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First Veteran Receives FDA-Approved Qalsody for SOD1-ALS at Houston VA Medical Center

NeurologyRare DiseaseDrug ApprovalFDA Approval
  • Roy Swearingen, a 65-year-old Marine Corps veteran from Texas, became the first veteran in the United States to receive Qalsody, an FDA-approved medication for SOD1-mutated ALS.
  • Qalsody represents the first approved treatment targeting a genetic cause of ALS, specifically for patients with mutations in the superoxide dismutase 1 (SOD1) gene, affecting 1-2% of ALS patients.
  • The Houston VA ALS Center's rapid implementation of this cutting-edge therapy demonstrates the VA's commitment to providing veterans with timely access to innovative treatments.
  • Veterans face higher ALS risk compared to the general population, making early detection and specialized care crucial for optimal quality of life management.

Vorasidenib Shows Promise as First Targeted Therapy for IDH-Mutant Low-Grade Gliomas

Targeted TherapyDrug ApprovalOncology
  • Vorasidenib, the first targeted therapy developed specifically for brain cancer, more than doubled progression-free survival in patients with recurrent grade 2 glioma carrying IDH1/IDH2 mutations, extending the time without disease progression from 11.1 months to 27.7 months.
  • The international INDIGO trial involving 331 patients demonstrated that vorasidenib delayed the need for chemotherapy and radiation by nearly 17 months compared to placebo, with 85.6% of patients going 18 months before requiring next treatment.
  • The drug showed excellent tolerability with limited adverse effects, offering a new treatment option for younger patients typically in their 30s and 40s who face cognitive deficits from standard radiation and chemotherapy treatments.
  • Results from this phase 3 study, published in the New England Journal of Medicine and presented at ASCO, are expected to establish a new standard of care for IDH-mutant low-grade gliomas pending FDA approval.

U.S. Tuberous Sclerosis Drug Market Projected to Reach $1.25 Billion by 2030, Growing at 23.1% CAGR

Drug ApprovalNeurologyRare Disease
  • The U.S. tuberous sclerosis drug market is expected to grow from $237.2 million in 2022 to $1.25 billion by 2030, driven by FDA approvals of new treatments and increasing disease prevalence.
  • FDA-approved treatments include Afinitor (everolimus) for patients aged 2 and above, approved in 2018, and Epidiolex (cannabidiol) for patients aged 1 and above, approved in 2020.
  • mTOR inhibitors are gaining significant traction in treatment protocols, showing effectiveness for internal tumors and skin lesions, with FDA approval for treating lung complications like Lymphangioleiomyomatosis (LAM) in 2015.

USDA Approves First Therapeutic Treatment for Canine Parvovirus Using Monoclonal Antibody Technology

Monoclonal AntibodyDrug Approval
  • The USDA has granted conditional license for Elanco's Canine Parvovirus Monoclonal Antibody, marking the first approved therapeutic solution to treat canine parvovirus.
  • In efficacy studies, all 28 treated puppies survived with significantly faster recovery times for vomiting, inappetence, and lethargy compared to traditional supportive care.
  • The single intravenous dose treatment could reduce hospitalization costs and make parvovirus treatment more accessible to pet owners who previously couldn't afford intensive care.
  • This breakthrough represents the first monoclonal antibody treatment for Elanco and addresses a critical unmet need in veterinary medicine for a disease with over 90% fatality rate if untreated.

FDA Approves Two Bispecific T-Cell Engagers for Relapsed/Refractory B-Cell Lymphomas

Drug ApprovalOncology
  • The FDA has granted accelerated approval to epcoritamab-bysp (Epkinly), the first T-cell engaging bispecific antibody for relapsed/refractory diffuse large B-cell lymphoma after two or more lines of systemic therapy.
  • Epcoritamab demonstrated a 61% overall response rate with a 38% complete response rate in the EPCORE NHL-1 trial, with a median duration of response of 15.6 months.
  • The FDA also approved glofitamab-gxbm (Columvi), another CD20/CD3-targeted bispecific T-cell engager, showing a 56% overall response rate and 43% complete response rate in clinical trials.
  • Both therapies carry boxed warnings for serious immune-related adverse reactions including cytokine release syndrome and immune effector cell-associated neurotoxicity syndrome.

A Study to Assess the Anti-Tumor Activity and Safety of Odronextamab in Adult Patients With B-cell Non-Hodgkin Lymphoma Who Have Been Previously Treated With Other Cancer Therapies

NCT03888105RecruitingPhase 2
Regeneron Pharmaceuticals
Posted 11/13/2019

Study to Investigate the Safety and Tolerability of Odronextamab in Patients With CD20+ B-Cell Malignancies

NCT02290951CompletedPhase 1
Regeneron Pharmaceuticals
Posted 1/9/2015

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

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

Novel BCMA-Targeted Therapies and Emerging Agents Transform Treatment Landscape for Relapsed/Refractory Multiple Myeloma

CAR-TDrug Approval
  • BCMA-directed therapies including CAR T-cell products ide-cel and cilta-cel, along with bispecific T-cell engager teclistamab, have demonstrated unprecedented efficacy in heavily pretreated patients with relapsed/refractory multiple myeloma.
  • Emerging bispecific antibodies elranatamab and talquetamab show promising response rates of 61-74% in triple-class refractory patients, offering new treatment options for those who have exhausted conventional therapies.
  • Novel agents including mezigdomide (a CELMoD), purinostat mesylate (HDAC inhibitor), and bispecific CS1-BCMA CAR T cells are expanding therapeutic options with manageable safety profiles.
  • The treatment paradigm is shifting toward earlier use of BCMA-targeted therapies and novel combinations, as patients increasingly become refractory to multiple drug classes including immunomodulators, proteasome inhibitors, and anti-CD38 antibodies.

Isatuximab, Pomalidomide, Elotuzumab and Dexamethasone in Relapsed And/or Refractory Multiple Myeloma

NCT04835129RecruitingPhase 2
Medical College of Wisconsin
Posted 1/10/2022

Selinexor and Backbone Treatments of Multiple Myeloma Patients

NCT02343042RecruitingPhase 1
Karyopharm Therapeutics Inc
Posted 10/1/2015

Trastuzumab Deruxtecan Gains Regulatory Approvals in China and Europe for HER2-Positive Cancers

Drug ApprovalMonoclonal Antibody
  • China's National Medical Products Administration approved trastuzumab deruxtecan for HER2-positive breast cancer patients who received prior anti-HER2 therapies, based on DESTINY-Breast03 trial showing 72% reduction in disease progression risk.
  • The European Commission approved trastuzumab deruxtecan as second-line treatment for HER2-positive advanced gastric cancer, marking the first antibody-drug conjugate approved in Europe for this indication.
  • DESTINY-Breast03 demonstrated superior efficacy with median progression-free survival not reached for trastuzumab deruxtecan versus 6.8 months for trastuzumab emtansine.
  • The approvals expand treatment options for patients with HER2-positive cancers who face poor outcomes after initial therapy progression.

Trastuzumab Deruxtecan (DS-8201a) Versus Investigator's Choice for HER2-low Breast Cancer That Has Spread or Cannot be Surgically Removed [DESTINY-Breast04]

NCT03734029Active, Not RecruitingPhase 3
Daiichi Sankyo
Posted 12/27/2018

FDA Approves Elacestrant as First Oral SERD for Advanced ER+/HER2- Breast Cancer

Drug Approval
  • The FDA has approved elacestrant (Orserdu), marking the first oral selective estrogen receptor degrader (SERD) to demonstrate improved efficacy over standard of care treatments in advanced breast cancer.
  • In the phase 3 EMERALD trial, elacestrant reduced the risk of progression or death by 30% in all patients and by 45% in patients with ESR1 mutations compared to standard endocrine therapy.
  • The approval provides a new oral treatment option for patients with estrogen receptor-positive/HER2-negative advanced or metastatic breast cancer following prior endocrine therapy including CDK4/6 inhibitors.
  • Elacestrant showed manageable safety profile with nausea being the most common adverse event, occurring in 35% of patients in the treatment arm.

Phase 2 Study of Amcenestrant (SAR439859) Versus Physician's Choice in Locally Advanced or Metastatic ER-positive Breast Cancer

NCT04059484TerminatedPhase 2
Sanofi
Posted 10/22/2019

Study of Abemaciclib and Elacestrant in Patients With Brain Metastasis Due to ER+/HER-2- Breast Cancer

NCT05386108RecruitingPhase 1
Stemline Therapeutics, Inc.
Posted 8/31/2022

A Study to Investigate Efficacy and Safety With Oral AZD9833 Compared With Intramuscular Fulvestrant in Post-menopausal Women at Least 18 Years of Age With Advanced ER-positive HER2 Negative Breast Cancer

NCT04214288Active, Not RecruitingPhase 2
AstraZeneca
Posted 4/22/2020

A Study of Imlunestrant, Investigator's Choice of Endocrine Therapy, and Imlunestrant Plus Abemaciclib in Participants With ER+, HER2- Advanced Breast Cancer

NCT04975308Active, Not RecruitingPhase 3
Eli Lilly and Company
Posted 10/4/2021

Study of Efficacy of Ribociclib After Progression on CDK4/6 Inhibition in Patients With HR+ HER2- Advanced Breast Cancer

NCT02632045CompletedPhase 2
Melissa K Accordino
Posted 3/1/2016

Phase 3 Trial of Elacestrant Versus Standard of Care for the Treatment of ER+/HER2- Advanced Breast Cancer

NCT03778931CompletedPhase 3
Stemline Therapeutics, Inc.
Posted 5/10/2019

ELACESTRANT in Women and Men With CDK4/6 Inhibitor-Naive Estrogen Receptor Positive, HER-2 Negative Metastatic Breast Cancer Study

NCT05596409Active, Not RecruitingPhase 2
Stemline Therapeutics, Inc.
Posted 5/19/2023

Open-Label Umbrella Study To Evaluate Safety And Efficacy Of Elacestrant In Various Combination In Participants With Metastatic Breast Cancer

NCT05563220RecruitingPhase 1
Stemline Therapeutics, Inc.
Posted 1/24/2023

A Study Evaluating the Efficacy and Safety of Giredestrant Compared With Physician's Choice of Endocrine Monotherapy in Participants With Previously Treated Estrogen Receptor-Positive, HER2-Negative Locally Advanced or Metastatic Breast Cancer (acelERA Breast Cancer)

NCT04576455Active, Not RecruitingPhase 2
Hoffmann-La Roche
Posted 11/27/2020

FDA Approves First CGRP-Targeting Therapy for Pediatric Migraine Prevention

Drug ApprovalMonoclonal Antibody
  • The FDA approved fremanezumab (Ajovy) for preventive treatment of episodic migraine in children and adolescents aged 6-17 years, marking the first anti-CGRP monoclonal antibody approved for pediatric use.
  • The Phase III SPACE trial demonstrated statistically significant reductions in monthly migraine days (-2.5 vs -1.4 with placebo) and a 47.2% responder rate compared to 27.0% with placebo.
  • This approval addresses a significant unmet need in pediatric migraine care, where approximately 7.7% of children and adolescents are affected but have had limited FDA-approved preventive treatment options.

Fremanezumab Compassionate Use Program for Pediatric Patients

NCT03539393available
Teva Branded Pharmaceutical Products R&D, Inc.

A Study to Test if Fremanezumab is Effective in Preventing Episodic Migraine in Patients 6 to 17 Years of Age

NCT04458857CompletedPhase 3
Teva Branded Pharmaceutical Products R&D, Inc.
Posted 7/15/2020

FDA's Accelerated Approval Program Shows Strong Performance Despite Recent Criticisms, Analysis Reveals

Drug ApprovalOncology
  • A comprehensive 30-year analysis of FDA's accelerated approval program demonstrates that 50% of accelerated approvals have been successfully converted to traditional approval in a median time of 3.2 years, indicating the pathway's effectiveness in providing early access to life-saving treatments.
  • The program has evolved significantly over three decades, with 83% of accelerated approvals in the last decade (2012-2021) being for oncology indications, reflecting advances in targeted therapies and established surrogate endpoints in cancer treatment.
  • While concerns exist about delayed confirmatory trials, only 12% of accelerated approvals have been withdrawn over the program's history, with FDA demonstrating improved oversight by reducing median withdrawal time from 10.4 years in the 1990s to 3.5 years in the most recent decade.
  • Government analysis reveals that Medicare and Medicaid spent over $18 billion from 2018-2021 on drugs with incomplete confirmatory trials past their original completion dates, highlighting the financial implications of program oversight challenges.

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