena Therapeutics Initiates First-in-Human Trial of JUV-161 for Muscle Regeneration in Myotonic Dystrophy

Orphan Drug
  • Juvena Therapeutics has begun enrolling participants in the first human clinical trial of JUV-161, a novel fusion protein designed to enhance muscle regeneration for treating Myotonic Dystrophy Type 1 and sarcopenia.
  • JUV-161, described as "insulin for muscle," works by restoring AKT signaling pathways that regulate muscle growth and metabolism, offering a unique approach compared to existing RNA-targeting or gene therapy strategies.
  • The therapy was discovered using Juvena's proprietary JuvNET platform, which combines AI and stem cell secretome biology to identify therapeutic proteins with regenerative potential.

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.

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.

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