Roche’s GAZYVA/GAZYVARO Achieves Key Phase III Success in Primary Membranous Nephropathy; Novartis’ VANRAFIA Slows Kidney Function Decline in Phase III IgA Nephropathy Trial; PTC Therapeutics Shares Latest Regulatory Developments for Translarna; BridgeBio’s Oral Infigratinib Achieves Key Body Proportionality Improvements in Phase 3 Achondroplasia Trial; Upstream Bio’s Verekitug Achieves Key Endpoints in Phase 2 Trial for Severe Asthma Treatment

Roche Reports Positive Phase III Data for GAZYVA/GAZYVARO in Primary Membranous Nephropathy

Roche has reported highly encouraging results from the Phase III MAJESTY study, which evaluated the efficacy and safety of GAZYVA/GAZYVARO (obinutuzumab) in adults suffering from primary membranous nephropathy (PMN). PMN is a rare but debilitating autoimmune kidney disease that can lead to irreversible organ damage if not effectively managed. The study successfully met its primary endpoint, demonstrating that a significantly higher proportion of patients treated with GAZYVA/GAZYVARO achieved complete remission at the two-year mark (104 weeks) compared to those receiving tacrolimus, a current standard of care. 

This achievement is particularly notable as PMN currently has limited dedicated treatment options, and GAZYVA/GAZYVARO has the potential to become the first therapy specifically indicated for this condition. Beyond the primary endpoint, the trial also reached key secondary goals, including a significant increase in overall remission rates, combining both complete and partial remission, at week 104, as well as superior complete remission rates as early as week 76. These data suggest that the drug not only helps more patients reach a state of deep clinical recovery but also maintains kidney function over a longer duration, potentially delaying the need for invasive interventions like dialysis or transplant.

The safety profile observed in the MAJESTY trial was consistent with the well-established record of GAZYVA/GAZYVARO in other indications, with no new or unexpected safety signals emerging during the study. This consistency provides further confidence for its use in chronic kidney conditions. The success of this trial marks the fourth positive Phase III result for GAZYVA/GAZYVARO across a spectrum of immune-mediated kidney diseases, following similar wins in lupus nephritis and idiopathic nephrotic syndrome. By targeting and depleting the B cells that drive the underlying inflammation and glomerular damage in PMN, GAZYVA/GAZYVARO addresses the root cause of the disease. Roche’s leadership expressed optimism that these “unprecedented” results could fundamentally change the standard of care, offering hope to the thousands of patients who currently struggle with the long-term complications of this autoimmune disorder.

Novartis’ VANRAFIA Demonstrates Phase III Efficacy in Reducing Kidney Function Decline in IgA Nephropathy

Novartis recently unveiled the final results from the Phase III ALIGN study, providing critical insights into the long-term benefits of VANRAFIA (atrasentan) for adults with IgA Nephropathy (IgAN). The study focused on the drug’s ability to slow the decline of kidney function, a major concern for IgAN patients who face a high risk of progressing to end-stage renal disease. The final analysis at Week 136, which occurred four weeks after the treatment period ended, revealed a positive difference in the estimated glomerular filtration rate (eGFR) change from baseline compared to the placebo group. 

Specifically, VANRAFIA demonstrated a preservation of kidney function with a mean difference of 2.39 ml/min/1.73m² over the placebo. While the p-value at this final time point was 0.057, narrowly missing the conventional 0.05 threshold for statistical significance, the results at the end of the treatment period (Week 132) reached nominal significance with a p-value of 0.039. Novartis highlighted that these findings, spanning over two and a half years of follow-up, represent some of the most durable data in the IgAN therapeutic landscape to date, reinforcing the drug’s potential as a foundational, disease-modifying treatment.

One of the most significant aspects of the ALIGN trial was the observation of clinical benefits in a prespecified exploratory group of patients who were already receiving SGLT2 inhibitors. This suggests that VANRAFIA can be effectively integrated into modern treatment regimens that include multiple classes of medication to combat the complex drivers of IgAN. 

Unlike some other therapies in the endothelin receptor antagonist class, VANRAFIA is an ETA-selective oral drug that does not require a Risk Evaluation and Mitigation Strategy (REMS) program, making it easier for healthcare providers to prescribe and for patients to manage. Having already received accelerated approval in both the U.S. and China for the reduction of proteinuria, these new data on kidney function preservation are expected to support Novartis’ plans for a traditional regulatory submission in 2026. The company remains committed to establishing VANRAFIA as a pillar of their renal franchise, aiming to offer a treatment that targets the chronic inflammation and scarring that typically lead to kidney failure in this patient population.

PTC Therapeutics Issues Update on Translarna Regulatory Status

PTC Therapeutics has issued a significant and disappointing update regarding the regulatory status of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (DMD). The company announced its decision to withdraw the New Drug Application (NDA) resubmission in the United States following direct feedback from the Food and Drug Administration (FDA). During recent discussions, the FDA indicated that the data included in the submission were unlikely to meet the agency’s threshold for “substantial evidence of effectiveness” required for approval. 

This development marks a major setback for PTC, which has spent over two decades attempting to bring this small-molecule therapy to the U.S. market. The decision follows a long history of regulatory hurdles, including a previous rejection in 2017 and subsequent appeals. Despite the company’s efforts to provide a totality of evidence from various clinical trials and real-world data, the persistent differences in data interpretation between the manufacturer and the regulator could not be resolved, leading to the abandonment of the current filing.

The regulatory environment for Translarna has also grown increasingly difficult in international markets. In the European Union, the European Commission decided in 2024 and 2025 not to renew the drug’s conditional marketing authorization after multiple reviews of its efficacy and safety profile, which led to the expiration of its authorization in March 2025. This double blow in both the U.S. and Europe creates a period of intense uncertainty for the DMD community, particularly for the boys and young men with nonsense mutations who have relied on the drug for years. 

PTC has stated that it will evaluate the implications of this withdrawal on the continued supply of ataluren for those currently enrolled in programs or receiving the therapy. Moving forward, the company’s financial and strategic focus is expected to shift toward its other rare disease assets, such as Sephience for phenylketonuria, as the path for Translarna in Duchenne muscular dystrophy appears increasingly closed. The news has been met with deep sadness by patient advocacy groups, who have highlighted the urgent, unmet need for therapies that target the underlying genetic causes of DMD.

BridgeBio Announces Positive Phase 3 Results Showing First Statistically Significant Gains in Body Proportionality for Oral Infigratinib in Achondroplasia

BridgeBio Pharma has announced groundbreaking Phase 3 results from its PROPEL 3 trial, demonstrating that oral infigratinib provides significant benefits for children with achondroplasia. Achondroplasia is the most common form of skeletal dysplasia, often resulting in disproportionate short stature and various medical complications. The study met its primary endpoint by showing a statistically significant improvement in annualized height velocity (AHV) compared to placebo. 

Children treated with infigratinib achieved a mean growth rate of 5.96 cm/year, representing a gain of 2.10 cm/year over those in the placebo group. This growth rate is the highest reported to date in any randomized clinical trial for this condition. Furthermore, the drug showed significant improvements in height Z-scores, which measure a child’s height relative to a reference population of children with achondroplasia, suggesting a meaningful shift toward a more standard growth trajectory.

Perhaps the most unique finding of the PROPEL 3 study was the statistically significant improvement in body proportionality, a first for any therapy in a randomized achondroplasia trial. In a prespecified exploratory analysis of children younger than eight years old, infigratinib demonstrated a clear treatment difference in upper-to-lower body ratios. Proportionality is a high priority for families and patients, as it can directly impact physical function, mobility, and the ability to perform daily activities. 

By inhibiting the overactive FGFR3 signaling that causes the condition, infigratinib targets the disease at its genetic source. The drug’s oral formulation also offers a convenient alternative to existing daily injectable treatments, potentially improving long-term adherence. Given these robust data across both height and proportionality metrics, BridgeBio plans to move forward with regulatory submissions in the United States and Europe in the second half of 2026. This success positions infigratinib as a potential new standard of care that addresses both the physical growth and the functional proportions of children living with this genetic disorder.

Upstream Bio Announces Positive Phase 2 VALIANT Trial Results for Verekitug in Severe Asthma

Upstream Bio has reported positive topline results from its Phase 2 VALIANT trial, evaluating verekitug as a treatment for adults with severe asthma. Verekitug is a novel monoclonal antibody and is currently the only clinical-stage antagonist targeting the thymic stromal lymphopoietin (TSLP) receptor. The trial successfully met its primary endpoint, with verekitug demonstrating a statistically significant and clinically meaningful reduction in the annualized asthma exacerbation rate (AAER) across two different dosing schedules. 

Specifically, patients receiving 100 mg every 12 weeks saw a 56% reduction in asthma attacks, while those on a 400 mg dose every 24 weeks experienced a 39% reduction. These results are particularly impressive because they suggest that verekitug can provide robust protection against dangerous asthma flares with much less frequent dosing than many currently available biologics, which often require injections every two to four weeks.

In addition to preventing exacerbations, the VALIANT study showed that verekitug led to substantial improvements in lung function and markers of airway inflammation. Patients in the treatment arms experienced an increase in their forced expiratory volume (FEV1), a key measure of how much air a person can exhale, with gains of up to 139 mL over placebo at week 60. 

The trial also observed a significant drop in fractional exhaled nitric oxide (FeNO) levels, indicating a potent suppression of the underlying inflammation that drives severe asthma symptoms. The safety profile of the drug remained consistent with earlier, smaller studies, with no new safety concerns reported among the 478 participants. 

Based on these strong Phase 2 outcomes, Upstream Bio has announced plans to rapidly advance verekitug into Phase 3 clinical trials for both severe asthma and chronic rhinosinusitis with nasal polyps (CRSwNP). The potential for a high-efficacy treatment with a “once-every-six-months” dosing option could represent a major shift in how severe respiratory diseases are managed, offering patients greater freedom and better control over their symptoms.