Roche nabs blockbuster FDA approval for Hemlibra, big label expansion for Gazyva

Roche had a big day at the FDA on Thursday, winning two critical drug approvals.

Call it a big day for Roche at the FDA, as the drug giant picked up two critical approvals on Thursday, first for hemophilia A drug Hemlibra and then with a label expansion for Gazyva. 

Hemlibra won the agency's backing to prevent or reduce bleeding in adults or children with hemophilia A who have developed antibodies called factor VIII inhibitors. Treatment will cost $482,000 for the first year and $448,000 for subsequent years, according to multiple analysts who wrote about the nod. Thursday's approval came ahead of a February 23, 2018, PDUFA date at the agency. 

Consensus analyst estimates for Hemlibra are $2 billion by 2025, but the team at Jefferies is expecting much more from the drug over time. They're projecting $5 billion in sales that year, saying the drug "appears significantly underplayed" by the consensus figure. 

"Whilst this approval is currently just in the smaller, inhibitor patient population, we expect the HAVEN 3 study in the larger noninhibitor patient population to read out by year end," they wrote in a note on Thursday. In their analysis, the team pointed to a recent statement from Roche head of medical affairs Stefan Frings that there's no reason to expect that study in the larger patient pool won't show similar efficacy. 

The drug is expected to compete with Shire's hemophilia franchise, which that company picked up in its $32 billion Baxalta buyout. Shire's Advate and Adynovate cost $394,000 and $537,000 per patient per year before discounts, respectively, at the midrange of patient weight on the label, according to Bernstein analyst Ronny Gal. 

"We note that choosing a price in line with the noninhibitor market and far below the inhibitor market, Roche … will have a sharp impact on early payer coverage (as the product is now approved for inhibitor patients only)," Gal wrote in a note on Thursday. He added that the price sends a signal that Roche expects to "effectively compete in the noninhibitor segment" as well. 

Jefferies' Jeffrey Holford wrote that the drug appears to be priced at a "significant discount of at least 50% on average versus the current cost of existing agents, which we believe on top of the superior clinical and dosing profile will aid rapid adoption and penetration of the market."

In short, the approval poses a significant risk to Shire and a growth opportunity for Switzerland's Roche, according to the analysts. The FDA approved the drug with a boxed warning for the potential of severe blood clots when used with activated prothrombin complex concentrate to treat bleeds for more than 24 hours.

As market watchers were digesting the Hemlibra approval, the FDA came through with another important decision for Gazyva. The drug won a first-line follicular lymphoma nod that Jefferies analysts expect will bring an estimated $3.5 billion at peak. Again, they acknowledged they're significantly above consensus of $1.2 billion by 2021. 

"This is an important event as it significantly expands the treatable patient population for Gazyva and will drive an inflection in its growth," Holford wrote in a separate note.

With the nods, the FDA is continuing on its speedy approval pace for the year, potentially inching toward a high-water mark in recent history. The biopharma industry is just a handful of approvals short of 2015's banner year.