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GSK Receives FDA Orphan Drug Designation for Autosomal Dominant Polycystic Kidney Disease Treatment

Monoclonal AntibodyRare DiseaseFDA Approval
  • GSK received FDA Orphan Drug Designation for its investigational treatment of autosomal dominant polycystic kidney disease (ADPKD), a rare genetic disorder affecting approximately 600,000 people in the U.S.
  • The designation provides critical incentives including tax credits, reduced regulatory fees, and seven years of market exclusivity upon approval for GSK's monoclonal antibody candidate currently in Phase 1 trials.
  • GSK's kidney disease strategy extends beyond ADPKD through a £50 million, five-year partnership with University of Cambridge focusing on immune-related kidney diseases using AI-driven precision therapies.
  • The company's diversified approach includes Atacicept, a B-cell inhibitor for IgA nephropathy that recently completed Phase III trials, positioning GSK to capture market share in the growing kidney disease therapeutics sector.

Arctx Medical Receives FDA IDE Approval for Pivotal Trial of Novel Cooling Therapy for Acute Pancreatitis

FDA Approval
  • Arctx Medical received FDA Investigational Device Exemption approval to conduct a pivotal clinical trial of its Cool Catheter Kit in approximately 200 acute pancreatitis patients at up to 25 US hospitals.
  • The company successfully completed a first-in-human trial with 21 acute pancreatitis patients treated with pancreatic cooling therapy, with results being prepared for peer-review publication.
  • The novel therapy addresses a significant unmet medical need, as acute pancreatitis has no dedicated treatments available and causes over $3 billion in annual US healthcare costs.
  • Dr. Robert Kieval joined as CEO in July 2025, bringing over 20 years of medical device leadership experience to commercialize this first-of-its-kind treatment.

Allarity Therapeutics Receives FDA Fast Track Designation for Stenoparib in Advanced Ovarian Cancer

FDA Approval
  • Allarity Therapeutics received FDA Fast Track designation for stenoparib, an investigational dual PARP/WNT inhibitor for advanced ovarian cancer treatment.
  • The designation accelerates development timelines and enables more frequent FDA interactions with potential eligibility for expedited approval pathways.
  • The company has initiated a Phase 2 clinical trial building on prior data showing patients remaining on treatment for over 22 months.
  • ALLR stock surged over 119% intraday following the regulatory milestone, with trading volume increasing by 2,728% to $304.8 million.

Agilent Technologies Receives FDA Approval for First-of-Its-Kind Colorectal Cancer Companion Diagnostic

FDA Approval
  • Agilent Technologies received FDA approval for its MMR IHC Panel pharmDx as the only FDA-approved IHC panel for selecting colorectal cancer patients eligible for specific immunotherapies.
  • The companion diagnostic enables precise identification of patients eligible for treatment with Bristol Myers Squibb's Opdivo, either alone or in combination with Yervoy.
  • This regulatory milestone showcases Agilent's partnership with Bristol Myers Squibb in delivering diagnostics that guide targeted cancer treatments.
  • The approval represents a significant advancement in precision oncology, allowing clinicians to better match colorectal cancer patients with appropriate immunotherapy options.

FDA Rejects Blu Disposable E-Cigarette Marketing Application Over Dual-Use Concerns

FDA Approval
  • The FDA denied marketing authorization for Fontem US's Blu disposable classic tobacco-flavored 2.4% nicotine e-cigarette on August 19, citing insufficient evidence of public health benefits.
  • The agency determined that users would likely continue smoking cigarettes while using the Blu product, potentially exposing them to higher toxicant levels than exclusive cigarette use.
  • Fewer than 30 vaping products have received FDA marketing approval, with only four brands currently authorized: Juul, Altria's Njoy, Reynolds American's Vuse, and Japan Tobacco's Logic.
  • The FDA emphasized that authorized e-cigarettes must demonstrate complete switching from cigarettes or significant reduction in cigarette consumption to receive approval.

Vanda Pharmaceuticals Appeals to FDA Commissioner Makary Over Generic Hetlioz Approvals Following Court Victory

FDA Approval
  • Vanda Pharmaceuticals has requested FDA Commissioner Martin Makary to review the agency's approval of two generic versions of Hetlioz, challenging what it calls an "unacceptable culture of bias" toward generic drugs.
  • The company argues that outgoing CDER director Jacqueline Corrigan-Curay upheld generic approvals despite significant study flaws, including bioequivalence studies conducted only in Indian male subjects for use in the general US population.
  • This appeal follows Vanda's recent federal court victory over the FDA regarding Hetlioz's jet lag disorder indication, where the court found the company had provided meaningful evidence of efficacy.
  • Vanda contends that one generic version showed exposure levels more than double those of original Hetlioz, raising safety concerns for American patients.

Boehringer Ingelheim Partners with AnGes for HGF Gene Therapy Manufacturing Ahead of FDA Filing

FDA Approval
  • Boehringer Ingelheim has signed a contract development and manufacturing agreement with AnGes for the drug substance of their investigational hepatocyte growth factor gene therapy targeting peripheral arterial disease.
  • The partnership follows AnGes' completion of clinical trials and plans to submit a biologics license application to the FDA for their HGF gene therapy product.
  • Peripheral arterial disease affects 200 million people worldwide and can lead to severe complications including ulceration, infection, and limb amputation with mortality rates comparable to cancer.
  • The plasmid DNA molecule has been manufactured using proprietary microbial technology in E. coli at Boehringer's Vienna facility for nearly 20 years since the collaboration began.

Agilent's MMR IHC Panel PharmDx Receives FDA Approval as Companion Diagnostic for Colorectal Cancer Immunotherapy

FDA ApprovalPrecision Medicine
  • Agilent Technologies received FDA approval for its MMR IHC Panel pharmDx (Dako Omnis) as a companion diagnostic test to identify mismatch repair deficient colorectal cancer patients eligible for Bristol Myers Squibb's Opdivo immunotherapy treatments.
  • The test detects loss of function in four key mismatch repair proteins (MLH1, PMS2, MSH2, and MSH6) in colorectal cancer tissue, making it the only FDA-approved companion diagnostic IHC panel for this specific indication.
  • This diagnostic tool enhances healthcare providers' ability to tailor immunotherapy treatments for colorectal cancer patients, potentially improving tumor control and progression-free survival outcomes.

A Study of Nivolumab Alone or Nivolumab Combination Therapy in Colon Cancer That Has Come Back or Has Spread

NCT02060188CompletedPhase 2
Bristol-Myers Squibb
Posted 3/12/2014

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

Neurovalens Secures £6M to Expand FDA-Approved Neurostimulation Devices for Mental Health

FDA Approval
  • Belfast-based Neurovalens closed a £6 million funding round led by Investment Fund for Northern Ireland to accelerate commercial expansion of its neurostimulation medical devices in the US and global markets.
  • The company's technology uses mild electrical stimulation to treat insomnia, anxiety, and PTSD, with existing FDA clearance for devices targeting insomnia and anxiety management.
  • Funding will support scaling sales of current medical devices and pursuing additional FDA approvals to expand the product portfolio for treating serious global health challenges through non-invasive neurostimulation.

Merit Medical Enrolls First Patient in WRAPSODY Registry for Hemodialysis Vascular Access

FDA ApprovalReal World Evidence
  • Merit Medical Systems has enrolled the first patient in its WRAP North America registry, a prospective multicenter study designed to collect real-world safety and effectiveness data on the WRAPSODY Cell-Impermeable Endoprosthesis for hemodialysis patients.
  • The registry will enroll up to 250 US and Canadian hemodialysis patients experiencing vascular access obstructions such as stenosis or occlusion, with clinical outcomes evaluated over three years.
  • The WRAPSODY CIE received FDA premarket approval in December 2024 and Health Canada approval in April 2025, following superior performance demonstrated in the WAVE pivotal trial.
  • If completed as designed, this registry would represent the largest cohort of patients treated with an implantable device to restore vascular access for hemodialysis.

WRAP North America

NCT06807099Recruiting
Merit Medical Systems, Inc.
Posted 7/31/2025

Merit WRAPSODY™ Endoprosthesis for Treatment of Stenosis or Occlusion (WRAP)

NCT05062291Recruiting
Merit Medical Systems, Inc.
Posted 6/23/2022

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