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Biomarin- Gene Therapy and Hemophilia

Biomarin (BMRN) just recently announced that the FDA has accepted for Priority Review the BLA for ValRox (valoctocogene roxaparvovec) for adults with hemophilia A, notes biotech expert John McCamant, editor of The Medical Technology Stock Letter

This constructive action by the FDA marks the first marketing application accepted for a gene therapy (GT) product for any type of hemophilia in the United States. The PDUFA date is August 21, 2020.

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For the past 2+ years, we have known that the FDA has been proactive with gene therapy companies with an emphasis on hemophilia. Hence, the recent news may not be the biggest surprise.

However, with specific issues of variability and durability relating to ValRox data to date, the BLA acceptance is absolutely good news. Moreover, the agency also stated that, as of now they do not expect to convene an outside Advisory panel before the PDUFA. This tells us that a few things.

First, the agency believes it has enough information to make decision on its own. And second, the agency has beefed up its own internal gene therapy (and likely gene editing) efforts since ~2018.

Of course, we won’t know the final answer until this summer. In the meantime, investors will continue to not only assess the likelihood of ValRox approval but also the potential market size and competitive landscape.

We know Biomarin has a major lead time over the other players in hem A GT. The company has made enormous investments in gene therapy manufacturing and in our view, bodes incredibly well for ValRox and the company with regards to the FDA acceptance of its application. 

Either way, the floodgates for hem A gene therapy are open once more. As a reminder, back in January the EMA also validated the firm's ValRox application and that, too, is scheduled for an accelerated review.

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This is important positive news for Biomarin and the revolutionary advances for patients via biotechnology. We believe the stock is undervalued at current levels, and remains ripe for an acquisition. The latest news should help that process along.

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