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FDA Denies Approval for Elamipretide Despite Dramatic Heart Function Improvements in Barth Syndrome Patients

Rare DiseaseDrug ApprovalOrphan Drug
  • The FDA denied approval for elamipretide, a drug treating Barth syndrome, despite an advisory committee's 10-6 recommendation for approval and documented heart function improvements from 20% to normal levels.
  • One-year-old Jaylen Karle experienced heart failure from birth due to Barth syndrome, but his heart function normalized within a month of starting elamipretide treatment in early 2024.
  • Barth syndrome affects approximately 150 people in the U.S., and families fear losing access to the experimental drug if FDA approval doesn't come within six months.
  • The FDA provided a pathway for accelerated approval, but advocates call the denial "unconscionable" given the drug's life-saving potential for this rare genetic disorder.

Researchers Unveil New Strategies to Harness Tumor Microenvironment for Enhanced Cancer Immunotherapy

Drug Approval
  • Researchers at the 2025 ASCO Annual Meeting revealed how the tumor microenvironment's complex balance of pro-tumor and anti-tumor immune forces can be manipulated to improve immunotherapy responses.
  • A clinical study combining the JAK inhibitor itacitinib with pembrolizumab achieved a 67% overall response rate in non-small cell lung cancer patients by resetting chronic interferon signaling.
  • The dual blockade of PD-1 and LAG3 with relatlimab plus nivolumab produces "marathon runner" T-cells with enhanced durability compared to PD-1 inhibition alone.

Study of Mogamulizumab + MEDI4736 (Durvalumab) and Mogamulizumab + Tremelimumab in Subjects w/ Advanced Solid Tumors

NCT02301130CompletedPhase 1
Kyowa Kirin, Inc.
Posted 11/26/2014

A Study to Evaluate the Efficacy and Safety of Simtuzumab Combined With Gemcitabine for Metastatic Pancreatic Adenocarcinoma

NCT01472198CompletedPhase 2
Gilead Sciences
Posted 11/1/2011

Phase 1b Study of Dato-DXd in Combination With Immunotherapy With or Without Carboplatin in Advanced or Metastatic Non-Small Cell Lung Cancer

NCT04612751Active, Not RecruitingPhase 1
AstraZeneca
Posted 2/2/2021

Study of E7046 in Subjects With Selected Advanced Malignancies

NCT02540291TerminatedPhase 1
Eisai Inc.
Posted 7/30/2015

A Phase 3 Study of Pembrolizumab + Epacadostat or Placebo in Subjects With Unresectable or Metastatic Melanoma (Keynote-252 / ECHO-301)

NCT02752074CompletedPhase 3
Incyte Corporation
Posted 6/21/2016

HS-20089 in Patients With Ovarian Cancer and Endometrial Cancer

NCT06014190RecruitingPhase 2
Hansoh BioMedical R&D Company
Posted 12/18/2023

A Study of Safety, Pharmacokinetics, Pharmacodynamics of JNJ-61610588 in Participants With Advanced Cancer

NCT02671955TerminatedPhase 1
Janssen Research & Development, LLC
Posted 1/1/2016

Basket Study to Evaluate the Therapeutic Activity of Simlukafusp Alfa as a Combination Therapy in Participants With Advanced and/or Metastatic Solid Tumors

NCT03386721TerminatedPhase 2
Hoffmann-La Roche
Posted 2/19/2018

A Study To Investigate The Safety And Toleration Of A Single Dose Of PF-04418948 In Healthy Volunteers

NCT01002963CompletedPhase 1
Pfizer
Posted 9/1/2009

Novartis Secures FDA Approval for Leqvio, First Twice-Yearly Cholesterol Drug

Drug Approval
  • Novartis has received FDA approval for Leqvio (inclisiran), a first-in-class siRNA therapy that reduces LDL cholesterol with just two injections per year.
  • The drug achieved up to 52% LDL cholesterol reduction in phase 3 trials when added to statins in patients with high cholesterol and atherosclerotic cardiovascular disease.
  • Approximately 16 million Americans with ASCVD taking statins are not reaching their recommended LDL cholesterol targets, representing a significant market opportunity.
  • The approval comes after a one-year delay due to manufacturing facility issues, with Novartis transferring production to its own Austrian facility.

MHRA Grants Conditional Approval for CAR-T Therapy Aucatzyl to Treat Relapsed B-Cell ALL

CAR-TDrug Approval
  • The MHRA granted conditional marketing authorization for obecabtagene autoleucel (Aucatzyl), a CAR-T cell therapy for adult patients with relapsed or refractory B-cell precursor acute lymphoblastic leukemia on April 25, 2025.
  • The European Medicines Agency's CHMP adopted a positive opinion for Aucatzyl on May 22, 2025, recommending conditional approval for adults aged 26 years and older with the same indication.
  • B-cell precursor ALL is a blood cancer affecting white blood cells where bone marrow produces abnormal, immature B-lymphocytes that grow and divide rapidly.
  • This approval represents a significant advancement in CAR-T cell therapy for treating this aggressive form of blood cancer in patients who have failed previous treatments.

CDSCO Requests Additional Clinical Data from Fresenius for Calcium Chloride Infusion Approval in India

Drug Approval
  • Fresenius submitted a proposal to manufacture and market calcium chloride dihydrate 100 mmol/l solution for infusion in India for use in renal replacement therapies and plasma exchange procedures.
  • The company sought waivers for Phase III and Phase IV clinical trials, citing existing approvals in Portugal, UK, Brazil, Switzerland, France, Denmark and other European countries.
  • India's SEC committee for Renal recommended that Fresenius submit basis of approval, Phase III clinical trial data, and post-marketing surveillance data from approved countries before further consideration.
  • The calcium chloride infusion is indicated for calcium substitution in CRRT, SLEDD, and TPE procedures using citrate anticoagulation in both adults and children.

China Approves First Domestically Developed 9-Valent HPV Vaccine, Becomes Second Country with Independent High-Valency HPV Production

Drug Approval
  • China has approved its first domestically developed nine-valent HPV vaccine, Cecolin 9, making it only the second country after the United States with independent high-valency HPV vaccine production capability.
  • Clinical trials involving over 11,000 participants demonstrated over 98% protection against persistent HPV infections and 100% protection against cervical infections for the targeted viral strains.
  • The vaccine offers a two-dose regimen for girls aged 9-17, providing immune responses comparable to three-dose schedules in older populations, with efficacy lasting at least 30 months.
  • This achievement builds on China's previous success with its domestically produced two-valent HPV vaccine, which received WHO prequalification in 2021 and has entered 21 international markets.

Epcoritamab Demonstrates Durable 3-Year Remissions in Relapsed/Refractory Large B-Cell Lymphoma

Drug ApprovalOncology
  • Epcoritamab-bysp achieved durable complete responses in patients with relapsed/refractory large B-cell lymphoma, with 96% of complete responders at 2 years maintaining remission at 3 years.
  • The median duration of complete response was 36.1 months, with the longest ongoing complete response exceeding 43 months in the EPCORE NHL-1 study.
  • Among patients who discontinued treatment for reasons other than disease progression, complete response was maintained for a median of 14 months after stopping therapy.
  • The bispecific T-cell engager demonstrated manageable safety profile with COVID-19, cytokine release syndrome, and diarrhea as the most common treatment-emergent adverse events.

First-in-Human (FIH) Trial in Patients With Relapsed, Progressive or Refractory B-Cell Lymphoma

NCT03625037Active, Not RecruitingPhase 1
Genmab
Posted 6/26/2018

A Safety and Preliminary Efficacy Study of CC-99282, Alone and in Combination With Anti-lymphoma Agents in Participants With Relapsed or Refractory Non-Hodgkin Lymphomas (R/R NHL)

NCT03930953Active, Not RecruitingPhase 1
Celgene
Posted 5/20/2019

A Study Comparing the Efficacy and Safety of Polatuzumab Vedotin With Rituximab-Cyclophosphamide, Doxorubicin, and Prednisone (R-CHP) Versus Rituximab-Cyclophosphamide, Doxorubicin, Vincristine, and Prednisone (R-CHOP) in Participants With Diffuse Large B-Cell Lymphoma

NCT03274492Active, Not RecruitingPhase 3
Hoffmann-La Roche
Posted 11/16/2017

Open Label Study to Evaluate the Safety and Efficacy of Lenalidomide With MOR00208 in Patients With R-R DLBCL

NCT02399085CompletedPhase 2
MorphoSys AG
Posted 3/29/2016

A Study to Evaluate Adverse Events and Change in Disease Activity of Subcutaneous (SC) Epcoritamab in Combination With Oral and Intravenous Anti-Neoplastic Agents in Adult Participants With Non-Hodgkin Lymphoma

NCT05283720RecruitingNot Applicable
Genmab
Posted 6/14/2022

Study to Compare the Effectiveness and Safety of Golcadomide Plus R-CHOP vs Placebo Plus R-CHOP in Participants With Previously Untreated High-risk Large B-cell Lymphoma

NCT06356129RecruitingPhase 3
Celgene
Posted 6/19/2024

Safety and Efficacy Trial of Epcoritamab Combinations in Subjects With B-cell Non-Hodgkin Lymphoma (B-NHL)

NCT04663347Active, Not RecruitingPhase 1
Genmab
Posted 11/3/2020

The Pathogenesis and Prognostic Factors of Lymphoma

NCT06203652Recruiting
Shanghai Zhongshan Hospital
Posted 6/5/2023

Trump Administration Halts Major HIV Vaccine Research Programs Despite Scientific Progress

Drug Approval
  • The Trump administration has terminated funding for multiple HIV vaccine research programs, including major efforts at Duke Human Vaccine Institute and Scripps Research Institute that have been running since 2012.
  • Scientists warn the cuts represent a decade-long setback for HIV vaccine development, coming at a critical time when researchers report seeing "light at the end of the tunnel" with promising clinical trial results.
  • The decision affects 27 separate HIV/AIDS programs totaling $7.5 billion in funding, with HHS citing "duplication of efforts" as justification for prioritizing currently available HIV prevention approaches.
  • The timing coincides with the FDA's upcoming June 19 decision on lenacapavir, a twice-yearly injectable HIV prevention drug by Gilead Sciences that showed 100% effectiveness in preventing transmission.

Study of Lenacapavir for HIV Pre-Exposure Prophylaxis in People Who Are at Risk for HIV Infection

NCT04925752Active, Not RecruitingPhase 3
Gilead Sciences
Posted 6/28/2021

Investor Lawsuit Challenges Novartis' $1.7 Billion Acquisition of Regulus Therapeutics Over Kidney Disease Drug

Drug Approval
  • A shareholder lawsuit seeks to block Novartis' acquisition of Regulus Therapeutics, alleging the deal primarily benefits company insiders rather than public stockholders.
  • The $1.7 billion transaction includes Regulus' lead candidate farabursen for treating the most common inherited kidney disease, with regulatory approval triggering additional payments.
  • The complaint claims Regulus violated securities laws by omitting critical information about the sales process, conflicts of interest, and financial projections in its recommendation statement.
  • The acquisition is structured as $7 per share upfront plus contingent value rights of up to $7 per share upon farabursen's regulatory approval.

Mabwell's Novel Long-Acting G-CSF Drug MAILISHENG Receives NMPA Approval for Chemotherapy-Induced Neutropenia

Drug ApprovalOncology
  • Mabwell received NMPA approval for Albipagrastim alfa (MAILISHENG), marking China's first novel G-CSF drug developed with albumin long-acting fusion technology.
  • Phase III trial data in 496 breast cancer patients demonstrated superior efficacy with 42% relative risk reduction in febrile neutropenia compared to control.
  • The drug addresses a significant unmet need, as 65.5% of patients receiving certain chemotherapy regimens experience grade 3 or 4 neutropenia.
  • MAILISHENG represents Mabwell's first commercialized innovative drug, utilizing human serum albumin fusion to extend half-life and reduce dosing frequency.

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