Clinical Trial News
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- Several pharmaceutical manufacturers have withdrawn patent listings following FTC warning letters, with some companies subsequently announcing caps on patient out-of-pocket costs for medications like asthma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.
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'.
Highlighted Clinical Trials:
Hadassah Medical Organization
Posted 1/1/2021
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
- 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 Biktarvy Label Update With Data for Pregnant Adults with HIV
The FDA has approved an updated label for Biktarvy, including data supporting its safety and efficacy for treating pregnant individuals with HIV-1 who have suppressed viral loads. This update is based on Study 5310, which evaluated Biktarvy's pharmacokinetics, safety, and efficacy during pregnancy and postpartum.
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.
First Personalized mRNA Vaccine for Melanoma Enters UK Clinical Trials
- British patient Steve Young becomes one of the first to receive a groundbreaking personalized mRNA vaccine designed to prevent melanoma recurrence after surgical removal of tumors.
- The vaccine, mRNA-4157 (V940), is custom-built to match each patient's unique tumor genetic signature and works alongside immunotherapy drug pembrolizumab to help the immune system target remaining cancer cells.
- Phase III trials are underway across eight UK centers, aiming to recruit 60-70 patients, with experts suggesting this technology could potentially revolutionize treatment for melanoma and other cancers including lung, bladder, and kidney tumors.
TEASE-3 Study Reports Positive Interim Data for Stargardt Disease Treatment
Alkeus Pharmaceuticals Inc. has announced encouraging interim results from the TEASE-3 study, showing that gildeuretinol (ALK-001) halted the progression of early-stage Stargardt disease in three teenagers for up to 6 years. This marks a significant advancement in treating a disease that leads to vision loss and blindness due to a genetic mutation causing material buildup in the retina.
Advancing Cancer Treatment: The Promise of Oncolytic Virotherapy Combinations
• Oncolytic viruses (OVs) selectively replicate within tumor cells, causing direct cell lysis while simultaneously triggering immune responses against cancer, offering a dual mechanism of action against malignancies.
• While OV monotherapy has shown limited efficacy in clinical trials, combining oncolytic viruses with other treatment modalities such as immune checkpoint inhibitors, chemotherapy, and cell therapies has demonstrated synergistic anti-tumor effects.
• Recent advances in engineering oncolytic vaccinia viruses to express immunomodulatory factors, including cytokines and antibodies targeting immune checkpoints, have shown promising results in remodeling the tumor microenvironment and enhancing therapeutic outcomes.