1. Gene therapy OAV101IT improved motor scores in SMA patients
On Mar 19, 2025, Novartis announced positive safety and efficacy results from the Phase III program for investigational intrathecal onasemnogene abeparvovec (OAV101IT) in patients aged 2-18 years old with spinal muscular atrophy (SMA). In the Phase III STEER study, OAV101IT treatment significantly improved Hammersmith Functional Motor Scale Expanded (HFMSE) by 2.39 points, where there were only 0.51 points of improvement in the placebo arm. OAV101IT is an investigational gene replacement therapy that replaces the non-functional SMN1 gene.
2. U.S. FDA approved oral Fabhalta® in treating C3 glomerulopathy
On March 20, 2025, Novartis announced that oral Fabhalta® (iptacopan) has been approved by the U.S. FDA approval to treat adults with C3 glomerulopathy (C3G) reducing proteinuria as the third approved indication. Iptacopan inhibits the alternative complement pathway by selectively targeting factor B. The excessive activation of this pathway would lead to C3 deposition and renal inflammation in the kidney. By blocking factor B, iptacopan effectively prevents the C3 convertase enzyme complex formation. The drug has been previously approved for paroxysmal nocturnal hemoglobinuria and primary immunoglobulin A nephropathy.
3. U.S. FDA granted Fast Track designation to nipocalimab for moderate-to-severe Sjögren’s disease
On March 18, 2025, Johnson & Johnson announced that the U.S. FDA had granted investigational nipocalimab Fast Track designation (FTD) for the treatment of adult patients with moderate-to-severe Sjögren’s disease. Currently, there is no FDA-approved treatment that could directly address the underlying causes. Nipocalimab is an investigational monoclonal antibody blocking FcRn with high affinity and reducing levels of circulating immunoglobulin G (IgG) antibodies. The Phase 2 DAHLIAS study achieved the primary endpoint in the 15 mg/kg biweekly nipocalimab group, showing a greater than 70% relative average improvement in systemic disease activity at Week 24 compared to placebo and IgG reductions of more than 77%.
4. U.S. FDA approved TREMFYA® to treat Crohn’s adult patients with both subcutaneous and intravenous induction options
On March 20, 2025, Johnson & Johnson announced that the U.S. Food and Drug Administration (FDA) has approved TREMFYA® (guselkumab) for the treatment of adults with moderately to severely active Crohn’s disease with both subcutaneous (SC) and intravenous (IV) induction options. TREMFYA® blocks IL-23 and CD64, which are key drivers in multiple immune-mediated diseases. The approval was supported by multiple Phase 3 trial results. Both GRAVITI (SC induction vs. placebo) and GALAXI (IV induction vs. placebo) trials showed improved clinical remission and endoscopic responses. This is the fourth indication to be approved following moderate-to-severe plaque psoriasis, active psoriatic arthritis and moderately to severely active ulcerative colitis.
Week 12 Results | GRAVITI | GALAXI 2 | GALAXI 3 |
TREMFYA® 400 mg SC induction at Weeks 0, 4 and 8 vs. placebo | TREMFYA® 200 mg IV induction at Weeks 0, 4 and 8 vs. placebo | TREMFYA® 200 mg IV induction at Weeks 0, 4 and 8 vs. placebo | |
Clinical remission | 56% vs. 22% (p<0.001) | 47% vs. 20% (p<0.001) | 47% vs. 15% (p<0.001) |
Endoscopic response | 34% vs. 15% (p<0.001) | 36% vs. 9% (p<0.001) | 34% vs. 13% (p<0.001) |
5. EU approved Imfinzi for adults with limited-stage small cell lung cancer
On 17 March 2025, Astra Zeneca announced that Imfinzi (durvalumab) has been approved by EU as monotherapy for the treatment of adults with limited-stage small cell lung cancer (LS-SCLC) whose disease has not progressed following platinum-based chemoradiation therapy (CRT). The approval was supported by the ADRIATIC Phase III trial. In the trial, results showed Imfinzi reduced the risk of death by 27% vs. the placebo group. The estimated median overall survival was 55.9 months for Imfinzi versus 33.4 months for placebo. An estimated 57% of patients treated with Imfinzi were alive at three years compared to 48% for placebo. The treatment also reduced the risk of disease progression or death by 24% compared to the placebo group. Median progression-free survival was 16.6 months for Imfinzi versus 9.2 months for placebo. An estimated 46% of patients treated with Imfinzi had not experienced disease progression at two years compared to 34% for placebo.
6. Eneboparatide (AZP-3601) met its primary endpoint in the Phase III CALYPSO trial
On 17 March 2025, Astra Zeneca announced eneboparatide (AZP-3601) met its primary endpoint with statistical significance improvement in adults with chronic hypoparathyroidism (HypoPT) at 24 weeks in Phase III CALYPSO trial. Eneboparatide is an investigational parathyroid hormone (PTH) receptor 1 agonist. HypoPT is a rare endocrine disease with a deficiency of PTH and impaired regulation of calcium and phosphate levels in the blood. The primary endpoint consists of the normalisation of albumin-adjusted serum calcium levels and independence from active vitamin D and oral calcium therapy. The trial will continue to be monitored until Week 52.
7. U.S. FDA accepted the sNDA and granted Priority Review for finerenone in heart failure patients with LVEF ≥40%
On March 17, 2025, Bayer announced that the U.S. FDA had accepted the supplemental New Drug Application (sNDA) and granted Priority Review designation for finerenone adult patients with heart failure with a left ventricular ejection fraction (LVEF) of ≥40%. Finerenone is a non-steroidal, selective mineralocorticoid receptor antagonist (nsMRA) targeting the mineralocorticoid receptor (MR) pathway with cardiovascular benefits. The acceptance was supported by positive data from the FINEARTS-HF and will be submitted for further regulatory applications.
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