Page 23 - HFA Dateline 2022 Special Edition

moderate hemophilia, said pediatric and 2017 alone, 18 new products increase in half-life, compared to
hematologist Robert F. Sidonio Jr., MD, from nine different manufacturers conventional therapies, according
MSc, medical director of hemophilia, were introduced. A few examples: to research published in September
Aflac Cancer & Blood Disorders Center extended half-life clotting factor 2020 in the New England Journal of
of Children’s Healthcare of Atlanta, VIII and IX concentrates became Medicine. While BIVV001 remains
and an associate professor at Emory commercially available in 2014, an intravenous injection, the half-life
University School of Medicine. In that followed a year later by the first extension allows for once-weekly
case, they would still need to have
access to factor, especially in the event
of an injury or surgery. “Right now, the mild or moderates
will probably not be eligible for gene
Cost is also a factor that can’t be
ignored. Factor therapy costs about therapy, and the insurance companies
$300,000 a year for someone with
severe hemophilia—and the price is will likely say it’s not worth trying to
even higher for those with inhibitors.
It remains to be seen how long a treat them with gene therapy.”
gene therapy treatment will last and
whether the associated price tag will —David Clark, PhD
be higher or lower than the cost of
factor—and how much of the cost recombinant product for von factor infusions, something people
will be covered by insurance.
Willebrand disease and the first with factor VIII deficiency have yet
“Right now, the mild or moderates factor X product. to experience.
will probably not be eligible for Then came Hemlibra, or emicizumab- Furthermore, all of the currently
gene therapy, and the insurance kxwh, the first non-factor available factor products on the
companies will likely say it’s not replacement therapy. market require intravenous infusion.
worth trying to treat them with gene But researchers are working on
therapy,” Clark said. “It’s not clear In 2017, the FDA approved products that could be delivered
yet how the insurance companies are emicizumab-kxwh injections for by subcutaneous injection instead.
going to deal with that.” people with hemophilia A with One of these is Catalyst Biosciences’
factor VIII inhibitors, then in 2018 Dalcinonacog alfa (DalcA), which is in
Explosion of Products on expanded the approval for routine a Phase 2 clinical trial. The treatment
the Market prophylaxis to prevent or reduce will likely be daily, but a subcutaneous
Setting aside the promise of gene bleeding in people with hemophilia A injection is easier for many people
therapy, advances in other types of with or without factor VIII inhibitors. than accessing a vein for intravenous
products continue to occur, and People who were eligible to use treatment, Long said.
it’s important to consider their this bispecific monoclonal antibody
potential impact as a replacement no longer had to give themselves If not complete game changers,
for factor products. frequent intravenous factor infusions. these types of products could be
Instead, they were looking at time savers for many people with
The treatments for hemophilia have weekly, biweekly or even monthly hemophilia—who are accustomed
come a long way since 1965, when subcutaneous injections instead. to carving out regular times in their
Judith Graham Pool discovered day-to-day lives for prophy infusions.
cryoprecipitate, the first available Other products in development now
treatment other than whole blood. hold a lot of promise, Clark said. One But ultimately, “there’s nothing
But the number of products on example: Sanofi’s BIVV001, which on the market or in the pipeline
the market has really exploded represents a new class of factor VIII currently that would completely
in recent years, bringing more replacement therapy. Phase 1/2a obviate the need for factor
options to people with all types of studies suggest that BIVV001 could replacement in the future,” Sidonio
bleeding disorders. Between 2000 achieve a three- or even four-fold said. “That’s just the bottom line.”

Special Issue 2022 23