Latest & Greatest
FDA Expands
Eltrombopag Indication to Even Younger
Pediatric Patients
with ITP
The U.S. Food and Drug Administration (FDA) expanded the indication
for eltrombopag for the treatment of
thrombocytopenia in pediatric patients
one year old or older with chronic
idiopathic thrombocytopenia (ITP)
who have had an insufficient response
to corticosteroids, immunoglobulins,
or splenectomy. The updated label also
includes a new oral suspension formulation designed for younger children.
Eltrombopag was already approved for
treating chronic ITP in children who are
six years or older earlier this year, based
on the results of two randomized, doubleblind, placebo-controlled trials: the phase
II PETIT (Pediatric Patients with Thrombocytopenia from ITP) and the phase
III PETIT2 trial. In each of these studies,
the most commonly reported treatmentrelated adverse events included upper respiratory tract infection, nasopharyngitis,
cough, diarrhea, pyrexia, rhinitis, abdominal pain, oropharyngeal pain, toothache,
alanine aminotransferase increased, rash,
aspartate aminotransferase increased, and
rhinorrhea.
Source: Novartis press release, August 24, 2015.
California State
Assembly Approves
Right-to-Die Bill
The End of Life Operation Act, which
seeks to allow terminally ill patients to
end their life with the assistance of a
physician, passed in California’s Senate
and will now move on to Governor Jerry
Brown for review. The bill allows patients who are given six months or less
to live by two doctors to request to seek
physician-assisted suicide, after submitting a written request and two oral
requests at least 15 days apart. Patients
must also possess the mental capacity
to make their own health-care decision
and be physically able to swallow the
lethal drug combination.
The bill faces criticism from
religious groups and disability rights
advocates who question the protection
these laws afford older adults and those
24
ASH Clinical News
with disabilities, as people who worry
that they are a financial burden on their
families could feel pressured into taking
these drugs instead of pursuing more
expensive, life-sustaining treatments,
they argue.
However, supporters note that the
bill has a number of protections in place
to prevent abuse of the law; for instance,
the bill makes it a felony for health
insurance companies to deny treatment
or coverage based on whether a patients
seeks these drugs.
Currently in the United States, four
other states have right-to-die laws, including Oregon, Vermont, Washington,
and Montana. Oregon was the first state
to take this step in 1994 with the passage of the Death with Dignity law.
Source: NPR, “California approves physician-assisted suicide; bill
heads to governor’s desk,” September 12, 2015.
House Passes Bill
Reauthorizing Stem
Cell Research
On September 8, 2015, the U.S. House
of Representatives passed “HR 2820:
Stem Cell Therapeutic and Research
Reauthorization Act of 2015,” legislation reauthorizing programs to conduct medical research and transplants
using stem cells from bone marrow and
umbilical cord blood. The program will
allocate $23 million annually for the National Cord Blood Inventory Program
and $30 million annually for the C.W.
Bill Young Cell Transportation Program
through 2020. The terms of the bill do
not reauthorize stem cell research from
destroyed human embryos, which is a
more contentious issue.
Source: The Hill. “House passes stem cell research reauthorization.”
September 8, 2015.
ACE910 Receives
Breakthrough Designation from the FDA
The U.S. FDA granted breakthrough
therapy designation to the investigational
drug ACE910 for the prophylactic treatment of hemophilia A with factor VIII
(FVIII) inhibitors in patients 12 years old
and older. The “breakthrough therapy”
designation is designed to accelerate the
development and review of medicines
that demonstrate early clinical evidence
of a substantial improvement over current
treatment options for serious diseases.
The FDA’s decision was based on the
results of a phase I trial, as well as a subsequent phase I/II extension study, in which
ACE910 was administered as a weekly
subcutaneous injection in patients with
hemophilia A with and without inhibitors
to factor VIII. In the phase I trial, which
included 18 patients, treatment with a
weekly subcutaneous injection of ACE910
led to a decrease in annualized bleeding
rate (compared with ABR 6 months prior
to study enrollment) among all patients:
from 32.5 to 1.7 in the 0.3 mg/kg cohort
(n=6); from 18.3 to 0 in the 1 mg/kg
cohort (n=6); and from 15.2 to 0 in the 3
mg/kg cohort (n=6).
In terms of safety, adverse events
were observed in 18 patients and all
were reported as mild or moderate; the
most common of these was injection site
erythema. There was also no evidence of
clinically relevant abnormalities of coagulation (determined by clinical findings
and laboratory tests) in all cohorts, and
there were no thromboembolic adverse
events observed.
ACE910 is