FDA Approves LEQEMBI IV Dosing for Early Alzheimer’s; Vanda Accepts FDA Hearing on Tradipitant for Gastroparesis; Cartesian Gains FDA Protocol Approval for Myasthenia Gravis Trial; Zai Lab Secures FDA Orphan Status for DLL3 ADC in SCLC; Dyne Receives FDA Fast Track for DYNE-101 in Myotonic Dystrophy

FDA Approves LEQEMBI® (Lecanemab-Irmb) IV Maintenance Dosing for Early Alzheimer’s Disease Treatment

Eisai Co., Ltd. and Biogen Inc. announced that the FDA has approved the Supplemental Biologics License Application (sBLA) for LEQEMBI (lecanemab-irmb), allowing intravenous (IV) maintenance dosing every four weeks for patients with early-stage Alzheimer’s disease. LEQEMBI is intended for those in the mild cognitive impairment or mild dementia stages of AD. This new dosing regimen follows an initial 18-month biweekly treatment phase and is designed to maintain the clinical and biomarker benefits observed in pivotal trials like Clarity AD and Study 201.

The decision is supported by data showing that the transition to less frequent dosing continues to clear toxic protofibrils and amyloid plaques, which are central to Alzheimer’s progression. Studies have also demonstrated that ongoing treatment helps slow cognitive decline and preserves patients’ ability to maintain daily activities for longer. The approval offers a more convenient option for patients and caregivers, making long-term treatment adherence more feasible.

LEQEMBI has received approval in multiple countries, including the U.S., Japan, South Korea, and parts of Europe, and Eisai has filed applications in 17 additional regions. The FDA has also accepted an application for a subcutaneous autoinjector version of LEQEMBI, with a decision expected by August 2025. Eisai leads the global development and regulatory efforts for LEQEMBI, with both companies co-promoting the treatment.

Vanda Pharmaceuticals Accepts FDA Hearing Opportunity for Tradipitant in Gastroparesis

Vanda Pharmaceuticals announced an update on its tradipitant development program following the FDA’s rejection of its New Drug Application for the treatment of gastroparesis. Gastroparesis, a debilitating digestive disorder causing nausea, vomiting, and abdominal pain, has seen no new FDA-approved treatments in over 40 years. Vanda, which has been studying tradipitant for nearly a decade, argued that its data demonstrated the drug’s efficacy in alleviating nausea severity in patients. However, the FDA concluded that the evidence did not meet the “substantial evidence” standard for approval.

The FDA based its rejection on mixed results from two key studies. In Study 1, tradipitant showed a statistically significant improvement in nausea severity compared to placebo (p=0.0359). However, Study 2 failed to demonstrate a significant difference between the drug and placebo, with both groups showing similar improvements. Vanda also presented supportive data from an open-label study, patients in an expanded access program, and pooled analyses, but the FDA dismissed these findings, stating that the evidence was insufficiently persuasive due to variability and the placebo effect.

Vanda has now accepted the FDA’s offer for a hearing, emphasizing the urgent unmet need for effective treatments for gastroparesis. The company maintains that the evidence provided meets the statutory standard of “substantial evidence,” which it describes as a relatively low threshold that Congress intentionally established to balance safety, efficacy, and accessibility. Vanda highlighted that tradipitant’s mechanism of action and prior studies in related conditions further support its potential as a therapeutic option for gastroparesis.

The hearing is expected to take place within 120 days, as mandated by law. Vanda remains committed to pursuing approval for tradipitant and ensuring that patients with gastroparesis have access to effective treatment options, particularly in a field where therapeutic advancements have been severely limited for decades.

Cartesian Therapeutics Secures FDA Special Protocol Assessment for Phase III AURORA Trial in Myasthenia Gravis

Cartesian Therapeutics has achieved a significant milestone with the FDA agreement on the Special Protocol Assessment (SPA) for its Phase III AURORA trial of Descartes-08 in myasthenia gravis. This trial will evaluate the efficacy of the company’s mRNA cell therapy candidate, which has shown promise in earlier studies. The SPA agreement provides regulatory clarity, indicating that the trial’s design is acceptable to support a future Biologics License Application, contingent on favorable outcomes. This represents a crucial step forward for Descartes-08, which aims to offer a convenient, outpatient-based treatment option without the need for preconditioning chemotherapy.

The randomized, double-blind, placebo-controlled trial will enroll approximately 100 participants with acetylcholine receptor autoantibody-positive (AChR Ab+) MG. Participants will be administered six weekly infusions of Descartes-08 or placebo, with the primary endpoint assessing the proportion of patients achieving an MG Activities of Daily Living (MG-ADL) score improvement of three points or more at Month 4. In Phase IIb trials, Descartes-08 demonstrated encouraging results, with participants achieving an average MG-ADL score reduction of 5.5 at Month 4. Notably, the therapy was well-tolerated, enabling outpatient administration and reinforcing its potential as a convenient alternative to existing treatments.

“This SPA agreement marks an important milestone in the development of Descartes-08 for MG, providing critical regulatory clarity and a clear path toward potential approval,” said Dr. Carsten Brunn, President and CEO of Cartesian. He further emphasized the promising data from earlier studies, stating, “Supported by compelling Phase IIb results where we observed deep and durable improvements in patients with MG, we firmly believe that Descartes-08 has the potential to serve as a meaningful new therapy.” The company is set to initiate the AURORA trial in the first half of this year, aiming to advance this novel treatment toward regulatory approval and broader patient access.

Zai Lab Receives FDA Orphan Drug Designation for ZL-1310 (DLL3 ADC) in Small Cell Lung Cancer

Zai Lab Limited announced that the FDA had granted Orphan Drug Designation to ZL-1310, a first-in-class DLL3 antibody-drug conjugate (ADC), for the treatment of small cell lung cancer (SCLC). This designation recognizes the potential of ZL-1310 to address the significant unmet needs of patients with SCLC, a disease that has limited treatment options and poor prognosis. Zai Lab is advancing the clinical development of ZL-1310, which has already shown promising objective response rates and a favorable safety profile in the ongoing Phase I trial.

“Receiving an Orphan Drug Designation for ZL-1310 recognizes its potential to treat patients with SCLC. These patients have an urgent need for innovative treatment options with improved efficacy, safety, and ready access in tertiary care and community settings,” said Rafael G. Amado, M.D., President, Head of Global Research and Development, Zai Lab. “ZL-1310 has demonstrated promising objective response rates and a favorable safety profile from the ongoing Phase I trial in patients with recurrent SCLC recently disclosed. We look forward to continuing to advance the clinical development of this promising asset across lines of therapy in SCLC and other DLL3-expressing tumors.”

With the Orphan Drug Designation, ZL-1310 will be eligible for various development incentives, including a waiver of the Prescription Drug User Fee Act (PDUFA) registration application fee, tax credits for qualifying clinical trials, and the possibility of receiving a seven-year U.S. market exclusivity period upon product approval. This regulatory achievement follows the presentation of promising data from the ongoing Phase Ia/Ib study in patients with previously treated extensive-stage SCLC (ES-SCLC), which was presented at the EORTC-NCI-AACR (ENA) Symposium 2024 in October.

Dyne Therapeutics Granted FDA Fast Track Designation for DYNE-101 to Treat Myotonic Dystrophy Type 1

Dyne Therapeutics, Inc. has announced that the FDA has granted Fast Track designation for its investigational treatment, DYNE-101, for myotonic dystrophy type 1. This designation follows encouraging clinical data from the ongoing Phase I/II ACHIEVE global clinical trial, which showed significant functional improvements across various clinical measures and a compelling impact on the key disease biomarker, splicing correction. DM1 currently lacks approved therapies, and DYNE-101 has the potential to offer transformative benefits for patients.

Doug Kerr, M.D., Ph.D., Chief Medical Officer of Dyne, remarked, “This Fast Track designation comes on the heels of robust clinical data from our ACHIEVE trial, which demonstrated substantial functional benefit for patients across a range of clinical measures and a compelling effect on the key disease biomarker of splicing correction. DM1 is a devastating disease with no approved therapies, and we are driven to deliver DYNE-101, a potentially transformative medicine, to patients as quickly as possible.”

The FDA’s Fast Track designation aims to expedite the development and review process for drugs addressing serious conditions with unmet medical needs. As a result, DYNE-101 may benefit from more frequent interactions with the FDA, rolling reviews of the Biologic License Application (BLA), and potentially earlier drug approval. Additionally, if certain criteria are met, DYNE-101 could be eligible for Accelerated Approval and Priority Review, speeding up access to this promising therapy for DM1 patients.