The FDA has granted breakthrough therapy designation following positive Phase II data released in November. Credit: grandbrothers via Shutterstock.

After a high-profile failure in an Alzheimer’s related agitation trial earlier this week, Otsuka has garnered a positive update with a breakthrough therapy designation for its rare kidney disease drug sibeprenlimab.   

The US Food and Drug Administration (FDA) designation is for sibeprenlimab’s use in immunoglobulin A nephropathy (IgAN), sometimes referred to as Berger’s disease, a disorder that occurs when antibody immunoglobin A builds up in the kidneys, leading to inflammation and damage. This results in blood and protein in urine, as well as high blood pressure and kidney failure over time. 

Go deeper with GlobalData

Data Insights

The gold standard of business intelligence.

Find out more

Related Company Profiles

View all

The FDA made the decision following positive results from the Phase II ENVISION trial (NCT04287985), announced in November 2023. The 155-patient study evaluated the dose response of different doses of sibeprenlimab by measuring proteinuria or high levels of protein in the urine.  

Data from the trial, published in The New England Journal of Medicine, showed that after 12 months, sibeprenlimab demonstrated significant reductions in 24-hour urine protein creatinine ratio (uPCR) and clinically relevant smaller changes in estimated glomerular filtration rate (eGFR), a test that measures the level of kidney function, compared to placebo. 

Sibeprenlimab is a humanised monoclonal antibody that that binds to APRIL, a tumour necrosis factor cytokine. APRIL plays a crucial role in B-cell signalling, particularly in driving IgA class switch recombination and the production and survival of IgA-secreting plasma cells. 

In 2021, Calliditas Therapeutics’ IgAN drug Tarpeyo (budesonide) became the first drug indicated to treat IgA nephropathy via the accelerated approval pathway. In December 2023, it gained full FDA approval, following a priority review four months before. According to GlobalData’s Pharma Intelligence Center, Tarpeyo is forecast to generate $485m in 2029. 

How well do you really know your competitors?

Access the most comprehensive Company Profiles on the market, powered by GlobalData. Save hours of research. Gain competitive edge.

Company Profile – free sample

Thank you!

Your download email will arrive shortly

Not ready to buy yet? Download a free sample

We are confident about the unique quality of our Company Profiles. However, we want you to make the most beneficial decision for your business, so we offer a free sample that you can download by submitting the below form

By GlobalData
Visit our Privacy Policy for more information about our services, how we may use, process and share your personal data, including information of your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

GlobalData is the parent company of Pharmaceutical Technology. 

This positive announcement regarding sibeprenlimab comes after Otsuka announced its Phase III Alzheimer’s agitation trial failed to meet its primary endpoint earlier this week. Otsuka’s stock price dropped by 10.6% following the announcement on 13 February. The company stated that it plans to conduct further tests on the data set to fully determine the drug’s potential. 

In the announcement accompanying the breakthrough designation, Otsuka chief medical officer John Kraus said: “This is an important milestone that recognises the potential value that this investigational candidate may have in the future for people living with one of the most common causes of kidney failure. Otsuka and Visterra are committed to advancing the Phase III trial for sibeprenlimab.”