DUPIXENT has now been approved in multiple regions, including Japan, the UAE, and Brazil. It is expected to provide a new, effective option for over 300,000 patients in the U.S. with CSU that remains inadequately controlled on standard treatments. The clinical trials demonstrated DUPIXENT’s ability to increase the likelihood of well-controlled disease or complete response after 24 weeks of treatment.
“DUPIXENT is the first new targeted treatment for chronic spontaneous urticaria, or CSU, in over ten years, with pivotal trials demonstrating its ability to help patients significantly reduce the hallmark symptoms of intense itch and unpredictable hives associated with this disease,” said George D. Yancopoulos, M.D., Ph.D., Board co-Chair, President and Chief Scientific Officer at Regeneron.
The approval provides a much-needed option for patients who struggle with the chronic nature of CSU and often experience debilitating symptoms. Alyssa Johnsen, M.D., Ph.D., Global Therapeutic Area Head, Immunology and Oncology Development at Sanofi, emphasized, “CSU patients with uncontrolled disease experience highly burdensome itch and hives that can significantly disrupt daily living. This FDA approval provides a new treatment option to help address the underlying drivers of these severe and recurring signs and symptoms.”
Gilead’s TRODELVY Plus KEYTRUDA Shows Significant Benefit in PD-L1+ Metastatic TNBC
Gilead Sciences has announced positive results from the Phase III ASCENT-04/KEYNOTE-D19 study, showing that the combination of TRODELVY (sacituzumab govitecan-hziy) and KEYTRUDA (pembrolizumab) significantly improves progression-free survival (PFS) in patients with inoperable locally advanced or metastatic triple-negative breast cancer (mTNBC) whose tumors express PD-L1 (CPS ≥ 10). The study met its primary endpoint, demonstrating a statistically significant and clinically meaningful improvement in PFS compared to KEYTRUDA and chemotherapy.
“These findings are the first to show the transformative potential of an antibody-drug conjugate combined with an immuno-oncology agent in early treatment lines of metastatic breast cancer,” said Dietmar Berger, MD, PhD, Chief Medical Officer at Gilead Sciences. “For patients with this difficult-to-treat type of breast cancer, these results potentially offer a new pathway that may redefine their treatment options.”
The safety profile of TRODELVY plus KEYTRUDA was consistent with the known safety profiles of each drug, and no new safety signals were identified. An early trend toward improved overall survival (OS) was observed, though OS data was not mature at the time of the PFS analysis. Gilead plans to continue monitoring OS outcomes and conduct further analyses.
“For patients with metastatic triple-negative breast cancer, there is a critical need for more effective treatment options,” said Dr. Sara Tolaney, MD, MPH, Dana-Farber Cancer Institute and primary investigator of the study. “These data suggest that the combination of sacituzumab govitecan-hziy and pembrolizumab may offer a new treatment approach—bringing together a potent antibody drug conjugate with immunotherapy to improve outcomes for patients.”
TRODELVY has already demonstrated survival advantages in two types of metastatic breast cancer, and it continues to show consistent results across multiple studies. Gilead is conducting further research into TRODELVY’s efficacy in various tumor types and disease stages, including ongoing Phase III trials for other breast cancer subtypes and additional cancers.
Tempest Secures FDA Orphan Drug Designation for TPST-1495 in FAP
Tempest Therapeutics has announced that the FDA has granted Orphan Drug Designation (ODD) to TPST-1495, a novel dual receptor inhibitor targeting prostaglandin (PGE2) signaling, for the treatment of Familial Adenomatous Polyposis (FAP). This milestone marks a significant advancement in Tempest’s mission to develop innovative therapies for cancers with unmet medical needs.
“Receiving orphan drug designation for TPST-1495, our second clinical program, is a significant milestone in our mission to bring innovative therapies to patients with unmet medical need,” said Stephen Brady, President and CEO of Tempest. “This designation for the treatment of FAP underscores Tempest’s mission to make a meaningful difference in the lives of patients and builds on the momentum from prior designations received for amezalpat in hepatocellular carcinoma.”
Tempest is set to begin a Phase II study evaluating TPST-1495 in FAP patients later this year, funded by the National Cancer Institute (NCI) Division of Cancer Prevention and conducted by the Cancer Prevention Clinical Trials Network. Data from the study are expected in 2026.
uniQure’s AMT-130 Receives FDA Breakthrough Therapy Designation for Huntington’s Disease
uniQure has announced that the FDA has granted Breakthrough Therapy designation to AMT-130 for the treatment of Huntington’s disease, a rare and inherited neurodegenerative disorder with no current disease-modifying therapies. This designation complements the previously granted Regenerative Medicine Advanced Therapy (RMAT), Orphan Drug, and Fast Track designations for AMT-130.
“Receiving Breakthrough Therapy designation underscores both the urgent need for effective treatments for Huntington’s disease and the encouraging interim data demonstrating that AMT-130 has the potential to slow disease progression,” said Walid Abi-Saab, M.D., Chief Medical Officer of uniQure. “It’s a powerful recognition of the promise of AMT-130 and the important progress we’ve made. We deeply value the FDA’s continued commitment to advancing innovative gene therapies for patients with critical unmet needs, and we look forward to working closely with the agency to bring AMT-130 to the Huntington’s disease patient community as quickly as possible.”
The Breakthrough Therapy designation is based on clinical data from the ongoing Phase I/II trials of AMT-130. In July 2024, interim data revealed a dose-dependent slowing of disease progression in treated patients compared to natural history controls. To date, 45 patients have received AMT-130. This designation allows for expedited development and review, providing uniQure with increased support and guidance from the FDA.
NeuroNOS’ BA-102 Granted FDA Orphan Drug Designation for Phelan-McDermid Syndrome
NeuroNOS, a biopharmaceutical company focused on treatments for autism and Alzheimer’s disorders, has announced that the FDA has granted Orphan Drug Designation (ODD) to its lead investigational therapy, BA-102, for Phelan-McDermid Syndrome (PMS), a genetic disorder associated with Autism Spectrum Disorder (ASD). The company plans to begin first-in-human clinical trials for ASD in the United States in 2026.
“Receiving orphan drug designation from the FDA is a significant step forward for this autism program, as well as our broader mission to bring targeted therapies to individuals and families affected by rare neurodevelopmental conditions,” said Amir Avniel, CEO of NeuroNOS. “By focusing on the genetic underpinnings of Phelan-McDermid Syndrome—a syndromic form of autism—we aim to address the root cause of symptoms and offer new hope where few options currently exist.”
PMS is caused by deletions or mutations of the SHANK3 gene, leading to developmental delays, intellectual disability, severe speech impairments, and often features of ASD. Currently, no FDA-approved treatments are specifically indicated for PMS. The Orphan Drug Designation offers key development incentives, including market exclusivity upon approval and access to FDA support.
“Phelan-McDermid Syndrome represents a critical unmet medical need,” said Prof. Haitham Amal, CSO of NeuroNOS. “We are committed to working closely with the FDA, patient advocacy groups, scientific foundations, and clinical investigators to accelerate development of a therapy that could meaningfully improve quality of life for those living with this challenging genetic condition.”
