Carol Roberts, President of The PBCers Organization, commented on the approval, saying, “The availability of a new treatment option that can help reduce this intense itching while also improving biomarkers of active liver disease is a milestone for our community.” Gilead’s CEO, Daniel O’Day, highlighted the significance of Livdelzi, stating, “People living with PBC have been waiting for treatment advancements for many years. Today’s approval of Livdelzi, with its distinct profile, provides them with an important new option.”
Livdelzi, an oral PPAR delta agonist, is set to challenge the current PBC standard of care, which often falls short for many patients. The drug has shown a durable efficacy and safety profile across several studies, including the long-term open-label ASSURE study and the ongoing confirmatory Phase III AFFIRM study. The RESPONSE study demonstrated Livdelzi’s ability to improve both pruritus and key biomarkers of cholestasis, making it a significant advancement in the treatment of PBC.
Incyte, Syndax bag FDA Approval for Potential Blockbuster Niktimvo in Graft-versus-host Disease
Incyte and Syndax Pharmaceuticals announced FDA approval for Niktimvo (axatilimab-csfr), an innovative anti-CSF-1R antibody for chronic graft-versus-host disease (GVHD). This marks Niktimvo as the first anti-CSF-1R antibody approved to target inflammation and fibrosis associated with chronic GVHD. The approval is supported by the pivotal AGAVE-201 study, which demonstrated that Niktimvo provided durable responses across various organs and patient subgroups.
Hervé Hoppenot, CEO of Incyte, highlighted the significance of this development, stating, “With the approval of Niktimvo, patients with chronic GVHD whose disease has progressed after prior therapies now have a new treatment option with a novel mechanism of action.” Similarly, Michael A. Metzger, CEO of Syndax, emphasized, “The approval of Niktimvo represents a significant treatment advancement for patients with chronic GVHD who have failed at least two lines of previous therapy.”
The AGAVE-201 study involved 241 patients with refractory chronic GVHD who had previously received at least two lines of systemic therapy. Results showed that 75% of patients receiving Niktimvo at the approved dose achieved an overall response rate within the first six months, with a median time to response of 1.5 months. However, 44% of patients experienced serious adverse reactions, including infections and respiratory issues. Despite this, the trial’s findings provide a promising new treatment avenue for this challenging condition.
Niktimvo will be co-commercialized by Incyte and Syndax in the U.S., with Incyte holding exclusive rights outside the U.S. The companies plan to seek approval for smaller vial sizes to facilitate dosing and minimize waste, with an anticipated U.S. launch in early Q1 2025.
FDA Lifts Partial Hold on BioNTech, MediLink’s ADC Cancer Trial
The FDA has lifted the partial clinical hold on MediLink Therapeutics’ Phase I trial evaluating BNT326/YL202 (NCT05653752), a HER3-targeting antibody-drug conjugate developed in collaboration with BioNTech SE. This decision follows a comprehensive response from MediLink that included data analysis, updates to the investigator brochure, informed consent forms, and an amended clinical trial protocol with additional risk mitigation measures. The trial will now resume recruitment, focusing on dose levels up to 3 mg/kg, where safety was manageable and clinical activity promising.
The partial hold was initially imposed due to observed dose-dependent treatment-related adverse events (TRAEs), particularly neutropenia and mucositis. The study sponsor addressed these TRAEs, which are common with established chemotherapies, through proactive measures such as dose adjustments and updated safety protocols. The updated clinical trial protocol includes revised guidance on dose modifications and prophylactic treatments to mitigate these risks.
BioNTech and MediLink remain committed to advancing BNT326/YL202 for solid tumors with significant unmet medical needs, ensuring patient safety while exploring the potential of this promising candidate.
FDA Approves IMFINZI for the Treatment of Resectable Non-Small Cell Lung Cancer Before and After Surgery
AstraZeneca’s IMFINZI (durvalumab) has received FDA approval for the treatment of adult patients with resectable early-stage (IIA-IIIB) non-small cell lung cancer (NSCLC) who do not have known epidermal growth factor receptor (EGFR) mutations or anaplastic lymphoma kinase (ALK) rearrangements. The regimen includes IMFINZI in combination with neoadjuvant chemotherapy before surgery and as adjuvant monotherapy after surgery.
The approval is supported by positive results from the pivotal AEGEAN trial, published in The New England Journal of Medicine in October 2023. The trial’s interim analysis demonstrated a 32% reduction in the risk of recurrence, progression, or death with the IMFINZI-based regimen compared to chemotherapy alone (EFS hazard ratio of 0.68). Additionally, the final analysis showed a pathologic complete response (pCR) rate of 17.2% with IMFINZI plus neoadjuvant chemotherapy, significantly higher than the 4.3% pCR rate for chemotherapy alone.
John V. Heymach, MD, PhD, from The University of Texas MD Anderson Cancer Center, highlighted that this approval offers a crucial new treatment option that could become a standard approach for patients with resectable NSCLC, addressing the high recurrence rates seen with current treatments. Dave Fredrickson, Executive Vice President at AstraZeneca, emphasized that this approval extends IMFINZI’s impact from unresectable Stage III disease to early-stage lung cancer, aligning with their goal of curative treatment.
IMFINZI is also approved in the UK, Switzerland, and Taiwan for this indication, and regulatory reviews are ongoing in the EU, China, and other regions. As per the PACIFIC Phase III trial, it remains a global standard for unresectable, Stage III NSCLC.
FDA Grants Tentative Approval of YUTREPIA™ for Pulmonary Arterial Hypertension and PH-ILD
Liquidia Corporation announced that the FDA granted tentative approval for YUTREPIA (treprostinil) inhalation powder. This therapy is intended for adults with pulmonary arterial hypertension (PAH) and pulmonary hypertension associated with interstitial lung disease (PH-ILD). Tentative approval means that YUTREPIA has met all necessary regulatory standards for quality, safety, and efficacy but must wait for the expiration of exclusivity granted to a competing product before it can receive final approval.
Dr. Roger Jeffs, Ph.D., CEO of Liquidia, expressed satisfaction with the FDA’s acknowledgment of their NDA amendment but voiced disappointment over the FDA granting regulatory exclusivity to United Therapeutics for Tyvaso DPI. This exclusivity covers the chronic use of dry-powder formulations of treprostinil for three years. Liquidia plans to challenge this exclusivity decision to ensure timely patient access to YUTREPIA.
The tentative approval is supported by results from the Phase III INSPIRE trial, which demonstrated that YUTREPIA is safe and well-tolerated for both new users and those switching from nebulized treprostinil. Published findings confirm that YUTREPIA meets regulatory standards for manufacturing and quality. Liquidia remains dedicated to addressing the needs of patients with PAH and PH-ILD and aims to secure final approval for YUTREPIA as soon as possible.
