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Regeneron Secures FDA Approval for BCMA-Targeted Bispecific Antibody in Multiple Myeloma

Drug ApprovalOncology
  • Regeneron has received FDA approval for its BCMA-targeted bispecific antibody therapy for multiple myeloma treatment.
  • The approval allows Regeneron to enter the competitive US market for BCMA-targeted bispecific antibodies in multiple myeloma.
  • This regulatory milestone represents a significant achievement for Regeneron after a prolonged approval process.
  • The drug joins an increasingly crowded field of BCMA-targeted therapies for multiple myeloma patients.

CAR-T Cell Therapy Funding Surges to $141.2 Billion as Industry Expands Globally

Drug ApprovalCAR-TOncology
  • The CAR-T cell therapy industry has raised over $141.2 billion through various financing mechanisms, with estimates suggesting total industry funding could reach $281.7 billion when including undisclosed deals.
  • More than 170 companies worldwide are developing CAR-T products with 1,944 therapies in development, while 13 CAR-T cell therapies have received regulatory approval globally since 2017.
  • Despite a slowdown in IPOs and M&A activity in 2024, venture capital funding remains strong with 89 CAR-T companies securing $7.7 billion since 2014, supporting advancement in both blood cancer and solid tumor applications.

FDA Initiates PDUFA VIII Reauthorization Process with Public Meeting and Assessment Report

Drug ApprovalReal World Evidence
  • FDA has announced a hybrid public meeting on July 14, 2025, to begin the reauthorization process for PDUFA VIII, as the current authorization expires in September 2027.
  • The agency has completed an independent assessment of product quality information request communications between FDA and sponsors, with findings now available for public comment.
  • PDUFA VII introduced several enhancements including new meeting types, pilot programs for rare diseases and real-world evidence, and strengthened support for cell and gene therapy product development.

Vivo Capital Secures $740 Million for Third Cycle of Healthcare Investment Fund

Drug ApprovalRare DiseaseOncology
• Vivo Capital has closed the third cycle of its Opportunity Fund with over $740 million in commitments, focusing on preclinical and clinical-stage life sciences companies developing novel therapies.
• The fund has previously backed companies that achieved significant milestones, including FDA approvals for treatments targeting COPD, myelodysplastic syndrome, narcolepsy, and Prader-Willi Syndrome.
• Several Vivo-backed companies have been acquired by pharmaceutical giants in billion-dollar deals, including RayzeBio (Bristol-Myers Squibb, $4.1B), Chinook Therapeutics (Novartis, $3.2B), and Sierra Oncology (GSK, $1.9B).

FDA Approves Miplyffa for Niemann-Pick Type C Disease, Offering New Hope for Rare Genetic Disorder

Drug ApprovalRare DiseaseOrphan DrugFDA Approval
  • The FDA has approved Miplyffa, a new medication for treating Niemann-Pick Type C (NPC), a fatal genetic disorder affecting approximately 900 people in the United States.
  • Miplyffa works by protecting cells from fat accumulation and slowing disease progression, helping patients maintain neurological function for longer periods.
  • The drug is approved for children over two years old and has shown promising results in early patients, with some experiencing stabilization or improvement in disease markers within three months.
  • NPC is a progressive disorder that typically robs patients of their ability to speak, think clearly, swallow, walk, and move, often affecting children and young adults.

Jaguar Health Advances Canalevia-CA1 Field Study for Canine Cancer Treatment as Company Pursues Full FDA Approval

Drug ApprovalOncology
  • Jaguar Health has established the first study site for its field study of Canalevia-CA1, the FDA conditionally approved prescription drug for treating chemotherapy-induced diarrhea in dogs.
  • The company is pursuing two parallel goals: obtaining full FDA approval for CID treatment and expanding crofelemer's indication to treat general, non-infectious diarrhea in dogs.
  • Approximately 1 in 4 dogs develop cancer during their lifetime, with roughly 6 million new cancer diagnoses made in dogs annually in the U.S.
  • Jaguar is actively seeking partnerships to fund development and commercialization of crofelemer for general canine diarrhea treatment, representing a significant unmet medical need.

EMERALD Trial Data Reveals Duration of Prior CDK4/6 Inhibitor Therapy as Key Predictor for Elacestrant Success in ESR1-Mutant Breast Cancer

Drug ApprovalTargeted TherapyPrecision Medicine
  • The EMERALD trial demonstrated that patients with ESR1-mutant metastatic breast cancer who received CDK4/6 inhibitor therapy for more than 12 months achieved significantly better outcomes with elacestrant, with median progression-free survival of 8.6 months versus 3.8 months in optimal patients.
  • Elacestrant maintained efficacy across challenging disease presentations including visceral disease, multiple metastatic sites, and even in patients with traditionally poor prognostic factors like p53 mutations.
  • The FDA has approved elacestrant specifically for ESR1-mutant metastatic breast cancer, with new requirements for lipid profile monitoring before treatment initiation and as needed thereafter.

Atrasentan Receives FDA Accelerated Approval for IgA Nephropathy After Decades-Long Development Journey

Drug Approval
  • Atrasentan, an ETA receptor antagonist originally developed by Abbott in the 1990s for prostate cancer, has received FDA accelerated approval in 2025 for IgA nephropathy (IgAN).
  • The drug's development path included a pivot from oncology to chronic kidney diseases, with AbbVie conducting the large SONAR trial involving over 5,000 patients before out-licensing to Chinook.
  • Novartis acquired Chinook for $3.2 billion, demonstrating the significant commercial value placed on kidney disease therapeutics despite the complex development challenges.
  • The approval exemplifies the lengthy and complex process of advancing kidney therapies from bench to bedside, highlighting both the scientific and commercial hurdles in nephrology drug development.

A Study of Zidesamtinib (NVL-520) in Patients With Advanced NSCLC and Other Solid Tumors Harboring ROS1 Rearrangement (ARROS-1)

NCT05118789RecruitingPhase 1
Nuvalent Inc.
Posted 1/4/2022

Phase III Clinical Trials Reveal Advances in Non-Infectious Uveitis Treatment Options

Monoclonal AntibodyDrug Approval
  • Multiple phase III trials demonstrate that locally delivered corticosteroid implants, including fluocinolone acetonide and dexamethasone devices, effectively control inflammation in non-infectious uveitis but carry significant risks of glaucoma surgery and cataract formation.
  • Adalimumab emerges as a proven biologic therapy, with VISUAL I and II trials showing 50% reduction in treatment failure risk and 43% delay in uveitis relapse, though associated with higher rates of serious adverse events including infections.
  • Comparative studies reveal that intravitreal treatments outperform periocular injections for uveitic macular edema, while suprachoroidal triamcinolone acetonide offers promising results with lower intraocular pressure elevation risks.
  • Systemic immunomodulatory therapy demonstrates comparable efficacy to methotrexate and mycophenolate mofetil as first-line treatments, with the FAST trial showing similar 67% and 57% success rates respectively in achieving uveitis control.

A Study of the Safety and Efficacy of a New Treatment for Non-Infectious Intermediate or Posterior Uveitis

NCT00333814CompletedPhase 2
Allergan
Posted 5/1/2006

To Assess the Efficacy and Safety of Intravitreal Ranibizumab in People With Vision Loss Due to Macular Edema

NCT01846299CompletedPhase 3
Novartis Pharmaceuticals
Posted 10/1/2013

Safety and Efficacy of AIN457 in Patients With Active Non-infectious Uveitis

NCT01095250TerminatedPhase 3
Novartis Pharmaceuticals
Posted 4/1/2010

A Study of the Long-term Safety and Efficacy of Adalimumab in Subjects With Intermediate-, Posterior-, or Pan-uveitis

NCT01148225CompletedPhase 3
AbbVie
Posted 11/23/2010

PeriOcular and INTravitreal Corticosteroids for Uveitic Macular Edema Trial

NCT02374060CompletedPhase 3
JHSPH Center for Clinical Trials
Posted 6/16/2015

Phase III Study in Refractory Behcet's Disease

NCT00995709CompletedPhase 3
Novartis Pharmaceuticals
Posted 10/1/2009

First-line Antimetabolites as Steroid-sparing Treatment Uveitis Pilot Trial

NCT01232920CompletedPhase 3
University of California, San Francisco
Posted 10/1/2010

Efficacy and Safety of Adalimumab in Patients With Active Uveitis

NCT01138657CompletedPhase 3
AbbVie (prior sponsor, Abbott)
Posted 8/1/2010

Multicenter Uveitis Steroid Treatment (MUST) Trial

NCT00132691CompletedPhase 4
JHSPH Center for Clinical Trials
Posted 9/1/2005

Effect of Adalimumab for the Treatment of Uveitis in Juvenile Idiopathic Arthritis

NCT01385826CompletedPhase 2
Assistance Publique - Hôpitaux de Paris
Posted 6/29/2011

Safety and Efficacy of an Injectable Fluocinolone Acetonide Intravitreal Insert

NCT01694186CompletedPhase 3
EyePoint Pharmaceuticals, Inc.
Posted 8/2/2013

Safety and Efficacy of Fluocinolone Acetonide Intravitreal Implant vs Standardized Therapy

NCT00468871CompletedPhase 2
Bausch & Lomb Incorporated
Posted 4/1/2002

Study Assessing Double-masked Uveitis Treatment

NCT01358266CompletedPhase 3
Santen Inc.
Posted 5/1/2011

Suprachoroidal Injection of CLS-TA in Subjects With Macular Edema Associated With Non-infectious Uveitis

NCT02595398CompletedPhase 3
Clearside Biomedical, Inc.
Posted 11/17/2015

Safety and Efficacy of Fluocinolone Acetonide Intravitreal Implants

NCT00407082CompletedPhase 2
Bausch & Lomb Incorporated
Posted 12/1/2000

MAGNOLIA: Extension Study of Patients With Non-infectious Uveitis Who Participated in CLS1001-301

NCT02952001Completed
Clearside Biomedical, Inc.
Posted 12/13/2017

Methotrexate and Mycophenolate Mofetil for UVEITIS

NCT01829295CompletedPhase 3
University of California, San Francisco
Posted 8/1/2013

Safety and Efficacy Study of a FAI Insert in Subjects With Chronic Non-infectious Posterior Uveitis

NCT02746991CompletedPhase 3
EyePoint Pharmaceuticals, Inc.
Posted 6/2/2015

Macular Edema Ranibizumab v. Intravitreal Anti-inflammatory Therapy Trial

NCT02623426CompletedPhase 3
JHSPH Center for Clinical Trials
Posted 3/9/2017

Randomized, Controlled Trial to Test the Efficacy of Interferon Beta in the Treatment of Intermediate Uveitis

NCT00344253CompletedPhase 3
Heidelberg University
Posted 3/1/2006

Efficacy and Safety of Adalimumab in Subjects With Inactive Uveitis

NCT01124838CompletedPhase 3
AbbVie (prior sponsor, Abbott)
Posted 8/1/2010

Efficacy of Gevokizumab in the Treatment of Patients With Behçet's Disease Uveitis (EYEGUARD™-B)

NCT01965145TerminatedPhase 3
Institut de Recherches Internationales Servier
Posted 11/1/2012

Molecular Testing Advances Personalized Treatment Strategies in Ovarian Cancer

Targeted TherapyDrug Approval
  • Comprehensive biomarker testing in ovarian cancer now includes BRCA1/2, homologous recombination deficiency, HER2 status, and folate receptor alpha expression to guide personalized treatment decisions.
  • Next-generation sequencing is increasingly performed earlier in the disease course to identify actionable targets and support clinical trial enrollment for front-line therapy optimization.
  • Emerging biomarkers beyond BRCA/HRD include tumor-agnostic markers like MSI and TMB for immunotherapy response, and HER2 testing following April 2024 FDA approval of trastuzumab deruxtecan.
  • Novel predictive biomarkers such as KRAS for MEK/FAK inhibitor combinations and CCNE1 amplification associated with platinum resistance are being integrated into treatment planning.

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