FDA panel recommends Chelsea Therapeutics drug
* FDA panel recommends Northera for FDA approval
* Vote follows FDA staff position opposing approval
* Final FDA decision due by March 28
* Shares up 66 percent as trading resumes
(Updates throughout with details, quotes and background)
By David Morgan
WASHINGTON, Feb 23 (Reuters) - Chelsea Therapeutics
International Ltd's <CHTP.O> hypotension drug Northera should be
approved for use in the United States, a committee of
independent experts recommended on Thursday, sending the
company's shares sharply higher in afterhours trade.
The panel assembled by the Food and Drug Administration
voted 7-4 for approval of the drug known generically as
droxidopa, despite government concerns about whether the
treatment is safe and effective for treating chronic illness.
One committee member abstained and another did not cast a vote
The recommendation will now be taken into consideration by
the U.S. regulatory agency, which is expected to render a final
opinion by March 28. The drug has been in use in Japan since
1989.
Shares in the Charlotte, North Carolina-based
biopharmaceutical company, which had been halted on Thursday,
zoomed 66 percent to $4 in the afterhours market from their
$2.55 close in regular trading on Wednesday.
FDA approval for Northera would guarantee Chelsea
Therapeutics marketing exclusivity for seven years, according to
analysts.
There is only one currently U.S.-approved drug for
symptomatic neurogenic orthostatic hypotension, or NOH, called
midodrine hydrochloride, which Shire Plc <SHP.L> markets under
the brand name ProAmatine. The FDA review document said
midodrine may be removed from the market soon because it has
never demonstrated clinical effectiveness.
On Tuesday, the FDA released a staff review document in
which a government physician recommended against regulatory
approval.
NOH is a rare, chronic and often debilitating drop in blood
pressure on standing up, associated with Parkinson's disease,
multiple-system atrophy and pure autonomic failure.
Symptoms include dizziness, lightheadedness, blurred vision,
fatigue and fainting episodes, which often severely limit a
person's ability to do daily activities that require standing
and walking.
Many committee members said they voted in favor of the drug's
approval because research data, though limited, showed it could
provide remarkable benefits for some who suffer from a
devastating disease for which no other effective treatment is
available.
"If there were other treatments available, the answer would
be 'no.' But there aren't," added Allan Coukell of Pew
Charitable Trusts, the panel's consumer representative.
Several public witnesses provided by Chelsea Therapeutics,
including disease sufferers, told panelists that the drug had
produced life changing effects and had allowed them to lead
relatively normal lives.
"If a wheelchair-bound patient can now stand up and do his
day-to-day activities, that's huge for him," said Dr. Vasilios
Papademetriou, a cardiologist with the Veterans Affairs Medical
Center in Washington.
FDA staff advised the committee that strong evidence that
Northera can confer at least one week of benefits on patients
with NOH including the raising of standing blood pressure.
But the evidence showed no durable benefits that could
compensate for safety concerns stemming from 19 deaths in data
too limited to point to a cause, the staff said.
Company data also showed nine cases of life-threatening
neuroleptic malignant syndrome over a 10-year period in Japan.
The condition, that includes muscle rigidity and fever, is known
to be caused by drug use.
But most panelists concluded the safety risks, though little
known, were acceptable given the potential quality-of-life
benefits for a treatment population with limited life
expectancy.
"The safety profile of this is just as good if not better
than what we're using right now," said Dr. Jeffrey Cohen, a
neurologist at Dartmouth Hitchcock Medical Center. |
