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Clinical Trial News

FDA Grants Orphan Drug Designation to Ractigen's RAG-21 for FUS-ALS

  • The FDA has granted Orphan Drug Designation to Ractigen Therapeutics' RAG-21 for amyotrophic lateral sclerosis (ALS) treatment, specifically targeting the FUS subtype.
  • RAG-21 is a small interfering RNA (siRNA) therapy designed to reduce toxic FUS protein production by targeting FUS mRNA transcripts in the central nervous system.
  • Preclinical data suggests RAG-21 can mitigate motor neuron degeneration and improve outcomes for patients with FUS-ALS, a severe and rapidly progressing form of ALS.
  • RAG-21 shares a similar administration method with Ractigen's RAG-17, which has shown promising early clinical data in SOD1-mutated ALS patients.

Safety and Tolerance of RAG-17 in Amyotrophic Lateral Sclerosis Patients With SOD1 Gene Mutation

NCT05903690CompletedEarly Phase 1
Beijing Tiantan Hospital
Posted 5/24/2023

FDA Approves Clinical Trial of Smoked Cannabis for PTSD Treatment in Veterans

  • The FDA has approved a Phase 2 clinical trial to study the safety and efficacy of smoked cannabis for treating PTSD symptoms in veterans.
  • The trial, funded by the Michigan Veteran Marijuana Research Grant Program, will involve 320 veterans with moderate to severe PTSD.
  • Researchers will investigate the effects of self-titrated doses of high-THC cannabis flower compared to a placebo, mirroring real-world consumption.
  • The FDA's decision marks a shift towards recognizing the potential value of cannabis research and its impact on informing medical use.

FDA Approves Attruby (Acoramidis) for ATTR-CM, Reducing Cardiovascular Death and Hospitalization

• The FDA has approved Attruby (acoramidis) for treating adults with transthyretin-mediated amyloid cardiomyopathy (ATTR-CM), addressing both wild-type and variant forms of the disease. • Attruby, an oral TTR stabilizer, demonstrated a significant reduction in cardiovascular death and cardiovascular-related hospitalizations in the Phase 3 ATTRibute-CM trial. • The approval marks Attruby as the first and only therapy with a label specifying near-complete stabilization of transthyretin (TTR), targeting the underlying cause of ATTR-CM. • Clinical data showed Attruby improved quality of life and reduced all-cause mortality and cardiovascular hospitalization as early as three months after treatment initiation.

FDA Approves Vetmedin Solution: First Oral Solution for Canine Congestive Heart Failure

  • The FDA has approved pimobendan oral solution (Vetmedin Solution) for managing congestive heart failure (CHF) in dogs, caused by myxomatous mitral valve disease (MMVD) or dilated cardiomyopathy (DCM).
  • Vetmedin Solution marks the first oral solution approved by the FDA for this indication, offering an alternative formulation for easier administration, especially in smaller dogs.
  • Clinical studies have demonstrated that pimobendan can increase survival time and improve the quality of life in dogs with MMVD or DCM, with the new solution bioequivalent to existing chewable tablets.
  • Boehringer Ingelheim's Vetmedin product line now includes chewable tablets and this new oral solution, providing options for concurrent therapy in treating CHF in canines.

Ultromics' AI Software for Cardiac Amyloidosis Receives FDA Approval

  • Ultromics' artificial intelligence (AI) software has secured FDA approval for the diagnosis of cardiac amyloidosis, offering a new tool for identifying the disease.
  • The AI software analyzes echocardiograms to detect subtle indicators of cardiac amyloidosis, potentially leading to earlier and more accurate diagnoses.
  • This approval marks a significant advancement in the application of AI in cardiology, with the potential to improve patient outcomes through timely intervention.
  • The technology addresses a critical need for improved diagnostic tools for cardiac amyloidosis, a condition often diagnosed late due to its complex presentation.

Medtronic's Simplera CGM and InPen Insulin Delivery System Receive FDA Clearance

  • Medtronic's Simplera CGM, a sensor requiring no fingersticks or scanning, has been cleared by the FDA, enhancing diabetes management.
  • The InPen integrated smart insulin pen system, now compatible with Simplera CGM, also received FDA clearance, streamlining insulin delivery.
  • These approvals mark a significant advancement in diabetes technology, offering more convenient and personalized treatment options for patients.
  • The integration of Simplera CGM with InPen aims to improve glycemic control and simplify the daily lives of individuals with diabetes.

Pfizer's Hympavzi Receives EU Approval, Novartis Raises Sales Outlook

• The European Commission has approved Pfizer's Hympavzi (marstacimab) for hemophilia A and B treatment, based on positive Phase III BASIS study data. • Novartis has increased its mid-term sales growth guidance, projecting a 5% CAGR for 2024-2029, driven by key drugs and pipeline candidates. • Lilly's muvalaplin demonstrated significant reduction in lipoprotein(a) levels in a Phase II study, showing promise for cardiovascular risk reduction.

NIH's TrialGPT Algorithm Accelerates Patient Matching to Clinical Trials

  • The National Institutes of Health (NIH) has introduced TrialGPT, an algorithm designed to expedite the matching of patients to relevant clinical trials.
  • TrialGPT aims to reduce the time clinicians spend on patient-trial matching, allowing them to focus on tasks requiring more specialized expertise.
  • The algorithm represents a significant advancement in accelerating medical research by improving the efficiency of clinical trial recruitment.
  • Researchers suggest TrialGPT could substantially improve patient access to clinical trials, potentially leading to faster development of new therapies.

FDA Considers Accelerated Approval for RP1 Plus Nivolumab in Advanced Melanoma

• The FDA is reviewing a biologics license application for RP1 plus nivolumab to treat advanced melanoma after PD-1 inhibitor failure. • Phase 1/2 IGNYTE trial data showed a 32.7% overall response rate in patients treated with the combination therapy. • Replimune's RP1 received breakthrough therapy designation, expediting the review process for this potential new treatment option. • A phase 3 trial, IGNYTE-3, is underway to confirm the benefits of RP1 plus nivolumab in patients with advanced melanoma.

Study of RP1 Monotherapy and RP1 in Combination With Nivolumab

NCT03767348RecruitingPhase 2
Replimune Inc.
Posted 9/20/2017

VO and Nivolumab vs Physician's Choice in Advanced Melanoma That Progressed on Anti-PD-1 & Anti-CTLA-4 Drugs (IGNYTE-3)

NCT06264180RecruitingPhase 3
Replimune Inc.
Posted 7/11/2024

FDA Issues Complete Response Letter for Avacincaptad Pegol sNDA in Geographic Atrophy

  • The FDA issued a Complete Response Letter (CRL) to Astellas Pharma for the supplemental New Drug Application (sNDA) of avacincaptad pegol (IZERVAY).
  • The sNDA aimed to include new Phase 3 data on avacincaptad pegol's efficacy and safety over two years for treating geographic atrophy (GA).
  • The FDA's decision was based on statistical concerns regarding proposed labeling language, with no issues raised about the drug's safety or benefit-risk profile.
  • Astellas Pharma intends to work closely with the FDA to address the feedback and advance solutions for patients with GA.

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