FDA Approves Boehringer’s JASCAYD as First New IPF Therapy in Over a Decade
Boehringer Ingelheim’s JASCAYD (nerandomilast) has received FDA approval as an oral treatment for adults with idiopathic pulmonary fibrosis (IPF), marking the first new therapy for the condition in over a decade. JASCAYD is the first and only preferential PDE4B inhibitor approved for IPF, offering a novel mechanism that delivers both antifibrotic and immunomodulatory effects to slow lung function decline.
“This milestone represents a new era in the treatment of IPF, a rare and debilitating chronic condition that worsens lung function,” said Dr. Toby Maher, Professor of Clinical Medicine at the Keck School of Medicine, USC. “Nerandomilast is a welcome new treatment option with a well-tolerated safety profile for physicians to consider for appropriate patients.”
Approval is supported by results from two clinical trials, FIBRONEER-IPF (NCT05321069) and Trial 2 (NCT04419506). In the phase III FIBRONEER-IPF study, nerandomilast significantly reduced the decline in Forced Vital Capacity (FVC) at week 52 compared to placebo. Patients on nerandomilast 18 mg and 9 mg showed mean declines of -106 mL and -122 mL, respectively, versus -170 mL in the placebo group.
“The FDA approval of nerandomilast is a pivotal moment for people living with IPF,” said Shashank Deshpande, Chairman of the Board of Managing Directors and Head of Human Pharma at Boehringer Ingelheim. “This new step forward, driven by the compelling results of the FIBRONEER-IPF trial, underscores our unwavering commitment to change the way we treat IPF.”
The most common adverse events (≥5%) included diarrhea, upper respiratory infections, depression, and decreased appetite, with higher rates of discontinuation seen in the nerandomilast groups compared to placebo. However, the product label includes no ‘Warnings and Precautions’ section. “There has been a long-standing need for new treatment options for IPF,” added Scott Staszak, President and CEO of the Pulmonary Fibrosis Foundation. “Nerandomilast provides an important addition to the care landscape.”
Denali Therapeutics’ Tividenofusp Alfa BLA Review for Hunter Syndrome Extended by FDA
Denali Therapeutics announced that the FDA has extended the review timeline for its Biologics License Application (BLA) seeking accelerated approval of tividenofusp alfa for mucopolysaccharidosis type II (MPS II), also known as Hunter syndrome. The Prescription Drug User Fee Act (PDUFA) date has been moved from January 5, 2026, to April 5, 2026.
The extension follows Denali’s submission of updated clinical pharmacology data in response to an FDA request. The agency classified the submission as a Major Amendment (MA), which, per standard procedure, triggered a three-month review extension. The company noted that the amendment does not affect the clinical pharmacology or benefit-risk profile of the BLA, and no new data were requested.
“We appreciate the FDA’s continued collaboration throughout the review process,” said Ryan Watts, Ph.D., CEO of Denali Therapeutics. “We continue to prepare for the potential approval and commercial launch of tividenofusp alfa. We feel the urgency to deliver for the MPS community.”
Tividenofusp alfa is being evaluated as a potential first-in-class therapy for MPS II, a rare genetic disorder with significant unmet medical need. Denali remains engaged with regulatory authorities and stakeholders as it advances toward a possible FDA decision in 2026.
Bicara Therapeutics announced that the FDA has granted Breakthrough Therapy Designation (BTD) to ficerafusp alfa in combination with pembrolizumab for first-line treatment of patients with metastatic or unresectable, recurrent head and neck squamous cell carcinoma (HNSCC) whose tumors are HPV-negative and have a PD-L1 combined positive score (CPS) ≥1.
“BTD provides external validation of the importance of ficerafusp alfa’s best-in-disease potential, and solidifies the foundation for our ongoing pivotal trial, FORTIFI-HN01,” said Claire Mazumdar, PhD, MBA, CEO of Bicara Therapeutics. “We look forward to working closely with the FDA to bring this therapy to patients as quickly as possible.”
The designation is supported by data from multiple phase I/Ib dose cohorts, most recently presented at the 2025 ASCO Annual Meeting. Results showed a median duration of response of 21.7 months and median overall survival of 21.3 months, with a favorable safety profile, highlighting the combination’s potential to provide durable benefit in a population with limited options.
“This recognition highlights the urgent unmet need for patients with HPV-negative R/M HNSCC,” added Dr. David Raben, Chief Medical Officer at Bicara. “It reinforces our conviction that the synergy between ficerafusp alfa and pembrolizumab can drive deeper, longer-lasting responses in this challenging cancer type.”
BeOne Medicines’ Sonrotoclax Granted FDA Breakthrough Designation for Cancer Treatment
BeOne Medicines announced that the FDA has granted Breakthrough Therapy Designation (BTD) to sonrotoclax, its next-generation investigational BCL2 inhibitor, for the treatment of adults with relapsed or refractory (R/R) mantle cell lymphoma (MCL). In addition, the FDA has accepted BeOne’s application to participate in Project Orbis, a global regulatory initiative that allows concurrent review of oncology products across multiple countries.
“Breakthrough Therapy Designation is reserved for medicines with the potential to transform outcomes for patients with serious diseases,” said Julie Lepin, Senior Vice President and Chief Regulatory Affairs Officer at BeOne. “This recognition affirms the strength of the emerging data for sonrotoclax and its potential to become a new standard of care for people with relapsed or refractory mantle cell lymphoma.”
The FDA’s decisions are based on results from the ongoing Phase I/II BGB-11417-201 study, which is evaluating sonrotoclax in R/R MCL patients previously treated with a BTK inhibitor and anti-CD20 therapy. Topline results suggest deep and durable responses, and full data are expected at an upcoming medical meeting. A Phase III confirmatory trial, CELESTIAL-RRMCL, is currently underway.
This marks the first BTD for sonrotoclax and the second for BeOne’s hematology pipeline. The investigational therapy has also received Fast Track Designation and Orphan Drug Designation in MCL and other B-cell malignancies, including WM, MM, and AML, supporting BeOne’s commitment to advancing care for patients with hard-to-treat blood cancers.
LIBTAYO Approved in U.S. as First and Only Adjuvant Immunotherapy for High-Risk CSCC Post-Surgery and Radiation
Regeneron Pharmaceuticals announced that the FDA has approved LIBTAYO (cemiplimab-rwlc) as an adjuvant treatment for adults with cutaneous squamous cell carcinoma (CSCC) at high risk of recurrence following surgery and radiation. This marks the first immunotherapy approved in this setting and was evaluated under Priority Review due to its potential to improve patient outcomes significantly.
“This approval provides patients with CSCC at high risk of disease recurrence following surgery and radiation a much-needed option, as LIBTAYO is the only immunotherapy to demonstrate efficacy in this setting,” said Dr. George D. Yancopoulos, Board co-Chair, President and Chief Scientific Officer at Regeneron. “Now with five FDA-approved indications, LIBTAYO is firmly established as a strong and versatile PD-1 inhibitor for a range of cancers.”
The approval is based on results from the pivotal phase III C-POST trial, which demonstrated a 68% reduction in the risk of disease recurrence or death with LIBTAYO compared to placebo (HR: 0.32; 95% CI: 0.20–0.51; p<0.0001). The safety profile was consistent with known effects of LIBTAYO, with the most common adverse events including rash, pruritus, and hypothyroidism.
“Many patients who undergo surgical resection of their CSCC are later found to be at high risk of recurrence,” noted Dr. Vishal Patel of George Washington University. “As the first and only approved adjuvant immunotherapy for these patients, LIBTAYO represents a practice-changing opportunity backed by compelling data.”
An additional regulatory decision for LIBTAYO in the European Union is expected in the first half of 2026. Regeneron also announced the launch of LIBTAYO Surround, a support program offering financial and educational resources to assist patients throughout their treatment.
