1. FDA Grants Priority Review to Breyanzi for Relapsed or Refractory Marginal Zone Lymphoma
On August 04, 2025, BMS announced that the U.S. FDA has granted priority review to the supplemental biologics license application (sBLA) for Breyanzi® (lisocabtagene maraleucel; liso-cel) as a potential treatment for adult patients with relapsed or refractory marginal zone lymphoma (MZL) who have received at least two prior lines of systemic therapy. The application based on results from the primary analysis of the MZL cohort in the TRANSCEND Phase II trial. Breyanzi is a CD19-directed CAR T cell therapy with a 4-1BB costimulatory domain, which enhances the expansion and persistence of the CAR T cells.
2. Orforglipron Achieves Significant Weight Loss and Cardiometabolic Improvements in Phase III ATTAIN-1 Trial
On Aug 07, 2025, Eli Lilly and Company announced positive results from the Phase III ATTAIN-1 trial. The study investigates orforglipron, a GLP-1 receptor agonist, in 3,127 adults with obesity or overweight with a weight-related medical problem and without diabetes. All three investigated doses met the primary endpoint, and all key secondary endpoints compared to the placebo arm. For the primary endpoint, participants taking the highest dose (36 mg) of orforglipron lowered their weight by an average of 12.4% (27.3 lbs) compared to 0.9% (2.2 lbs) with the placebo. In a key secondary endpoint, 59.6% of participants taking the highest dose of orforglipron lost at least 10% of their body weight, while 39.6% lost at least 15% of their body weight. Additionally, orforglipron also lowered key cardiovascular-associated risk factors, including non-HDL cholesterol, triglycerides, and systolic blood pressure.
3. CT-155 Digital Therapeutic Meets Primary Endpoint in Phase III Schizophrenia
On Aug 07, 2025, Boehringer Ingelheim and Click Therapeutics announced that the Phase III CONVOKE study of CT-155 (BI 3972080) for negative symptoms in schizophrenia met its primary endpoint. In schizophrenia patients, 60% experience negative symptoms. CT-155 is an investigational prescription digital therapeutic that aims to provide interactive psychosocial intervention techniques as an adjunct to standard antipsychotic therapy. The primary endpoint was achieved with the significant change in experiential negative symptoms from baseline to week 16 as measured by the Clinical Assessment Interview for Negative Symptoms, Motivation and Pleasure Scale (CAINS-MAP).
4. FDA grants accelerated approval to HERNEXEOS for HER2-mutant NSCLC with high response rate
On Aug 08, 2025, Boehringer Ingelheim announced that HERNEXEOS® (zongertinib tablets) has been granted accelerated approval by the U.S. FDA to treat adult patients with unresectable or metastatic non-squamous non-small cell lung cancer (NSCLC) accompanying HER2 (ERBB2) tyrosine kinase domain-activating mutations. Zongertinib is a tyrosine kinase inhibitor (TKI) that selectively inhibits HER2 (ERBB2). The approval was based on the data from the Phase Ib Beamion-LUNG 1 trial with an objective response rate of 75% (N=71). There are 6% of patients had a complete response, and 69% of patients had a partial response. 58% of patients showed more than 6 months of response duration.
5. FDA Approves AJOVY® for Preventive Treatment of Episodic Migraine in Pediatric Patients
On Aug 06, 2025, Teva Pharmaceuticals announced that the U.S. FDA has approved the monthly single-dose injection AJOVY® (fremanezumab-vfrm) for the preventive treatment of episodic migraine in children and adolescent patients aged 6-17 years who weigh 45 kilograms (99 pounds) or more. Fremanezumab targets calcitonin gene-related peptide (CGRP), a protein involved in the sensitisation of nerve cells and transmission of pain signals related to migraine. Preventive treatment can help reduce the frequency of migraine attacks, helping children and adolescents to better manage the symptoms.
The approval was supported by the Phase III SPACE trial. In the study, fremanezumab significantly reduced monthly migraine days (–2.5 vs –1.4), monthly moderate-to-severe headache days (–2.6 vs –1.5), and monthly acute medication days (–2.1 vs –1.0) compared to the placebo group at three months. Additionally, 47.2% of patients on fremanezumab achieved at least a 50% reduction in monthly migraine days versus 27% with placebo.
6. FDA Grants Accelerated Approval to Modeyso™ for H3 K27M-Mutated Diffuse Midline Glioma
On August 06, 2025, Jazz Pharmaceuticals, a global biopharmaceutical company in the U.S., announced that the U.S. FDA had granted accelerated approval for Modeyso™ (dordaviprone) to treat adult and pediatric patients with H3 K27M mutated diffuse midline glioma. Modeyso is administered as an oral capsule once weekly. It activates an enzyme called ClpP (mitochondrial caseinolytic protease P) and inhibits the dopamine D2 receptor, which further suppresses tumor growth, induces cancer cell death, and partially reverses abnormal DNA packaging.
The approval was based on an integrated analysis of 50 patients across five open-label, non‑randomized trials. The overall response rate was 22% and the median duration of response was 10.3 months, with 73% maintaining their response for at least six months and 27% for at least 12 months.
Post comments