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Jaguar Health Seeks Orphan Drug Designation for Crofelemer in Breast Cancer Brain Metastasis Patients

FDA Approval
  • Jaguar Health's subsidiary Napo Pharmaceuticals has submitted an orphan drug designation application to the FDA for crofelemer to treat diarrhea in breast cancer patients with brain metastasis receiving targeted therapy.
  • The FDA has previously recognized breast cancer with brain metastasis as a distinct orphan condition, awarding seven orphan drug designations for various therapies between 2015 and 2024.
  • Crofelemer, a botanical drug derived from Amazon rainforest trees, has already received orphan drug designations for other conditions in both the US and EU.
  • The company plans to seek Breakthrough Therapy or Fast Track designation to expedite regulatory pathways for this indication.

Prophylaxis of Diarrhea in Adult Cancer Patients Receiving Targeted Cancer Therapy

NCT04538625CompletedPhase 3
Napo Pharmaceuticals, Inc.
Posted 10/7/2020

OrganOx Receives FDA Approval for Air Transport of Liver Perfusion Device, Expanding Transplant Access Nationwide

FDA Approval
  • OrganOx's metra® normothermic machine perfusion device has received FDA approval for operation during air transport, marking a major milestone in expanding organ utilization across the United States.
  • The approval enables transplant centers and organ procurement organizations to safely transport donor livers via chartered aircraft, significantly extending the reach of the technology from coast to coast.
  • Through partnership with Strata Critical Medical, the company aims to remove logistical barriers to broader organ utilization while maintaining extended preservation times and functional liver assessment capabilities.
  • The metra® device has been utilized in over 6,000 liver transplants to date, helping increase the number of organs available for transplant by keeping donor livers metabolically active outside the body.

Nyxoah Files Patent Infringement Lawsuit Against Inspire Medical Systems Over Sleep Apnea Devices

FDA Approval
  • Nyxoah filed a patent infringement lawsuit against Inspire Medical Systems in Delaware federal court, alleging that Inspire's IV and V devices infringe three of Nyxoah's patents.
  • The lawsuit seeks injunctive relief and damages, as Nyxoah defends its intellectual property portfolio for the Genio system, a leadless and battery-free hypoglossal neurostimulation therapy for obstructive sleep apnea.
  • Nyxoah's Genio system received FDA approval in August 2025 and offers differentiated features including bilateral stimulation, full body MRI compatibility, and an upgradable platform without requiring re-surgery for battery replacements.
  • The company reports positive feedback from physicians and patients in the first month following its U.S. commercial launch of the Genio system.

Humacyte's Symvess Demonstrates 92% Patency Rate in Hospital-Acquired Vascular Complications Study

FDA Approval
  • Humacyte published outcomes in the Journal of Vascular Surgery showing Symvess achieved 92% patency rates and 100% limb salvage in 12 patients with hospital-acquired vascular complications.
  • The bioengineered vessel demonstrated zero cases of conduit infection over an average follow-up period of 23.3 months in patients requiring arterial repair.
  • Hospital-acquired vascular complications comprise nearly 30% of patients requiring vascular repair, representing a significant unmet medical need in emergency surgical settings.
  • Symvess offers an off-the-shelf alternative to autologous vein grafts, eliminating the need for additional surgical procedures to harvest patient vessels.

Biocartis Receives FDA Approval for First Fully Automated Companion Diagnostic Test for Colorectal Cancer

FDA ApprovalPrecision Medicine
  • Biocartis has received FDA Premarket Approval for the Idylla™ CDx MSI Test, marking the first fully automated, cartridge-based companion diagnostic test approved in the US for colorectal cancer patients.
  • The test identifies microsatellite instability-high (MSI-H) colorectal cancer patients who may benefit from treatment with OPDIVO® (nivolumab) alone or combined with YERVOY® (ipilimumab).
  • The diagnostic test delivers results in under three hours with less than three minutes of hands-on time, detecting seven monomorphic biomarkers for MSI detection in colorectal cancer tissue samples.
  • The approval was developed through a partnership with Bristol Myers Squibb and is supported by data from the CheckMate-8HW trial.

A Study of Nivolumab, Nivolumab Plus Ipilimumab, or Investigator's Choice Chemotherapy for the Treatment of Participants With Deficient Mismatch Repair (dMMR)/Microsatellite Instability High (MSI-H) Metastatic Colorectal Cancer (mCRC)

NCT04008030Active, Not RecruitingPhase 3
Bristol-Myers Squibb
Posted 8/5/2019

Pulnovo Medical Secures FDA IDE Approvals for PADN System Clinical Trials with CMS Coverage

FDA Approval
  • Pulnovo Medical received two FDA Investigational Device Exemption approvals for its PADN catheter and generator system targeting different pulmonary hypertension patient groups.
  • The approvals include an HDE study for Group I pulmonary arterial hypertension patients and a PMA study for Group II pulmonary hypertension patients.
  • CMS has granted full coverage approval for the clinical trials, significantly advancing the system's clinical application and market acceptance in the United States.
  • PADN technology offers treatment options for patients with limited response to existing therapies, with over 1,000 clinical applications demonstrating safety and efficacy globally.

Biocon Inaugurates First US Manufacturing Facility in New Jersey with $30 Million Investment

FDA Approval
  • Biocon Limited has opened its first FDA-approved manufacturing facility in Cranbury, New Jersey, through its subsidiary Biocon Generics Inc, marking a significant milestone in the company's global expansion strategy.
  • The company invested over $30 million to establish the state-of-the-art oral solid dosage facility with an annual production capacity of 2 billion tablets after acquiring it from Eywa Pharma Inc. in 2023.
  • The facility enables Biocon to deliver vertically integrated, high-quality medicines more efficiently to US patients while strengthening supply chain resilience and accelerating global market expansion.
  • New Jersey Governor Phil Murphy attended the inauguration, highlighting the facility's role in fostering innovation, creating job opportunities, and strengthening the US healthcare ecosystem.

Trump Administration Withdraws DEA Marijuana Rules, Clearing Path for FDA-Compliant Cannabis Trials

Orphan DrugFDA Approval
  • The Department of Justice withdrew 16 pending rulemakings and 38 projected rules under Trump's Executive Order 14192, including key DEA marijuana research initiatives that had stalled medical cannabis development for years.
  • MMJ BioPharma, which has been waiting since 2018 for DEA registration approval despite securing FDA Orphan Drug Designations for Huntington's disease and Multiple Sclerosis trials, now faces no regulatory excuses for continued delays.
  • The withdrawal eliminates DEA's procedural shield of "pending rulemakings" and forces decisions under existing Controlled Substances Act law, potentially accelerating FDA-compliant medical marijuana research.
  • With constitutional challenges to DEA's hearing process and the removal of reform proposals, Administrator Terry Cole must now decide whether to approve compliant applications or continue blocking legitimate pharmaceutical-grade cannabis research.

Apple Watch Receives FDA Approval for Hypertension Detection Feature, Expected to Identify Over One Million Undiagnosed Cases

FDA Approval
  • Apple's new hypertension notification feature for Apple Watch Series 9 and later models has received FDA approval and will launch with watchOS 26 on September 15.
  • The feature uses optical heart sensor data to analyze blood vessel responses over 30-day periods, with Apple claiming it could identify more than one million previously undiagnosed hypertension cases within the first year.
  • Users receiving hypertension alerts will be prompted to conduct seven-day home blood pressure monitoring with upper-arm cuffs and share results with healthcare providers.
  • The algorithm was trained on data from over 100,000 participants and validated in a clinical study of more than 2,000 people, though detailed performance metrics remain to be disclosed.

Capsida Suspends Gene Therapy Trial After First Patient Dies Following Treatment for Rare STXBP1 Disorder

Rare DiseaseFDA Approval
  • Capsida Biotherapeutics has suspended its clinical trial of CAP-002 gene therapy after the first participant died shortly after receiving treatment for STXBP1-related developmental and epileptic encephalopathy.
  • The company received FDA approval to begin the trial just four months ago in May, with plans to enroll around 12 children at Weill Cornell Medicine and Children's Hospital of Philadelphia.
  • CAP-002 uses a specially engineered viral vector to deliver therapy to brain neurons and aims to compensate for deficient levels of protein encoded by the STXBP1 gene.
  • The death follows other patient fatalities in gene therapy trials over the past year, raising concerns about safety in this therapeutic area.

A Clinical Trial of CAP-002 Gene Therapy in Pediatric Patients With Syntaxin-Binding Protein 1 (STXBP1) Encephalopathy

NCT06983158SuspendedPhase 1
Capsida Biotherapeutics, Inc.
Posted 7/3/2025

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