BioMarin Pharmaceutical Inc. (BMRN - Analyst Report) recently received positive news when the company’s Biologics License Application (BLA) for its pipeline candidate Vimizim (BMN-110, elosulfase alfa) was accepted for review by the US Food and Drug Administration (FDA).
The BLA was submitted to the FDA earlier in the year. We note that BioMarin Pharma is seeking US approval of Vimizim (previously known as GALNS), for the treatment of patients suffering from mucopolysaccharidosis type IVA (MPS IVA) or morquio A syndrome.
The FDA will review the BLA for Vimizim on a priority basis. The FDA had requested for additional Chemistry, Manufacturing and Controls (CMC) information from the company while conducting an initial review. Upon receiving the required information from BioMarin Pharma, the FDA designated it as a major amendment to the application. Consequently, the Prescription Drug User Fee Act (PDUFA) date was pushed back by three months to Feb 2014.
BioMarin Pharma is also seeking approval for Vimizim in the European Union (EU) for the same indication. The European Medicines Agency (EMA) recently validated BioMarin Pharma’s Marketing Authorization Application (MAA) for Vimizim, submitted earlier in the year.
The EMA had earlier accepted BioMarin Pharma’s request for accelerated assessment for this MAA and the validation now allows EMA to start its formal review process.
BioMarin Pharma expects an opinion from the EMA’s Committee for Medicinal Products for Human Use (CHMP) on Vimizim in Dec 2013. The company expects a decision from the European Commission in the first quarter of 2014, provided the CHMP renders a positive opinion on Vimizim.
We expect investor focus to stay on the regulatory updates on Vimizim. We believe that an approval for Vimizin in both the US and EU and subsequent successful commercialization will augment BioMarin Pharma’s portfolio.
BioMarin Pharma currently carries a Zacks Rank #3 (Hold). Right now, Anika Therapeutics, Inc. , Gentium S.p.A and Aeterna Zentaris Inc (AEZS) look attractive with a Zacks Rank #1 (Strong Buy).