Biotech Ideas

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Vertex Pharmaceuticals - >>> Vertex Pharmaceuticals (VRTX 1.23%) has remained the premier presence in the cystic fibrosis treatment space for over a decade now since its first therapy to treat the underlying factors that cause the genetic disease was given the green light by the U.S. Food and Drug Administration.


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Since that time, Vertex has seen its portfolio of CFTR modulators -- the class of drugs that work to correct the root cause of cystic fibrosis -- expand to four approved therapies, and it remains the only company with approved CFTR modulators on the market at the time of this writing.

While its portfolio of top-selling therapies faces consistent and growing demand -- management estimates that as many as 20,000 cystic fibrosis patients could take its CFTR modulators but aren't yet doing so -- Vertex is looking toward future sources of growth to spur its business and financials forward in the years ahead.

Its current pipeline includes a non-opioid drug candidate for acute pain, a cystic fibrosis drug for patients who can't take CFTR modulators (management estimates that more than 5,000 patients fit this category), and a potential one-time functional care for two rare blood disorders, for which it's in the process of undergoing regulatory submissions.

The company is also wading into the lucrative diabetes care market. Last year, Vertex finished its acquisition of ViaCyte, a company that is working on stem cell therapies to treat type 1 and type 2 diabetes. The cystic fibrosis treatment market is set to reach a valuation of $32 billion by the year 2027, achieving a compound annual growth rate of more than 24% from its 2019 valuation of about $5 billion.

However, the broad opportunities within this space are just the tip of the iceberg for the long-term potential that Vertex appears to have as it continues to explore lucrative yet underpenetrated segments of the rare-disease drug market. For investors searching for a healthcare stock in which to retain a multi-year, buy-and-hold investment, Vertex looks like a compelling choice to consider.

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