- The recent approval and launch of Roche AG's longer-acting hemophilia A drug Hemlibra has brought with it predictions of disruption and sales erosion for current market leaders Shire plc, Novo Nordisk A/S and Bioverativ Inc.
- Shire CEO Flemming Ornskov, however, believes the rare disease biotech will remain a top company in the space, expressing some skepticism in an interview that the Swiss pharma's new medicine will recast the therapeutic landscape overnight.
- In particular, Ornskov drew a distinction between the market for patients who develop antibodies to current treatment — where Hemlibra is approved — and the broader population of individuals without such inhibitors, which the biotech chief says will prove tougher to break into.
Today, sales of hemophilia products make up about a quarter of Shire's global revenues, annualizing to about $3.6 billion per year. Only $800 million or so of those revenues are attributable to sales of the biotech's inhibitor therapies, such as Feiba (anti-inhibitor coagulant complex).
Shire already predicts it will lose about half, or $400 million, of those annual inhibitor sales over the next five years to new competition from drugs such as Hemlibra. Some analysts have virtually eliminated contributions from the business from their financial models, and forecast increasing pressure on Shire's more lucrative therapies for non-inhibitor patients too.
This year's annual meeting of the American Society of Hematology highlighted some of those competitive threats. Roche presented positive data from a study of Hemlibra that showed 95% of pediatric patients who received the drug experienced no bleeding events requiring treatment. Another previously announced study found Hemlibra to be similarly effective in adults and adolescents.
But an experimental gene therapy from BioMarin Pharmaceuticals Inc. stole the show in hemophilia A, cutting bleeding episodes and boosting levels of Factor VIII to normal or near-normal levels in a small group of patients. While early, the data suggests BioMarin's gene therapy could potentially control hemophilia in certain patients over years.
Progress with gene therapy could mean a one-time treatment for patients in the future. Even if that pans out, however, Shire's Ornskov thinks gene therapies and bispecific antibodies like Hemlibra will only be a part of the medicine cabinet for physicians treating hemophilia A.
"[Gene therapy] will be another option for patients in the future. But it won’t be for all," Ornskov said.
Ornskov acknowledges that hemophilia will become more competitive in the future. Yet he argues Shire's broad portfolio in the space will prove durable, particularly in the more numerous non-inhibitor patient population.
"The hurdle for a new entrant in the non-inhibitor population is higher while the existing treatment works pretty well for the patients," Ornskov explained.
Shire's non-inhibitor business could still see significant erosion, but the hit to Shire's overall business may prove to be more moderate, predicted Cowen & Co. equity analyst Steve Scala in a Nov. 17 note.
"Hemlibra clearly has a place in the hemophilia A market. However, the damage to Shire's business will likely be less than many feared," Scala wrote, citing inertia to treatment switching and some safety concerns with Roche's medicine. Although, Scala estimates that Hemlibra will hit sales of 1 billion Swiss Francs ($1.01 billion) in 2020, and 2 billion Swiss Francs ($2.02 billion) in 2024, which implies success in inhibitor patients but only modest success in non-inhibitor patients.
A recent success in a study of Hemlibra in patients without inhibitors could make Shire's job harder. And if the clinical reality of BioMarin's and others' gene therapies continue to match their promise, broader disruption may be in store.
To prepare, Ornskov says Shire has increased its R&D investment into hemophilia, noting a recent study initiation of its own gene therapy in hemophilia A and the July acquisition of bispecific technology through a deal with NovImmune.
"Shire is a leader, either top one, two or three in all its categories and I don't expect that to change," Ornskov said. "We want to defend our leadership position in hemophilia."