The Food and Drug Administration on Thursday approved a new hemophilia therapy that has the potential to be a more convenient option for some patients and, according to analysts, a lucrative product for its developers.
The therapy, named Altuviiio, is approved to both prevent and treat bleeding episodes in adults and children with hemophilia A, the more common form of the rare blood disorder. In hemophilia A, genetic mutations impair the production of a crucial blood-clotting protein called Factor VIII.
Many people with this form of the disease are treated with laboratory-engineered Factor VIII protein that’s administered through intravenous infusions two to three times a week. Altuviiio, though, was built to last longer, allowing for once-a-week dosing.
“When you think about classic Factor VIII molecules or preparations, the big limitation is the half life,” said Dietmar Berger, chief medical officer at Sanofi, which co-developed Altuviiio with another European drugmaker, Sobi.
“These are young adolescents and adults that have to have an IV every two days,” Berger added. “Now you go to an IV once a week, and the benefit is just tremendous.”
In designing Altuviiio, the drugmakers incorporated key elements from another important protein for blood clotting. This protein, known as von Willebrand factor, binds to Factor VIII and helps not only to transport it, but also protect it from being broken down by the body.
Scientists also equipped Altuviiio with two so-called “XTEN” polypeptides to further slow the molecule’s degradation.
Altogether, these characteristics allow the drug to keep levels of clotting protein higher for longer periods of time. In the clinical trial that led to Altuviiio’s approval, average Factor VIII levels were above 40% for most of the week between doses. Notably, the trial only enrolled people with severe hemophilia A, meaning they normally have less than 1% the amount of Factor VIII seen in people without the disease.
Sanofi executives have argued these effects make Altuviiio particularly attractive for hemophilia patients who want to be more active in their daily life. Berger noted how some of the children who received the drug are now riding bikes and playing sports without fear that they’ll experience bleeds.
“They literally forget that they have hemophilia,” he said. “With the other drugs, you still have to be careful, because your levels dip before you get your next infusion.”
Levels dip in Altuviiio-treated patients, too, with clinical tests showing that they hover around 10% a week after infusion. Still, the disease can be managed at those levels for a short while, Berger said.
Though the market for hemophilia drugs has grown crowded, Sanofi and Sobi see a place for Altuviiio and expect it to take share from some of the other currently available products. That includes another hemophilia A drug the companies share rights to, Eloctate, which was approved in 2014 and is usually administered two to three times a week.
For 2022, Sanofi recorded 580 million euros worth of sales from Eloctate — a decrease of 6% compared to the prior year, when accounting for currency exchange rates.
The drugmaker said it priced Altuviiio “at parity” to the annual cost of prophylaxis treatment with Eloctate.
Given its dosing schedule and the levels of blood-clotting protein seen in testing, Altuviiio “may return patients who experienced bleeds or [are] at high risk for bleeds to an almost near-normal state, enabling new daily lifestyle possibilities,” wrote Jefferies analyst Eun Yang, who covers Sobi, in a recent note to clients.
Cindy Leissinger, director of the Louisiana Center for Bleeding and Clotting Disorders at Tulane University, described Altuviiio as an important addition to hemophilia treatment. “In a crowded field of transformative therapies for hemophilia, [Altuviiio] stands out as a winner,” she wrote in an editorial published alongside the drug’s trial data this January.
Yet, Altuviiio may soon face added competition. BioMarin Pharmaceutical, for example, is attempting to secure approval for a hemophilia A gene therapy that, in some patients, may serve as a one-time fix for the disease. That therapy has hit multiple regulatory setbacks as the FDA has sought more information about its durability.
“The adoption of gene therapy will take some time, and especially for the first-generation gene therapies because of these questions about durability, about side effects, about it being really a genetic modification,” Berger said.
Altuviiio will also have to contend with Roche’s Hemlibra, a long-acting hemophilia A drug that first received FDA approval in 2017. The Switzerland-based pharmaceutical giant recorded a 27% increase in Hemlibra sales last year, to 3.8 billion Swiss francs. The drug is now used in more than 19,000 patients and holds 36% of the hemophilia A drug market across the U.S. and a handful of European countries, according to Roche.
For Sanofi, Thursday’s approval could help to further validate its $11 billion acquisition of the blood disease drugmaker Bioverativ. That deal, which happened in early 2018, came about a year after Bioverativ spun out of Biogen, the neuroscience-focused biotechnology company.
In buying Bioverativ, Sanofi got two marketed products in Eloctate and Alprolix — a hemophilia B drug — along with a slate of experimental medicines. The first of those medicines to reach market, Enjaymo, was granted FDA approval early last year as a treatment for a rare form of anemia.
Editor’s note: This story has been updated to include Sanofi comments on Altuviiio’s price.