AUSTIN (KXAN) - A top executive of BioMarin Pharmaceutical told shareholders this week that BMN 673 results have been "as good as it gets" when it comes to clinical trial studies.
"At the moment, our Phase 2 data is as good as anybody else's," said, Dan Spiegelman, the California drugmaker's chief financial officer. "As a phase two small patient study goes, it's as good as it gets. We think our numbers stack up well at this level. The drug is clearly the most potent of the compounds we're talking about."
Spiegelman made the remarks at the Morgan Stanley Global Healthcare Conference in New York City on Monday, the same day Austin's Andrea Sloan was in the nation's capital seeking help persuading BioMarin to give her access to the drug she hopes can save her life.
On Tuesday, at the Baird Healthcare Conference in New York City, Spiegelman said there will be more data released about the drug later this month during the European Cancer Congress.
"But for both the ovarian and breast cancer phase 2, we ran the response rates were in the 44 to 50 percent range and we're excited about starting the phase 3 in breast cancer in patients with the BRCA mutation breast cancer," he said.
Meantime, Sloan, who is battling ovarian cancer, spent Wednesday meeting with lawmakers in Washington, D.C, fighting for a compassionate-use exception from BioMarin to be allowed to take BMN 673, which is not yet FDA approved.
In a statment to KXAN, BioMarin said "it is too early to know if the experimental therapy is safe or effective, or will even prolong life, until we conduct the appropriate Phase 3 trials."
"You've got to run the race," Spiegelman said at Monday's conference. "We'll know the answer in Phase 3."
Sloan, a seven-year survivor of ovarian cancer, can no longer tolerate traditional therapies and her doctors at MD Anderson in Houston believe BMN 673 is the best hope for prolonging her life. Sloan told Huffington Post Live on Wednesday she is willing to accept the risks of taking the drug before it is FDA approved.
"With the way that medicine advances, there is still great hope for me out there in the form of this drug that has not yet been approved by FDA," Sloan said. "I as the patient need to accept the risks of taking a pre-approved drug. I'm 100 percent willing to accept those risks."
A BioMarin spokesperson told KXAN the company supports expanded access, or compassionate use, programs.
"We implement these programs when we have sufficient scientific evidence to support both the safety and the efficacy of a product," the statement read.
But the company has not developed a compassionate use policy specifically for BMN 673.
"I'm not sure why BioMarin on the one hand says that they support compassionate use and on the other hand communicates to me and my doctors that they do not have a compassionate use policy," Sloan said Wednesday.
The FDA has already indicated Sloan would be a good candidate for compassionate use, but BioMarin must make the request to the FDA and have, for weeks, declined to do so.
Time is short for Sloan, whose doctors have said she needs to begin treatment within weeks in order for that treatment to be effective.
Late Saturday night into early Sunday morning, a light band of freezing drizzle traversed the I-35 corridor eastward. With sub-freezing temperatures, even the light precipitation created major problems.
A 10-year-old was killed while standing outside a vehicle after the child's family was involved in a fender bender, DPS said.
APD is responding to a 25 vehicle accident near the 5400 block of Ed Bluestein near Thurgood Ave.
A man is dead after being hit by several vehicles in the eastbound lane of Highway 71 Saturday night.
A representative at the Fayette County Sheriff's office said that Fayette County is effectively shut down due to icy conditions.
Freezing drizzle causing dangerous conditions on roadways overnight.