FDA Approves Eisai & Biogen’s LEQEMBI IQLIK for Maintenance Treatment of Early Alzheimer’s

Eisai Co., Ltd. and Biogen Inc. announced that the FDA has approved the Biologics License Application (BLA) for LEQEMBI IQLIK (lecanemab-irmb), a once-weekly 360 mg subcutaneous autoinjector designed for maintenance dosing in patients with early Alzheimer’s disease. The device, which administers treatment in approximately 15 seconds, offers patients an alternative to intravenous (IV) infusions.

“We believe LEQEMBI IQLIK represents a major step forward in patient-centered Alzheimer’s care,” said Haruo Naito, CEO of Eisai. “By providing a simple at-home option, we are expanding access and improving convenience for patients and their care partners.”

The approval is based on sub-studies of the phase III Clarity AD open-label extension, which showed that weekly subcutaneous administration maintained clinical and biomarker benefits comparable to IV dosing. In more than 600 patients studied, the safety profile of the autoinjector was consistent with IV therapy, though systemic reactions were far less frequent (<1% compared with ~26% for IV). Mild local reactions, such as redness or itching at the injection site, were observed in about 11% of patients and did not interfere with continued treatment.

Christopher A. Viehbacher, CEO of Biogen, added: “The approval of this new formulation underscores our commitment to providing flexible options that can help patients maintain their independence for longer. LEQEMBI IQLIK simplifies ongoing therapy while supporting broader healthcare system efficiency.”

Alzheimer’s disease is a progressive condition driven by amyloid beta and tau pathology. LEQEMBI is the first and only approved therapy that targets both amyloid plaques and protofibrils, which are considered among the most toxic forms of amyloid beta. Maintenance treatment, administered through weekly subcutaneous injections or monthly IV infusions, ensures the continued slowing of disease progression and helps patients preserve their quality of life. Eisai and Biogen plan to launch LEQEMBI IQLIK in the U.S. on October 6, 2025.

Travere Therapeutics Gains FDA Nod for FILSPARI REMS Update in IgA Nephropathy

Travere Therapeutics, Inc. announced that the FDA has approved an update to the Risk Evaluation and Mitigation Strategy (REMS) for FILSPARI (sparsentan), the first and only Dual Endothelin Angiotensin Receptor Antagonist for IgA nephropathy (IgAN). The changes reduce liver function monitoring to every three months from treatment initiation and remove embryo-fetal toxicity monitoring from the REMS requirements.

“FILSPARI is becoming a foundational treatment for people living with IgA nephropathy, giving patients and their families hope for slowing the progression of their disease,” said Jula Inrig, M.D., chief medical officer of Travere Therapeutics. “Today’s approval of streamlined monitoring requirements reflects the strong safety profile of FILSPARI established to date across clinical and real-world use, simplifying access for patients.”

The FDA decision was supported by post-marketing surveillance data and results from the Phase III PROTECT study in IgAN, as well as findings from the Phase III DUPLEX and Phase II DUET studies. The agency also determined that embryo-fetal toxicity monitoring was no longer required, based on two decades of human pregnancy data with ERA medicines.

Looking ahead, a supplemental New Drug Application (sNDA) for FILSPARI in focal segmental glomerulosclerosis (FSGS) remains under FDA review, with a Prescription Drug User Fee Act (PDUFA) target action date of January 13, 2026. If approved, FILSPARI would become the first medicine available for FSGS, further expanding its impact across kidney disease care.

Vanda Pharmaceuticals’ VGT-1849B Granted FDA Orphan Drug Status for Polycythemia Vera

Vanda Pharmaceuticals Inc. announced that the FDA has granted Orphan Drug Designation to VGT-1849B, a selective peptide nucleic acid-based JAK2 inhibitor for the treatment of polycythemia vera, a rare chronic myeloproliferative disorder affecting an estimated 44 to 57 per 100,000 people in the U.S.

“This designation underscores the potential of VGT-1849B to address a significant unmet need in polycythemia vera by offering a highly selective approach to JAK2 inhibition,” said Mihael H. Polymeropoulos, M.D., president and CEO of Vanda Pharmaceuticals. “We believe this novel therapy could improve safety outcomes for patients who currently have limited options.”

VGT-1849B is designed using OliPass Peptide Nucleic Acid (OPNA) technology, which enhances cell permeability and RNA binding. By selectively targeting JAK2 mRNA, the therapy suppresses aberrant hematopoiesis while minimizing off-target kinase effects—a challenge seen with currently available JAK inhibitors such as Jakafi®, Inrebic®, Ojjaara®, and Vonjo®.

If approved, VGT-1849B could emerge as the first highly selective JAK2 inhibitor, aiming to reduce red blood cell overproduction, alleviate disease burden, and improve quality of life for PV patients through targeted efficacy and a favorable safety profile.

Teva Launches First FDA-Approved Generic SAXENDA for Weight Loss

Teva Pharmaceuticals, Inc., the U.S. affiliate of Teva Pharmaceutical Industries Ltd., announced that the FDA has approved and the company has launched the first generic version of SAXENDA (liraglutide injection), marking a major milestone in the weight loss treatment landscape.

“With this approval, and by launching a generic for SAXENDA, we will provide patients in the U.S. the first ever generic GLP-1 product specifically indicated for weight loss,” said Ernie Richardsen, SVP, Head of U.S. Commercial Generics at Teva. “This is the fifth first-to-market entry of a Teva generic this year and is an important addition to Teva’s diverse complex generics portfolio, demonstrating once again our proven ability to sustain a world-class Generics Powerhouse.”

Generic SAXENDA enters a fast-growing category of GLP-1 therapies, offering a more accessible option for patients seeking weight management solutions. SAXENDA recorded annual U.S. sales of $165 million as of June 2025, highlighting the strong demand for effective obesity treatments.

Liraglutide injection is indicated for adults with obesity or overweight who also have weight-related medical conditions, as well as adolescents aged 12–17 years with obesity weighing over 60 kg. The approval reinforces Teva’s Pivot to Growth Strategy and its continued commitment to expanding its complex generics portfolio.

Sanofi’s WAYRILZ Gets FDA Approval as First BTK Inhibitor for Immune Thrombocytopenia

The FDA has approved Sanofi’s WAYRILZ (rilzabrutinib) for adults with persistent or chronic immune thrombocytopenia (ITP) who have not responded adequately to a prior therapy. The approval is based on data from the pivotal LUNA III trial, which demonstrated rapid and durable platelet responses, improvements in ITP symptoms, and meaningful gains in quality of life.

“The burden of immune thrombocytopenia can be both physical and emotional with significant overlooked symptoms that can impact various aspects of daily living,” said Caroline Kruse, President and CEO of the Platelet Disorder Support Association. “We are pleased to have a new treatment option that can help ease the ongoing strain of managing the disease for patients and their families.”

WAYRILZ is the first oral, reversible Bruton’s tyrosine kinase (BTK) inhibitor approved for ITP. By acting through multi-immune modulation, it targets key pathways that drive the disease, representing a novel approach compared to existing therapies. In the LUNA III study, 23% of patients treated with WAYRILZ achieved a durable platelet response at week 25 compared to none in the placebo arm, with faster onset and longer duration of platelet response. Patients also reported notable improvements in quality-of-life measures.

“With its differentiated mechanism of action, WAYRILZ has the potential to become a treatment of choice for immune thrombocytopenia patients who have not responded to a prior therapy,” said Brian Foard, Executive Vice President, Head of Specialty Care at Sanofi. “This approval underscores Sanofi’s expertise and ambitions at the junction of rare and immunological disease.”

WAYRILZ was also recently approved in the UAE and is under regulatory review in the EU and China. It has received FDA Fast Track and Orphan Drug Designations for ITP, along with orphan designations in Japan and the EU. Beyond ITP, WAYRILZ is being investigated for other rare immune-mediated conditions, including warm autoimmune hemolytic anemia, IgG4-related disease, and sickle cell disease.