MedPath

Tagged News

Beam Therapeutics and Capricor Secure FDA Orphan Drug Designations for Rare Disease Treatments

Rare DiseaseFDA Approval
  • Beam Therapeutics received FDA orphan drug designation for its sickle cell disease treatment, providing regulatory incentives for this rare genetic disorder affecting hemoglobin production.
  • Capricor Therapeutics obtained orphan drug designation for its Becker muscular dystrophy therapy, targeting this progressive muscle-wasting condition.
  • Both designations offer companies seven years of market exclusivity, tax credits, and reduced regulatory fees to encourage development of treatments for rare diseases affecting fewer than 200,000 patients in the United States.

FDA Issues Draft Guidance to Streamline Color Additive Replacement in Drug Products

FDA Approval
  • The FDA has released draft guidance allowing most color additive replacements in approved drug products to be submitted as moderate changes requiring only 30-day notification rather than prior approval supplements.
  • The new guidance applies to prescription drugs, over-the-counter products, and compounded drugs, requiring that replacement color additives be listed in FDA's color additive regulations.
  • Manufacturers must provide comprehensive documentation including stability studies, dissolution testing, and updated labeling when replacing color additives in drug formulations.
  • The regulatory change addresses situations where color additives must be removed for safety reasons or replaced for business decisions while maintaining product quality standards.

Cerus Corporation Reports Positive Phase 3 Results for INTERCEPT Blood System at ISBT Congress

FDA Approval
  • Cerus Corporation will present positive Phase 3 ReCePI study results for its INTERCEPT Blood System red blood cell treatment at the 35th ISBT Congress in Milan.
  • The presentations demonstrate broad clinical applicability of the INTERCEPT system across multiple blood components including platelets, plasma, and red blood cells.
  • Clinical data showcases reduced hemoglobin use in complex cardiac surgery and effective pathogen inactivation capabilities including California encephalitis virus.
  • The INTERCEPT system represents the only FDA-approved pathogen reduction technology for platelets and plasma globally.

FDA Approves 7-Year Post-Market Study for BrainSee Alzheimer's Progression Prediction Tool

AIFDA Approval
  • The FDA has approved Darmiyan's 7-year post-market surveillance study for BrainSee, the first FDA-approved AI-powered tool that predicts Alzheimer's disease progression in patients with mild cognitive impairment.
  • BrainSee combines standard brain MRI, cognitive assessments, age, and biological sex to determine whether patients over 55 with amnestic mild cognitive impairment will progress to clinical Alzheimer's disease within five years.
  • The study will validate BrainSee's effectiveness across diverse patient populations and support pharmaceutical companies in identifying optimal candidates for next-generation Alzheimer's clinical trials.
  • Unlike traditional tests that detect amyloid beta plaques, BrainSee addresses the critical gap where roughly 40% of amyloid-positive patients remain stable and may not benefit from anti-amyloid drugs.

CoreMap Receives FDA IDE Approval to Expand Novel Atrial Fibrillation Mapping Technology Study to U.S.

FDA Approval
  • CoreMap has received FDA Investigational Device Exemption approval to extend its INvENI clinical study to the U.S., evaluating its proprietary electrophysiology mapping system in persistent atrial fibrillation patients.
  • The company's ultra-high resolution mapping technology has demonstrated safety and acute effectiveness in over 50 patients, with the ability to identify AF driver regions not detectable by prior mapping methods.
  • The INvENI study is a multi-phase randomized controlled trial comparing CoreMap-guided ablation plus pulmonary vein isolation against standard-of-care treatment in persistent AF patients.
  • The technology features a dense array of micro-scale electrodes that can record hundreds of thousands of electrical activations during a single AF map, enabling patient-specific ablation strategies.

Portal Access Secures $7M Series A to Advance Next-Generation Chemotherapy Port Through FDA Approval

OncologyFDA Approval
  • Portal Access, Inc. closed a $7 million Series A financing round with over 80% led by U.S. physician investors to support FDA approval of its Flexi-Port™ SLF-TANL™ device.
  • The novel subcutaneous port features self-tunneling technology designed to simplify vascular access for oncology patients and enable bedside or outpatient placement.
  • Despite being standard of care, nearly half of cancer patients do not receive chemo-ports due to various delays, highlighting the unmet need this device aims to address.
  • The company plans 510(k) submission in July 2025 with anticipated U.S. market launch in early 2026.

Pearl Achieves Historic FDA Clearance for First AI Platform Supporting Both 2D and 3D Dental Imaging Analysis

AIFDA Approval
  • Pearl becomes the first dental AI company to receive FDA 510(k) clearance for both 2D and 3D radiologic image analysis with its Second Opinion® 3D platform.
  • The newly cleared platform enables automated identification of critical anatomical structures in CBCT scans, including dentition, maxilla, mandible, and airway structures.
  • Second Opinion® 3D demonstrated high segmentation accuracy with Dice Similarity Coefficient scores exceeding clinical thresholds across all targeted anatomical categories during bench performance testing.
  • The platform supports enhanced diagnostic precision and treatment planning across multiple dental specialties including implantology, orthodontics, oral surgery, and airway management.

Abbott's Tendyne System Receives FDA Approval as First Transcatheter Mitral Valve Replacement for Severe Calcification

FDA Approval
  • Abbott received FDA approval for the Tendyne transcatheter mitral valve replacement system, marking the first device of its kind to replace mitral valves without open-heart surgery for patients with severe mitral annular calcification.
  • The minimally invasive device addresses a critical treatment gap for high-risk patients who cannot undergo traditional surgery or mitral valve repair with existing technologies like MitraClip.
  • The self-expanding valve is delivered through a small chest incision and features full repositionability during implantation, with multiple sizes available to accommodate diverse patient anatomies.
  • This approval expands Abbott's structural heart portfolio and builds on over two decades of mitral valve innovation, offering new hope for patients experiencing symptoms like chest pain, shortness of breath, and dizziness.

Sequana Medical Secures EUR 6.3 Million Financing to Support alfapump US Commercial Launch

FDA Approval
  • Sequana Medical raised EUR 6.3 million in additional convertible financing from SFPIM and existing shareholders, bringing total 2025 convertible loan investment to EUR 10.3 million.
  • The funding extends the company's cash runway into Q1 2026 and supports the planned Q3 2025 US commercial launch of alfapump through a specialty sales team targeting liver transplant centers.
  • The alfapump system, approved by FDA in December 2024, is the first active implantable device in the US that automatically removes ascites from patients with liver cirrhosis.
  • Clinical data from the POSEIDON study demonstrated the device virtually eliminates the need for therapeutic paracentesis and improves quality of life in patients with recurrent or refractory ascites.

Oxford Brain Diagnostics Launches Revolutionary MRI-Based Dementia Diagnosis Technology Following FDA and UK Regulatory Approvals

FDA Approval
  • Oxford Brain Diagnostics has received FDA 510(k) clearance and UKCA self-certification to commercially launch its Cortical Disarray Measurement (CDM®) technology for dementia diagnosis in the UK and US markets.
  • The patented software analyzes standard MRI scans to provide objective measurements of neurodegeneration, enabling earlier and more accurate assessment of brain health conditions like Alzheimer's disease.
  • The technology received FDA Breakthrough Device Designation in 2020 and is backed by multi-million-pound funding from BGF, positioning the company to address the growing need for precision diagnostics as over 55 million people worldwide live with dementia.
  • Beyond Alzheimer's diagnosis, the platform shows promise for detecting Parkinson's disease and multiple sclerosis, with applications extending to pharmaceutical trials and drug development.

Dominantly Inherited Alzheimer Network Trial: An Opportunity to Prevent Dementia: A Study of Potential Disease Modifying Treatments in Individuals at Risk for or With a Type of Early Onset Alzheimer's Disease Caused by a Genetic Mutation

NCT06424236TerminatedPhase 2
Washington University School of Medicine
Posted 6/3/2020

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.