|
 The FDA panel did not officially vote on an approval of the drug,
but 15 out of 17 members were of the opinion that the lack of
statistical significance in BioMarin's late-stage study weakened the
findings from two earlier studies.
The drug, drisapersen, is being developed to treat Duchenne muscular
dystrophy (DMD) - a disease that affects one in 3,600 newborn boys
and causes rapid muscle degeneration.
The FDA will decide on the approval of the drug by Dec. 27, after
considering the panel's comments.
The vote comes after FDA staff on Friday raised concerns about the
drug that included potentially fatal long-term side effects.
"I don't think they (BioMarin) will necessarily be able to convince
the FDA staff to change their mind," Heather Behanna, an analyst at
Wedbush Securities said, noting that additional data on the drug
could help making BioMarin's case.
But RBC Capital Markets analysts said the health regulator could
still find BioMarin's drug approvable given overall risks and
benefits.
The panel on Tuesday focused on the data and its interpretation
after hearing out 26 speakers, mostly parents who showed videos of
their boys benefiting from taking drisapersen.
Only one of the parents said the side effects were not manageable.
BioMarin's strategy going forward could be discussing with the FDA
on what else it could do, some analysts noted, after the health
regulator's staff on Friday posted their preliminary review.
Rival drugmaker Sarepta Therapeutics Inc is also developing a DMD
treatment. The drug eteplirsen, like BioMarin's drisapersen, skips a
faulty section of the gene to produce dystrophin, the lack of which
causes DMD.
[to top of second column] |
"Sarepta's review will also be tough. Their long-term prospects
because of better safety data are pretty good," Wedbush's Behanna
said.
The fate of Sarepta's drug, which will go through a similar vote in
January, will depend on how the FDA looks at dystrophin levels as a
biological indicator for DMD, Behanna added.
BioMarin's stock was placed on halt for all of Tuesday.
Sarepta shares were down about 1 percent at $36.98 after the bell
while shares of PTC Therapeutics Inc, which is also developing a
treatment for DMD, fell 5 percent in after-hours trading to $30.
(Reporting by Amrutha Penumudi and Vidya L Nathan in Bengaluru; ,
editing by G Crosse and Anil D'Silva)
[© 2015 Thomson Reuters. All rights
reserved.] Copyright 2015 Reuters. All rights reserved. This material may not be published,
broadcast, rewritten or redistributed.
 |
