In a phase III trial, the investigational drug,
emicizumab proved to be extremely useful via subcutaneous infusion in children
who had developed resistance to the traditional intravenous mode.
drug emicizumab via subcutaneous infusion brings relief to those resistant
to intravenous drug
- Ninety five
percent of children undergoing treatment did not experience any bleeding
- The drug also revealed a strong safety profile,
with no serious adverse events.
children usually develop antibodies or inhibitors which bind to the factor
molecules and render it ineffective. Emicizumab has been developed to target
Emicizumab is a bispecific humanized monoclonal
antibody developed to prevent bleeds in patients with hemophilia A with and
It is co-developed by Roche and Genentech. The
drug is almost similar to Factor VIII but the structure makes it unrecognizable
to Factor VIII antibodies and inhibitors. In earlier trials involving adults and adolescents, emicizumab
caused increased bleeding which Dr.
Young associates to a bypassing agent (BPA) that they consumed simultaneously.
‘A Phase III trial of a hemophilia A drug emicizumab in children was highly successful in causing no bleeding events in 95 percent of the children enrolled, suggesting the drug is highly effective at preventing the bleeding associated with hemophilia A.’
The present study enrolled 60 children from ages 1-12 years
of age who had developed antibodies which prevented effective treatment with
Factor VIII. 95% of the children did not experience bleeding after treatment
with subcutaneous, once-weekly emicizumab indicating that the drug has been
able to prevent bleeding episodes in hemophilia A
The drug has also passed the test for safety concerns and adverse events.
In the same field, an earlier study had showed that a
single infusion of an investigational gene therapy! enabled production
of normal or near-normal levels of Factor VIII
. This was the first
successful gene therapy indicating such high levels of improvement of FVIII.
This normal to near-normal production of FVIII could completely eliminate the
need for prophylactic or interventional intravenous FVIII infusions in people
Weekly subcutaneous emicizumab has the potential to reduce
overall treatment and disease burden and may provide a new standard of care for
hemophilia management by providing an effective, safe and convenient option for
pediatric hemophilia A with inhibitors.
"Before this drug, we didn't have very effective ways to
prevent joint bleeding in these patients," said lead study author Guy Young,
MD, director of the hemostasis and thrombosis program at Children's Hospital
Los Angeles and University of Southern California. "This drug has demonstrated
a very high level of efficacy at preventing those bleeding events. It's been
life-changing for the children I've treated."
A future trial will determine the safety and efficacy of
emicizumab in patients who do not have the inhibitors.With the
novel drug, the outlook for hemophilia A is extremely positive and is a huge
step in improving the quality of lives of patients globally.
Hemophilia A or Factor VIII deficiency is a classic form of
hemophilia. It is an X-linked genetic disorder characterized by missing or
mutated Factor VIII protein required for clotting. In some cases, spontaneous
mutations in the gene cause the disorder. People with hemophilia A are at risk
of prolonged bleeding from simple injuries and falls. Internal bleeding also
occurs in joints and muscles, stomach and in some cases brain bleeds. Bleeding
can be stemmed with immediate infusion of Factor VIII. Currently, the only
available treatment for hemophilia A is infusion of external Factor VIII either
as a prophylactic or response to bleeding episodes.
- New Therapies Improve Outlook for Bleeding and Clotting Disorders - (http://www.hematology.org/Newsroom/Press-Releases/2017/8065.aspx)
- Bleeding Disorder Future Therapies - (https://www.hemophilia.org/Bleeding-Disorders/Future-Therapies)