MedPath

Clinical Trial News

Iterum Therapeutics Resubmits NDA for Oral Sulopenem to Treat Uncomplicated UTIs

  • Iterum Therapeutics has resubmitted its New Drug Application (NDA) to the FDA for oral sulopenem, targeting uncomplicated urinary tract infections (uUTIs) in adult women.
  • The resubmission includes data from Phase 3 trials (REASSURE, SURE 1, SURE 2, SURE 3) demonstrating oral sulopenem's efficacy and safety compared to Augmentin and ciprofloxacin.
  • If approved, oral sulopenem could be the first oral penem antibiotic in the U.S., offering a new treatment option for the estimated 40 million uUTIs annually.
  • The FDA is expected to complete its review in early Q4 2024, provided the resubmitted NDA addresses prior deficiencies.

Immix Biopharma's NXC-201 Receives EU Orphan Drug Designation for Multiple Myeloma

• Immix Biopharma's NXC-201 has been granted Orphan Drug Designation by the European Commission for treating multiple myeloma, a rare and life-threatening condition. • The designation provides NXC-201 with 10 years of market exclusivity in the EU upon approval, along with access to centralized authorization procedures. • This regulatory milestone underscores NXC-201's potential clinical impact, particularly for frail relapsed/refractory multiple myeloma patients with limited treatment options. • NXC-201 is currently being evaluated in the NEXICART-1 clinical trial, showing a favorable tolerability profile and potential for 'Single Day CRS'.

NXC-201 (formerly HBI0101) Multiple Myeloma

NCT04720313Active, Not RecruitingPhase 1
Hadassah Medical Organization
Posted 1/1/2021

FTC Intensifies Scrutiny of Orange Book Patent Listings in Pharmaceutical Industry

  • The Federal Trade Commission has challenged over 400 patents as improperly listed in the FDA's Orange Book, targeting drug-device patents for products including asthma inhalers and diabetes treatments.
  • Several pharmaceutical manufacturers have withdrawn patent listings following FTC warning letters, with some companies subsequently announcing caps on patient out-of-pocket costs for medications like asthma inhalers.
  • The FTC argues that improperly listed patents may violate antitrust laws by deterring generic competition, with Chair Lina Khan describing the practice as using "junk patent filings" that delay affordable medicine access.

FDA Approves HERCESSI™ (trastuzumab-strf) as Biosimilar to Herceptin for HER2-Overexpressing Cancers

• Accord BioPharma's HERCESSI™ (trastuzumab-strf), a biosimilar to Herceptin®, has received FDA approval for treating HER2-overexpressing breast and gastric cancers. • The approval was based on comprehensive data demonstrating similarity in efficacy, safety, and quality to Herceptin® (trastuzumab). • HERCESSI™ is indicated for adjuvant treatment of HER2-overexpressing breast cancer and metastatic gastric or gastroesophageal junction adenocarcinoma. • This marks Accord BioPharma's first FDA-approved biosimilar, with plans to introduce additional biosimilars in the U.S. market over the next five years.

FDA Approves XOLREMDI (mavorixafor) as First Targeted Therapy for WHIM Syndrome

  • The FDA has approved XOLREMDI (mavorixafor) for patients 12 years and older with WHIM syndrome, marking the first targeted therapy for this rare immunodeficiency.
  • XOLREMDI, a CXCR4 antagonist, demonstrated significant improvements in absolute neutrophil and lymphocyte counts in the pivotal 4WHIM Phase 3 clinical trial.
  • The approval was based on data showing increased time above threshold for neutrophil and lymphocyte counts, along with a reduction in infection rates compared to placebo.
  • X4 Pharmaceuticals has launched X4Connect to support patients in accessing XOLREMDI, which will be available through PANTHERx Rare specialty pharmacy.

Pfizer Launches Beqvez Gene Therapy for Hemophilia B with Novel Warranty Program at $3.5M

• Pfizer's Beqvez (fidanacogene elaparvovec) receives FDA approval as a one-time gene therapy for moderate to severe hemophilia B, priced at $3.5 million with a unique durability-based warranty program.
• Clinical data demonstrates sustained efficacy with patients remaining free from prophylactic factor IX therapy for up to five years after a single dose of Beqvez.
• The therapy offers potential cost advantages over traditional factor IX replacement treatments, which can cost $300,000-$1 million annually, while reducing treatment burden for patients.

India's CDSCO Rejects Pfizer's Clinical Trial Waiver for Multiple Myeloma Drug Elranatamab

Oncology
  • India's Subject Expert Committee has rejected Pfizer's request for a local clinical trial waiver for elranatamab, a bispecific antibody targeting relapsed and refractory multiple myeloma.
  • Elranatamab, which received accelerated approval from the US FDA based on Phase II data, is a BCMA-directed CD3 T-cell engager designed for patients who have failed multiple prior therapies.
  • The decision came after the SEC's oncology meeting on April 3-4, 2024, where they reviewed Pfizer's proposal to import and market the injection in 44mg/1.1mL and 76mg/1.9mL formulations.

FDA Approves Pfizer's $3.5 Million Gene Therapy Beqvez for Hemophilia B Treatment

Drug ApprovalRare Disease
  • The FDA approved Pfizer's Beqvez, a one-time gene therapy for adults with moderate to severe hemophilia B, marking the company's first gene therapy approval in the U.S.
  • The treatment carries a $3.5 million price tag before insurance and rebates, making it one of the most expensive drugs in the United States.
  • Beqvez enables patients to produce factor IX protein themselves, potentially eliminating the need for regular intravenous infusions administered multiple times per week or month.
  • The therapy will compete with CSL Behring's Hemgenix, another gene therapy for hemophilia B approved in 2022 with a similar $3.5 million price point.

FDA Approves Biktarvy Label Update for Use in Pregnant Adults with HIV Based on Study 5310 Data

  • The FDA has approved an updated label for Biktarvy, including data from Study 5310, reinforcing its safety and efficacy in pregnant individuals with HIV-1 who have suppressed viral loads.
  • Study 5310 evaluated Biktarvy's pharmacokinetics, safety, and efficacy in pregnant individuals with HIV, showing sustained viral suppression through pregnancy and postpartum.
  • The updated label makes Biktarvy the only second-generation INSTI-based single-tablet regimen with in-label clinical trial data and FDA approval for virologically suppressed pregnant adults.
  • Biktarvy is now recognized in DHHS perinatal guidelines as an alternative complete regimen for use in pregnancy and for individuals trying to conceive, or those already on the treatment.

Genethon's AAV Gene Therapy GNT0004 Shows Promise in Duchenne Muscular Dystrophy Trial

  • Genethon's GNT0004 gene therapy demonstrated good tolerability in boys with Duchenne muscular dystrophy when combined with transient immunological prophylactic treatment.
  • The trial data revealed up to 85 percent of muscle fibers expressing microdystrophin eight weeks post-injection in patients receiving the higher dose.
  • Patients treated with GNT0004 experienced a decrease in CPK levels, a biomarker of muscular distress, between 50 percent and 87 percent.
  • One-year efficacy results for the first patient in cohort 2 showed a positive clinical evolution, supporting further pivotal phase development in Europe.

MedPath

Empowering clinical research with data-driven insights and AI-powered tools.

© 2025 MedPath, Inc. All rights reserved.