1. Novartis’ Phase III gene therapy treating spinal muscular atrophy met its primary endpoint

On December 30th, 2024, Novartis announced positive results from a Phase III STEER study evaluating the efficacy and safety of an investigational product, intrathecal onasemnogene abeparvovec (OAV101 IT) treating young spinal muscular atrophy (SMA) Type 2 patients aged between 2 to 18 years old. SMA is a rare genetic neuromuscular disease caused by a lack of a functional SMN1 gene, resulting in the irreversible loss of motor neurons and affecting essential muscle functions. OAV101 is an adeno-associated virus (AAV) based gene therapy that delivers a functional gene to motor neurons. The ongoing trial targets patients who can sit but have never walked independently. The study has met its primary endpoint, showing an increase in motor ability from the baseline based on the HFMSE scores. With the positive outcomes, Novartis is willing to share the topline results with regulatory agencies.

Link: https://www.novartis.com/news/media-releases/novartis-intrathecal-onasemnogene-abeparvovec-phase-iii-study-meets-primary-endpoint-children-and-young-adults-sma

2. Approval of combination of amivantamab with Lazertinib to treat lung cancer with ex19del or 21 L858R mutation

On December 30th, 2024, Johnson & Johnson announced that the European Commission (EC) had approved an extension of indication for RYBREVANT® (amivantamab) with LAZCLUZE® (lazertinib) as the first-line treatment of adult patients with advanced non-small cell lung cancer (NSCLC) with epidermal growth factor receptor (EGFR) exon 19 deletions (ex19del) or exon 21 L858R substitution mutations. Amivantamab is a fully human antibody targeting both EGFR and MET. The approval is supported by the Phase 3 MARIPOSA results. Amivantamab plus lazertinib reduced the risk of disease progression or death by 30% compared to osimertinib monotherapy. The median duration of response (DOR) was 9 months longer for patients receiving combination therapy than those having. This approval offers new first-line treatment options for eligible patients.

Link: https://www.jnj.com/media-center/press-releases/european-commission-approves-rybrevant-amivantamab-in-combination-with-lazcluze-lazertinib-for-the-first-line-treatment-of-patients-with-egfr-mutated-advanced-non-small-cell-lung-cancer

3. Approval of Mepolizumab for patients with CRSwNP in China

On January 3rd, 2025, GSK announced that Nucala (mepolizumab) has been approved in China to treat adult patients with chronic rhinosinusitis with nasal polyps (CRSwNP) when systemic corticosteroids and/or surgery do not adequate. CRSwNP is characterized by persistent inflammation of the nasal passages and sinuses accompanied by the growth of polyps. IL-5 drives type 2 inflammation and is present at high levels in nasal polyp tissue. Nucala is a monoclonal antibody targeting IL-5. The approval is supported by the phase III MERIT trial results focusing on Japanese, Chinese and Russian patients and data from the global phase III SYNAPSE study. Previously, mepolizumab has been approved in China for severe eosinophilic asthma and eosinophilic granulomatosis with polyangiitis.

Link: https://www.gsk.com/en-gb/media/press-releases/gsk-s-nucala-mepolizumab-approved-in-china-for-treatment-of-adults-with-chronic-rhinosinusitis-with-nasal-polyps/

4. Approval of zolbetuximab with chemotherapy to treat CLDN positive gastric cancer in China

On January 6th, 2025, Astellas Pharma announced that China’s National Medical Products Administration (NMPA) had approved VYLOYTM (zolbetuximab), in combination with fluoropyrimidine- and platinum-containing chemotherapy, for the first-line treatment of patients with locally advanced unresectable or metastatic human epidermal growth factor receptor 2 (HER2)-negative gastric or gastroesophageal junction (GEJ) adenocarcinoma whose tumors are claudin (CLDN) 18.2 positive. Zolbetuximab is designed to specifically target tumor cells expressing high CLDN18.2.

Gastric cancer is the third leading cause of cancer-related mortality in China and approximately 60% of patients are diagnosed at the advanced stage. The approval is supported by data from the global Phase 3 GLOW and SPOTLIGHT clinical trials. In the GLOW trial, a median PFS of 8.21 months was achieved with zolbetuximab plus chemotherapy (capecitabine and oxaliplatin) versus 6.80 months with placebo plus CAPOX. While in the SPOTLIGHT trial, the median PFS was 10.61 months versus 8.67 months, and the median OS was 18.23 months versus 15.54 months, with zolbetuximab plus chemotherapy (oxaliplatin, leucovorin and fluorouracil), compared to the placebo arm.

Link: https://www.astellas.com/en/news/29626