What happened

Shares of BioMarin Pharmaceutical (BMRN 1.90%) rose more than 10% last month, according to data from S&P Global Market Intelligence. The rare-disease specialist reported promising results from an ongoing phase 2 trial of an important drug candidate, held its annual research and development (R&D) day for investors, and submitted a marketing application for a gene therapy in the European Union. 

Despite the monthly gains, shares of BioMarin Pharmaceutical have lost nearly 6% of their value since the beginning of 2019. It was much worse -- the pharma stock was down 24% on the same basis at the end of September -- but shares have rallied in recent months. Can it finish the year strong?

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Image source: Getty Images.

So what

It was a busy month for BioMarin Pharmaceutical. The company reported that children with achondroplasia, the rare disease more commonly referred to as dwarfism, achieved statistically significant gains in height versus what might be expected when treated with vosoritide. The treated individuals gained an additional 9 centimeters (3.5 inches) in height compared with untreated individuals, using a database of achondroplasia patients, in 54 months spanning a phase 2 and phase 3 trial. 

That was the highlight of the R&D Day, but investors have additional developments to look forward to. The business expects to complete regulatory submissions in the United States and Europe before the end of 2019 for its gene therapy (valoctocogene roxaparvovec) targeting hemophilia A. In fact, it submitted the marketing application in the European Union on Nov. 21.

BioMarin Pharmaceutical also announced that it has selected its next two pipeline candidates, including a third gene therapy drug candidate, and that it's exploring the potential to develop vosoritide in dominantly inherited short stature applications. The business told investors it can achieve breakeven or better net income in 2020 -- if the gene therapy in hemophilia A launches in the second half of next year.

Now what

The rare-disease specialist is now valued at over $14 billion. While the business won't be profitable in 2019, it did achieve operating income in the third quarter of 2019. Depending on the growth trajectory of currently approved products and regulatory outcomes in the next six months or so, BioMarin Pharmaceutical could finally become a profitable biotech stock in 2020 or shortly thereafter. Achieving that milestone will greatly de-risk future development and expansion activities, especially in the ultra-expensive gene therapy space.