CLINICAL NEWS
New Treatment
Approved for
Hemophilia A
The FDA approved turoctocog alfa pegol,
an extended half-life factor VIII (FVIII)
molecule formerly known as N8-GP, for
the treatment of children and adults with
hemophilia A.
Turoctocog alfa pegol was approved
for the following indications: routine
prophylaxis to reduce the frequency of
bleeding episodes, on-demand treatment
and control of bleeding episodes, and
perioperative management of bleeding.
The agency’s decision to approve the
agent was based on the results from five
prospective, non–placebo-controlled,
multicenter clinical trials that enrolled
a total of 270 patients with previously
treated, severe hemophilia A and no his-
tory of inhibitors. Turoctocog alfa pegol
was administered through a fixed-dosing
regimen consisting of one injection every
four days in adults and adolescents, or
twice-weekly injections in children.
Treatment with turoctocog alfa pegol
resulted in an annualized bleeding rate of 1.18
in adults and adolescents. The overall safety
profile of turoctocog alfa pegol was similar to
what has been reported for other long-acting
FVIII products, with no safety concerns iden-
tified after more than five years of treatment
exposure, the researchers reported.
Novo Nordisk, the biologic’s manu-
facturer, said the treatment will not be
available in the U.S. until 2020 due to third-
party intellectual property agreements.”
Source: Novo Nordisk press release, February 19, 2019.
FDA Warns Consumers
About “Young Blood”
Plasma Infusions
Responding to the rising popularity of
“anti-aging” plasma infusions from young
adults, the FDA has warned consumers
that the practice has not been approved by
the regulatory agency and is potentially
unsafe, given the residual risks of infec-
tion and other complications.
Many establishments located in several
states have started offering infusions of plas-
ma obtained from young donors – charg-
ing up to thousands of dollars per infusion
– for a variety of conditions. However, as
the agency wrote in a Biologics Safety and
Availability Communication, “there is no
proven clinical benefit of the infusion of
plasma from young donors in the preven-
tion of conditions such as aging or memory
loss or for the treatment of such conditions
as dementia, Parkinson disease, multiple
sclerosis, Alzheimer disease, heart disease,
or post-traumatic stress disorder.”
ASHClinicalNews.org
While there are risks associated with
the use of any plasma product, the FDA
noted that the services advertised by
centers offering “young blood” infusions
are not guided by evidence from adequate
and well-controlled trials, likely increasing
the risks of allergic reactions, transfusion-
associated circulatory overload, and infec-
tious disease transmission.
“The administration of plasma for
indications other than those recognized
or approved by the FDA should be
performed by a qualified investigator or
sponsor who has an active Investigational
New Drug application with the FDA,”
FDA Commissioner Scott Gottlieb, MD
(who will step down from his role in
April) and Center for Biologics Evaluation
and Research Director Peter Marks, MD,
PhD, said in a statement. “Simply put,
we’re concerned that some patients are be-
ing preyed upon by unscrupulous actors
touting treatments of plasma from young
donors as cures and remedies.”
The FDA did not announce any regula-
tory or enforcement actions against compa-
nies promoting these treatments but noted
that it will “continue to closely monitor this
issue and take additional steps, as appropri-
ate, along with state and local health depart-
ments and blood establishments.” ●
Sources: FDA press announcement, February 19, 2019; FDA Biologics
Safety and Availability Communication, February 19, 2019.
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