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RedHill Biopharma Receives FDA Approval for Groundbreaking MAP-Targeted Crohn's Disease Study

FDA ApprovalOrphan Drug
  • RedHill Biopharma received positive FDA feedback for the first-ever clinical study targeting Mycobacterium avium subspecies paratuberculosis (MAP) infected Crohn's disease patients with RHB-204.
  • The novel Phase 2 study will test MAP as a root cause of Crohn's disease, potentially making RHB-204 a paradigm-shifting therapy treating both the suspected cause and symptoms.
  • RHB-204 is supported by positive Phase 3 data from RHB-104 showing 64% improvement in efficacy and offers a 40% pill burden reduction with patent protection through 2041.
  • The Crohn's disease market is expected to grow from $13.6 billion in 2024 to over $19 billion by 2033, presenting significant commercial potential for new therapies.

XVIVO Initiates US Continued Access Protocol Study for Heart Assist Transport Device Following Successful PRESERVE Trial

FDA Approval
  • XVIVO Perfusion has enrolled the first patient in its US PRESERVE Continued Access Protocol (CAP) study for the XVIVO Heart Assist Transport device, allowing up to 60 patients across 26 transplant centers to access the technology.
  • The FDA-approved CAP study enables continued clinical use of the heart transport device while XVIVO analyzes one-year follow-up data from the PRESERVE trial in preparation for Pre-Market Approval submission.
  • The Centers for Medicare & Medicaid Services has granted continued cost recovery approval, providing essential financial support for participating transplant centers and patients during the interim regulatory period.

Acute Ischemic Stroke Pipeline Shows Promise with 25+ Therapies in Development and Recent Clinical Breakthroughs

FDA ApprovalMonoclonal Antibody
  • DelveInsight's 2025 analysis reveals over 20 key companies are developing 25+ acute ischemic stroke therapies across various clinical phases, representing significant advancement in stroke treatment research.
  • Recent clinical trials demonstrate promising results, including the ASSET-IT trial showing 65.9% excellent recovery rates with tirofiban combination therapy compared to 54.9% with placebo.
  • Multiple FDA designations have been granted, including Fast Track status for PP-007 and Orphan Drug Designation for CIRARA, accelerating development timelines for innovative stroke treatments.
  • The pipeline encompasses diverse therapeutic approaches including monoclonal antibodies, small molecules, and cell therapies, targeting various mechanisms from neuroprotection to thrombolysis enhancement.

PMZ-1620 (Sovateltide) in Acute Ischemic Stroke Patients

NCT04046484CompletedPhase 2
Pharmazz, Inc.
Posted 1/19/2018

Efficacy of Sovateltide (PMZ-1620) in Patients of Acute Ischemic Stroke

NCT04047563CompletedPhase 3
Pharmazz, Inc.
Posted 11/10/2019

UroMems Receives FDA and ANSM Clearance for Pivotal Trial of Smart Artificial Urinary Sphincter

FDA Approval
  • UroMems received investigational device exemption approval from the FDA and French ANSM to begin the SOPHIA2 pivotal trial of its UroActive smart implant for treating male stress urinary incontinence.
  • The UroActive system represents the first automated artificial urinary sphincter with smart technology, utilizing a MyoElectroMechanical System that adapts treatment based on patient activity without complex manipulation.
  • The multicenter trial follows strong feasibility study results in France showing over one year with no need for revision or explant, with the study serving as the basis for regulatory submissions to commercialize in US and European markets.

Clinical Investigation to Assess a New Artificial Urinary Sphincter to Treat Urinary Incontinence in Men

NCT05547672Active, Not RecruitingNot Applicable
UroMems SAS
Posted 8/31/2022

A Study to Assess the Safety and Effectiveness of the UrOActive® Artificial Urinary sPHincter (AUS)

NCT06968741RecruitingNot Applicable
UroMems SAS
Posted 5/19/2025

Clinical Investigation to Assess a New Artificial Urinary Sphincter to Treat Urinary Incontinence in Women

NCT05828979Active, Not RecruitingNot Applicable
UroMems SAS
Posted 5/2/2023

Saluda Medical Launches EVA Sensing Technology for Personalized Spinal Cord Stimulation in U.S.

FDA Approval
  • Saluda Medical has launched EVA sensing technology in the U.S. following FDA approval in December 2024, marking a significant advancement in spinal cord stimulation therapy.
  • The technology objectively scans and analyzes patients' spinal cords to deliver personalized therapy with precision beyond human capability, utilizing neural response biomarkers.
  • EVA has been used in over 3,000 commercial patient visits during limited market release and automates manual programming steps to improve patient experience.
  • New clinical data will be presented at ASPN 2025 Annual Conference, adding to over 37 publications including the landmark EVOKE Study showing 36-month efficacy.

Stargardt Disease Pipeline Shows Promising Progress with Multiple Gene Therapies Advancing to Late-Stage Trials

Rare DiseaseFDA Approval
  • Ocugen's OCU410ST gene therapy has received FDA approval to launch a Phase 2/3 pivotal confirmatory trial for treating all forms of Stargardt disease, with the potential to serve as the foundation for a biologics license application.
  • SpliceBio has administered the first dose in its Phase 1/2 ASTRA clinical trial evaluating SB-007, a dual AAV vector-based gene therapy for Stargardt disease, marking another significant milestone in gene therapy development.
  • Alkeus Pharmaceuticals reported positive interim results from its TEASE-3 study, showing that early-stage Stargardt disease patients receiving oral gildeuretinol acetate exhibited no disease progression and maintained stable visual acuity over multiple years.
  • The Stargardt disease pipeline now includes over 20 companies developing more than 20 treatment therapies, with emerging therapies like MCO-010, Tinlarebant, and others expected to significantly impact the market.

Medtronic Initiates PELE Clinical Trial for Onyx Liquid Embolic System in Peripheral Arterial Hemorrhage

FDA Approval
  • Medtronic has enrolled the first patient in the PELE clinical trial to evaluate the Onyx Liquid Embolic System for treating arterial hemorrhage in peripheral vasculature.
  • The pivotal study will enroll up to 119 patients across 25 U.S. sites to assess safety and effectiveness through 30 days post-treatment.
  • The trial aims to provide evidence for U.S. regulatory approval, as Onyx LES is already available with peripheral indications in many international markets.
  • The first procedure was successfully performed by Dr. Christopher Stark at Albany Medical Center for a patient with bleeding from a ruptured blood vessel.

Photodynamic Therapy Shows Promise in Cancer Treatment with Global Market Expected to Reach $6 Billion by 2030

FDA Approval
  • The global photodynamic therapy market is projected to surpass $6 billion by 2030, driven by technological advances in LED-based light delivery systems and improved photosensitizer formulations.
  • Recent clinical developments include Sun Pharmaceutical's FDA-cleared BLU-U Blue Light PDT Illuminator and Biofrontera's successful Phase 3 study of Ameluz® for superficial basal cell carcinoma treatment.
  • Comprehensive meta-analysis evidence demonstrates PDT's effectiveness varies by cancer type, with strong benefits for cholangiocarcinoma when combined with stenting and superior cosmetic outcomes for skin cancers compared to traditional treatments.

Iovance Biotherapeutics Recruits Former FDA Oncology Leader Marc Theoret as Senior VP of Regulatory Strategy

FDA Approval
  • Iovance Biotherapeutics has appointed Marc R. Theoret, M.D., former FDA Deputy Center Director, to the newly created position of Senior Vice President, Regulatory Strategy.
  • Dr. Theoret brings over 15 years of FDA experience, including five years as Deputy Center Director of the Oncology Center of Excellence and leadership in regulatory reviews for hematological malignancies and solid tumors.
  • The appointment strengthens Iovance's regulatory capabilities as the company advances its tumor infiltrating lymphocyte (TIL) therapy pipeline across multiple solid tumor cancers.
  • Iovance's Amtagvi represents the first FDA-approved T cell therapy for a solid tumor indication, positioning the company as a leader in the TIL therapy space.

Neurizon Receives FDA Approval for Strategy to Lift Clinical Hold on ALS Drug NUZ-001

FDA Approval
  • Neurizon Therapeutics received written FDA confirmation accepting its strategy to lift the clinical hold on NUZ-001, an investigational treatment for amyotrophic lateral sclerosis (ALS).
  • The company has completed two required preclinical pharmacokinetic studies ahead of schedule to address FDA concerns about systemic exposure data.
  • Neurizon anticipates submitting its complete response to the FDA in the coming weeks and expects the clinical hold to be lifted by August 2025.
  • The regulatory progress positions NUZ-001 for entry into the HEALEY ALS Platform Trial in the second half of 2025.

A Study to Evaluate, Safety, Tolerability, Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS)

NCT05039099CompletedPhase 2
AL-S Pharma
Posted 9/2/2021

An Open Label Extension Study of Monepantel in Individuals With Motor Neurone Disease

NCT06177431CompletedPhase 1
Neurizon Therapeutics Limited
Posted 2/13/2024

A Study of Monepantel in Individuals With Motor Neurone Disease

NCT04894240CompletedPhase 1
PharmAust Ltd
Posted 6/28/2022

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