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Clinical Trial News

New Video Resource Highlights Critical Insights from Rare Disease Patient Community

  • Bedrock Healthcare Communications and Origins Insights have released a new video resource featuring stakeholder interviews that illuminate the daily challenges faced by rare disease patients.
  • The initiative reveals that one in 17 people globally will be affected by a rare disease, impacting over 350 million people worldwide, emphasizing the need for healthcare systems to reevaluate their approach.
  • The video compilation includes perspectives from patients, healthcare networks, and pharmaceutical companies, providing valuable insights for drug developers and healthcare providers working on orphan drugs.

Off-the-Shelf NK Immunotherapy Shows Promise in B-Cell NHL Treatment

A phase 2 trial reveals that cord blood-derived natural killer (NK) immunotherapy, combined with high-dose chemotherapy and autologous stem cell transplant (ASCT), induces early antitumor response in B-cell non-Hodgkin lymphoma (NHL) patients. The study highlights the safety and potential of this novel treatment approach.

Umbilical Cord Blood NK Cells, Rituximab, High-Dose Chemotherapy, and Stem Cell Transplant in Treating Patients With Recurrent or Refractory B-Cell Non-Hodgkin's Lymphoma

NCT03019640CompletedPhase 2
M.D. Anderson Cancer Center
Posted 10/10/2017

A Reflection on the COVID-19 Vaccine

The article reflects on the emotional journey of a healthcare professional receiving the COVID-19 vaccine, highlighting the mix of fear, guilt, admiration, and hope experienced during the pandemic. It also discusses the importance of vaccines in saving lives and the personal responsibility to lead by example in promoting vaccination.

Grünenthal Leaders Outline Four Key Principles for Future Pharmaceutical Marketing in Post-COVID Era

  • Grünenthal's commercial leaders advocate for a fundamental shift in pharma marketing, emphasizing true customer centricity and data-driven decision making as core principles for future success.
  • The COVID-19 pandemic has accelerated digital transformation in pharmaceutical marketing, necessitating new approaches that combine artificial intelligence with human insights for better customer engagement.
  • Industry experts highlight the critical importance of fostering an innovative culture that balances experimentation with discipline, while maintaining psychological safety and strong leadership.

Ipsen Advances Neurotoxin Research for Spasticity Treatment, Aims to Develop Longer-Acting Therapies

  • Ipsen is developing modified botulinum toxin-based treatments with extended duration of action, potentially reducing injection frequency and improving patient quality of life.
  • Despite their proven efficacy in treating spasticity and other neurological conditions, botulinum neurotoxin treatments were deprioritized during COVID-19, highlighting the need for better recognition of their therapeutic value.
  • Over 100 conditions have been identified as potential targets for neurotoxin therapy, with current applications including adult and pediatric spasticity, cervical dystonia, and severe hyperhidrosis.

FDA Grants Breakthrough Status to Devices Targeting Alzheimer's, Heart Failure, and More

  • The FDA granted breakthrough device designation to Boston Scientific's Vercise deep brain stimulation (DBS) system for mild probable Alzheimer's in patients 65 and older.
  • Cognito Therapeutics received breakthrough designation for its neurostimulation device, designed to reactivate the brain's immune system by stimulating it at a specific frequency.
  • Puzzle Medical Devices' ModulHeart, a minimally invasive transcatheter heart pump, received breakthrough status, offering a potential alternative to open-heart surgery for heart failure patients.

FDA Approves Prucalopride for Chronic Idiopathic Constipation

The FDA has approved prucalopride (Motegrity) for treating chronic idiopathic constipation (CIC), offering a new treatment option that enhances colonic peristalsis to increase bowel motility. This approval is based on clinical studies showing significant improvements in patients.

Jazz Pharmaceuticals Acquires GW Pharma in $7.2B Cannabis Medicine Deal

  • Jazz Pharmaceuticals has agreed to acquire GW Pharmaceuticals for $7.2 billion, marking a significant expansion into cannabis-derived medicines and creating a leader in neuroscience therapeutics.
  • GW's flagship product Epidiolex, the first FDA-approved cannabis-based medicine, achieved $510 million in sales in 2020 and holds patent protection until at least 2035.
  • The acquisition diversifies Jazz's portfolio beyond its narcolepsy drug Xyrem and includes GW's late-stage pipeline candidate nabiximols, currently in Phase 3 trials for MS spasticity.

Disease Rarity and Clinical Benefits Drive Rare Disease Drug Pricing in Germany's AMNOG System

  • Germany's AMNOG system evaluates rare disease drugs based on multiple factors, with drugs treating ultra-rare conditions generally commanding higher prices while staying below €50 million annual sales threshold.
  • Analysis of over 20 rare disease drugs reveals that G-BA benefit ratings significantly influence pricing, with treatments like Spinraza and Orkambi securing higher reimbursements due to demonstrated clinical benefits.
  • Clinical trial design and comparator choice show less impact on pricing decisions, with German payers focusing more on data robustness and incremental benefits regardless of trial methodology.

BMS Seeks FDA Approval for Zeposia in Ulcerative Colitis with May Decision Date

  • Bristol-Myers Squibb has submitted Zeposia (ozanimod) for FDA review in treating moderate to severe ulcerative colitis, with a priority review decision expected by May 30.
  • In clinical trials, Zeposia demonstrated significant efficacy with 18.4% of patients achieving clinical remission at 10 weeks compared to 6.8% on placebo, and 37% maintaining remission at one year.
  • If approved, Zeposia would become the first S1P modulator for ulcerative colitis, potentially expanding BMS's market presence beyond its current multiple sclerosis indication.

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