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F2G's Olorofim Shows 44% Response Rate in Phase 2b Trial for Invasive Fungal Infections

Orphan Drug
  • F2G announced positive Phase 2b data for olorofim showing a 44% complete or partial response rate at day 42 in 100 patients with invasive fungal infections who had limited treatment options.
  • The novel antifungal demonstrated low mortality rates of 15% at day 42 and 20% at day 84, with generally good tolerability except for drug-induced liver injury in 8% of patients.
  • Olorofim is the only antifungal with FDA Breakthrough Therapy Designation and works through a unique mechanism targeting dihydroorotate dehydrogenase in the pyrimidine synthesis pathway.
  • The drug showed activity against multiple resistant fungi including Aspergillus species, Lomentospora prolificans, and Coccidioides species, addressing significant unmet medical needs in immunocompromised patients.

Olorofim Aspergillus Infection Study

NCT05101187RecruitingPhase 3
F2G Biotech GmbH
Posted 3/31/2022

Evaluate F901318 (Olorofim) Treatment of Invasive Fungal Infections in Participants Lacking Treatment Options

NCT03583164CompletedPhase 2
F2G Biotech GmbH
Posted 6/6/2018

PharmaTher Secures U.S. Patent for Advanced Ketamine Production Technology

Orphan Drug
  • PharmaTher Holdings received U.S. Patent No. 11,286,230 for "Ketamine Flow Synthesis," a continuous-flow process technology that improves ketamine and analog production efficiency.
  • The patented technology addresses traditional batch production limitations by offering better yield, reproducibility, and purity while requiring a smaller manufacturing footprint.
  • The company plans to leverage this technology to support upcoming pivotal clinical studies for Parkinson's disease and ALS, with an FDA filing expected in Q4-2022.
  • PharmaTher's expanding intellectual property portfolio includes novel ketamine applications for neurological disorders and innovative delivery systems like microneedle patches.

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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