The Food and Drug Administration on Tuesday granted accelerated approval to Otsuka Pharmaceutical's injectable medicine, the first in a class of medicines for a rare, potentially life-threatening kidney disease.
The new medicine, known as Voyxact, is now approved for its ability to reduce protein in the urine of people with IgA nephropathy, or IgAN, an autoimmune disease that causes protein to build up in the kidneys and that can lead to failure of the organ. Voyxact comes as a self-administered, subcutaneous injection given every four weeks. Otsuka did not disclose pricing.
“The availability of Voyxact represents a novel targeted approach to help manage this complex disease for patients living with IgAN,” said John Kraus, executive vice president and chief medical officer of Otsuka, in a statement.
Several therapies exist to treat IgAN. However, because the disease still leads to kidney function decline, demand remains for new treatments. Some estimates hold that up to 130,000 people in the U.S. have the disease, presenting a market opportunity in the range of $6 billion to $10 billion, according to Jefferies analyst Farzin Haque.
Such an opportunity has attracted interest from large drugmakers like Vertex Pharmaceuticals, Biogen and Novartis.
Otsuka acquired Voyxact, scientifically known as sibeprenlimab, through a 2018 buyout of Visterra. The drug works by blocking a protein called A-proliferation-inducing-ligand, or APRIL, which plays a key role in the development and progression of IgAN.
Supporting the approval are interim results from a late-stage study, in which Voyxact reduced proteinuria levels by 51% after nine months compared to a 2% increase in a placebo group. The most common side effects observed were infections and injection site reactions.
The study is still ongoing to evaluate a change in kidney function measured by “estimated glomerular filtration rate,” or eGFR, over 24 months. Data is expected in 2026.
Other drugs with the same mechanism may arrive on market in the coming years. Most notably, Voyxact could compete with atacicept, a drug from Vera Therapeutics that targets APRIL as well as a protein called B cell activating factor, or BAFF.
In a note to clients, Leerink’s Haque argued that Voyxact’s labeling bodes well for Vera, as it omits a urine protein/creatinine cutoff, “breaking precedent set by four prior accelerated approvals,” which would have restricted use to a smaller patient population.
Vera submitted the drug to the FDA earlier this month. If approved, it could join enter the market as soon as next year.
