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 The company's shares rose 6.5 percent to $131 in after-hours trading
on Wednesday.
The drug, BMN 111 (vosoritide), was evaluated in children with
achondroplasia, the most common form of dwarfism, BioMarin said.
The company said 10 children receiving the highest dose of 15
micrograms per kilogram of the injectable drug a day showed a 50
percent increase in their growth velocity, compared with their
annualized prior 6-month natural baseline growth velocity.
"This increase in growth velocity, if maintained, could allow
children with achondroplasia to resume a normalized growth rate,"
said Wolfgang Dummer, senior VP, clinical development.
All 26 children with an average age of 7.8 years showed a favorable
safety profile, the company said.
Vosoritide has an orphan drug status in the United States and
Europe.
The status gives a developer several incentives including a
seven-year market exclusivity in the United States and 10-year
exclusivity in the Europe.
The drug if approved could rake in sales of about $700 million
worldwide, Raluca Pancratov of SunTrust Robinson Humphrey wrote in a
note to clients.
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Achondroplasia is a genetic disorder that affects about 1 in
15,000-40,000 newborns.
It is a form of short-limbed dwarfism. Average adult height for men
with achondroplasia is 131 cm (4ft 3 in) and for women 124 cm (4
ft).
BioMarin's pipeline includes five approved drugs for the treatment
of various rare diseases.
Shares of the company, which in November agreed to buy Dutch drug
developer Prosensa Holding NV for about $840 million, closed up 2
percent at $123.60 on the Nasdaq.
(Reporting by Rosmi Shaji in Bengaluru; Editing by Sriraj Kalluvila)
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