logo
Share SHARE
FONT-SIZE Plus   Neg

BioMarin Pharma: FDA Accepts Vimizim BLA And Grants Priority Review Designation

BioMarin Pharmaceutical Inc. (BMRN) announced that the U.S. Food and Drug Administration or FDA has accepted for review the Biologics License Application or BLA for Vimizim (BMN-110, elosulfase alfa), an enzyme replacement therapy under evaluation for the treatment of patients with the rare lysosomal storage disorder Mucopolysaccharidosis Type IVA or MPS IVA, also called Morquio A Syndrome.

The company said that the FDA has granted priority review designation to Vimizim, which is granted to drugs that offer major advances in treatment, or provide a treatment where no adequate therapy exists. During the initial review of the application, the FDA requested additional Chemistry, Manufacturing and Controls or CMC information.

The company provided the information as requested, and the FDA designated it as a major amendment to the application thus extending the PDUFA action date by three months. The extended PDUFA action date is February 28, 2014.

In the FDA's filing communication, the Agency informed the company that it is currently planning to hold an advisory committee meeting to discuss the application. No date has been set for this meeting.

by RTT Staff Writer

For comments and feedback: editorial@rttnews.com

Business News

Quick Facts

Editors Pick
Shares of International Consolidated Airlines Group S.A. were gaining around 3 percent in London trading after the airline reported Friday higher profit in its second quarter, benefited by higher passenger revenues, despite weak cargo revenues. The company now expects low double digit percentage growth in pre-exceptional operating profit in 2016. Swiss banking giant UBS Group AG reported Friday a 14 percent decline in second quarter profit with weakness in net interest income. Looking ahead, the company said the difficult conditions continue to persist, yet the firm is well positioned to benefit from even a moderate improvement in conditions. UBS shares were gaining around 3 percent in the morning trading in Zurich. We are well into the third quarter and past the middle of the year and it is time to take stock of regulatory decisions awaiting some biotech stocks in August. Despite the uncertain macroeconomic environment, the markets have run up, with the S&P 500 Index making new highs session after session. Valuation concerns may keep traders at bay. However, the biotech space is a different cup of tea.
comments powered by Disqus
Follow RTT