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First Patient Dosed in Phase 2b Trial of RT234, a Novel Inhaled Therapy for Pulmonary Arterial Hypertension

Orphan Drug
  • Respira Therapeutics has dosed the first patient in its Phase 2b VIPAH-PRN trial evaluating RT234, an innovative dry powder inhaled formulation of vardenafil for on-demand symptom relief in PAH patients.
  • RT234 represents a first-in-class approach for PAH, designed for "as needed" use to acutely improve exercise capacity and reduce breathlessness, unlike current maintenance therapies that don't address episodic symptoms.
  • The multicenter dose-escalation study will assess RT234's ability to provide rapid symptom relief for patients with NYHA Functional Class II-III PAH symptoms, potentially addressing a significant unmet need in disease management.

FDA Grants Breakthrough Therapy Designation to Rusfertide for Polycythemia Vera

Orphan Drug
  • The FDA has granted breakthrough therapy designation to rusfertide (PTG-300), an injectable hepcidin mimetic, for treating polycythemia vera patients to reduce erythrocytosis.
  • Phase 2 trial data showed rusfertide eliminated the need for therapeutic phlebotomies in most patients while maintaining target hematocrit levels below 45%.
  • The drug demonstrated ability to reverse iron deficiency and improve disease-related symptoms in heavily phlebotomy-dependent patients.
  • Rusfertide represents a potential first non-cytoreductive therapeutic option for polycythemia vera, offering an alternative to current treatment approaches.

Hepcidin Mimetic in Patients With Polycythemia Vera (REVIVE)

NCT04057040CompletedPhase 2
Protagonist Therapeutics, Inc.
Posted 10/1/2019

FDA Approves First Treatment for Primary Hemophagocytic Lymphohistiocytosis, Marking 24-Year Breakthrough

Monoclonal AntibodyDrug ApprovalRare DiseaseOrphan Drug
  • The FDA has approved emapalumab-lzsg (Gamifant) as the first treatment specifically indicated for primary hemophagocytic lymphohistiocytosis (HLH), an ultra-rare and life-threatening immune disorder.
  • In a pivotal phase 2/3 study of 27 patients with refractory disease, 63% demonstrated overall response and 70% proceeded to hematopoietic stem cell transplant.
  • The monoclonal antibody targets interferon gamma, representing the first significant improvement in primary HLH induction therapy in 24 years.
  • Primary HLH typically affects children within the first year of life and has a median survival of less than two months without treatment.

Study to Investigate Safety, Efficacy of an Anti-IFNγ mAb in Children With Primary Haemophagocytic Lymphohistiocytosis

NCT01818492CompletedPhase 2
Swedish Orphan Biovitrum
Posted 7/1/2013

Life Molecular Imaging Secures FDA Fast Track Designation for [18F]florbetaben in Cardiac Amyloidosis Diagnosis

Orphan Drug
  • Life Molecular Imaging received FDA Fast Track Designation for [18F]florbetaben to diagnose cardiac amyloid light-chain (AL) and amyloid transthyretin-related (ATTR) amyloidosis.
  • The PET imaging agent is already approved as Neuraceq® for detecting brain amyloid plaques and has demonstrated capability to identify amyloid deposits in the heart.
  • A Phase 3 trial (NCT05184088) is currently underway to validate [18F]florbetaben's efficacy in cardiac amyloidosis diagnosis.
  • The designation aims to streamline the complex diagnostic process for cardiac AL amyloidosis, potentially providing earlier access to therapy and improved patient monitoring.

Efficacy of [18F]Florbetaben PET for Diagnosis of Cardiac AL Amyloidosis

NCT05184088RecruitingPhase 3
Life Molecular Imaging GmbH
Posted 1/13/2023

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