1. Elevidys significantly improved the motor ability of patients with Duchenne muscular dystrophy
On January 27, 2025, Roche announced positive results from a global, randomised, double-blind phase III trial. The study evaluates Elevidys™ (delandistrogene moxeparvovec) as the first approved gene therapy to treat Duchenne muscular dystrophy patients. Duchenne is a genetic disorder characterised by progressive muscle degeneration and weakness in early childhood due to the alterations of dystrophin which keeps muscle cells intact. After two years of treatment, three key motor function measures of North Star Ambulatory Assessment (NSAA), Time to Rise (TTR) and 10-meter walk/run (10MWR) have been significantly improved compared to the external control group. Moreover, the muscle biopsies from a subset of patients taken 64 weeks after dosing have also shown consistent and sustained micro-dystrophin expression.
Link: https://www.roche.com/media/releases/med-cor-2025-01-27
2. Positive OS results favoured Itovebi-based regimen for a specific indication of breast cancer
On January 28, 2025, Roche announced positive overall survival (OS) analysis results evaluating the phase III INAVO120 study which investigated ItovebiTM (inavolisib) in combination with palbociclib (Ibrance®) and fulvestrant for people with PIK3CA-mutated, HR+, HER2-, endocrine-resistant, locally advanced or metastatic breast cancer. The study met its key secondary endpoint of improved OS benefit with the addition of ItovebiTM compared to palbociclib (Ibrance®) and fulvestrant alone. Itovebi-based regimen reduced the risk of disease worsening or death by 57% compared with palbociclib and fulvestrant alone. The U.S. FDA approved this Itovebi-based regimen in October 2024 for the indicated indication as detected by an FDA-approved test.
Link: https://www.roche.com/media/releases/med-cor-2025-01-28
3. Approval of Susvimo for diabetic patients with macular edema
On February 4, 2025, Roche announced that the U.S. FDA has approved Susvimo® (ranibizumab injection) 100 mg/mL to treat diabetic macular edema (DME). DME causes vision loss in adult patients with diabetes. Ranibizumab is a VEGF inhibitor which prevents endothelial cell proliferation, vascular leakage, and new blood vessel formation. Susvimo is the first and only FDA-approved treatment that could maintain vision in people with DME with fewer treatments than standard-of-care eye injections. The approval was based on the phase III Pagoda study where people with DME who received Susvimo refilled every six months achieved non-inferior improvements in vision compared with those receiving monthly 0.5 mg ranibizumab intravitreal injections.
Link:https://www.roche.com/media/releases/med-cor-2025-02-04
4. Approval of Enhertu for HR+ and HER2 low or ultralow breast cancer
On January 27, 2025, AstraZeneca and Daiichi Sankyo’s Enhertu (trastuzumab deruxtecan) has been approved in the US for the treatment of adult patients with unresectable or metastatic HR+, HER2-low (IHC 1+ or IHC 2+/ISH-) or HER2-ultralow (IHC 0 with membrane staining) breast cancer as detected by the FDA approved test. The approval also addressed the importance of standard IHC testing on tumour tissue to identify those who may be eligible for treatment with Enhertu following endocrine therapy. FDA decision was supported by the DESTINY-Breast06 Phase III trial, where Enhertu showed a 36% reduction in the risk of disease progression or death versus chemotherapy. A median progression-free survival (PFS) of 13.2 months was obtained in patients randomised to Enhertu compared to 8.1 months in those randomised to chemotherapy. The confirmed objective response rate (ORR) in the overall trial population was 62.6% for Enhertu versus 34.4% for chemotherapy.
5. Amycretin showed significant weight reduction effects across multiple dosings
On Janurary 24, 2025, Novo nordisk announced results from a phase 1b/2a clinical trial investigating amycretin, unimolecular GLP-1 and amylin receptor agonist in people with overweight or obesity. In participants who adhered to the treatment, those treated with amycretin achieved an estimated body weight loss of 9.7% on 1.25mg (20 weeks), 16.2% on 5mg (28 weeks) and 22.0% on 20mg (36 weeks), in contrast, those on placebo experienced an estimated 1.9%, 2.3% and 2.0% body weight gain, respectively. Meanwhile, the safety profile was consistent with incretin-based therapies and the most common adverse events were gastrointestinal with mild to moderate intensity.
6. Drugs received positive recommendations from EU CHMP in Jan 2025:
Company | Drug | Target | Type | Indication |
Merck | CAPVAXIVE™ (Pneumococcal 21-valent Conjugate Vaccine) | / | Vaccine | Active immunization for the prevention of invasive disease and pneumonia caused by Streptococcus pneumoniae in individuals 18 years of age and older |
J&J | Subcutaneous (SC) formulation of RYBREVANT®(amivantamab), with/without LAZCLUZE® (lazertinib) | EGFR-MET | Monotherapy/Combination | Monotherapy - adult patients with advanced NSCLC with activating EGFR exon 20 insertion mutations after failure of platinum-based therapy Combination - adult patients with advanced non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions or exon 21 L858R substitution mutations |
AstraZeneca | Imfinzi (durvalumab) | PD-L1 | Monotherapy | Adult patients with limited-stage small cell lung cancer (LS-SCLC) whose disease has not progressed following platinum-based chemoradiation therapy (CRT). |
AstraZeneca and Daiichi Sankyo | Datopotamab deruxtecan (Dato-DXd) | TROP2 | ADC | Adult patients with unresectable or metastatic hormone receptor (HR) positive, HER2 negative (IHC 0, IHC 1+ or IHC 2+/ISH-) breast cancer who have received endocrine therapy and at least one line of chemotherapy in the advanced setting |
BMS | Breyanzi® (lisocabtagene maraleucel; liso-cel), a CD19-directed chimeric antigen receptor (CAR) T cell therapy | CD19 | CAR-T | Adult patients with relapsed or refractory follicular lymphoma (FL) who have received two or more prior lines of systemic therapy |
BMS | Opdivo® (nivolumab) plus Yervoy® (ipilimumab) | PD-1 and CTLA-4 | Combination | Adult patients with unresectable or advanced hepatocellular carcinoma (HCC) |
Link:
https://www.daiichisankyo.com/files/news/pressrelease/pdf/202501/20250131_E.pdf
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