Although the stock market has been struggling for most of the year, some companies have performed very well. Biotech giant Vertex Pharmaceuticals (VRTX 1.70%) is an excellent example. The drugmaker's shares are up by 30% year to date, while the S&P 500 is down by nearly 21%. Although Vertex Pharmaceuticals is firing on all cylinders, the future could be even brighter. Let's consider why this biotech company is still worth investing in. 

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Business is booming 

Vertex has made a fortune thanks to its cystic fibrosis (CF) franchise. CF is a rare progressive disease that affects patients' lungs and other internal organs. The company currently markets the only drugs that treat the underlying causes of this rare disease. Leadership in this therapeutic area has allowed the drugmaker to grow its revenue and earnings for the past 10 years.

Vertex Pharmaceuticals' Kalydeco became the first medicine to treat the underlying causes of CF approved by the U.S. Food and Drug Administration (FDA) back in 2012. But CF is caused by dozens of mutations, and no single medicine Vertex has developed can address the entire patient population. Thankfully, in 2019, the FDA gave the green light to Vertex's Trikafta, which can treat up to 90% of CF patients.

Vertex Pharmaceuticals is working on potential therapies to address the remaining 10% of the CF population. Vertex's revenue jumped by 22% year over year to $2.10 billion in the first quarter. The company's bottom line increased by 17% year over year to $762 million. Of the 83,000 CF patients in North America, Europe, and Australia, more than 25,000 are eligible for Vertex's current therapies but have yet to start treatment.

Vertex Pharmaceuticals' revenue and earnings will continue growing as its CF lineup makes progress within its target market. 

Exciting new programs 

Vertex Pharmaceuticals will have to develop new medicines and diversify away from its CF franchise to remain successful in the long run. And to that end, the company has been looking to develop groundbreaking new treatments. Vertex Pharmaceuticals is, for the most part, still going after serious or rare diseases.  There are only few (if any) therapies approved that treat the underlying causes of some of the illnesses it is targeting.

If Vertex Pharmaceuticals can replicate the success it has had in the CF market in one or two other therapeutic areas, the company could continue on its upward path for many more years to come. Vertex Pharmaceuticals' pipeline programs include VX-147, a potential treatment for APOL1-mediated kidney disease, a serious kidney-related disorder caused by mutations in the APOL1 gene. There are no approved therapies that treat the underlying causes of this illness.VX-147 is currently undergoing a phase 2/3 clinical trial.

Perhaps Vertex Pharmaceuticals' most promising pipeline candidate is CTX001, a potential treatment for sickle cell disease and transfusion-dependent beta-thalassemia (TDT), two rare blood-related disorders. CTX001 promises to be a one-time curative treatment for these two diseases that have very few effective treatment options.

Vertex and its partner on this program, CRISPR Therapeutics, plan to submit CTX001 to regulatory authorities for review by the end of the year. The medicine has already produced very encouraging results in studies . For instance, all 15 TDT patients enrolled in a phase 1/2 clinical trial by Vertex Pharmaceuticals were transfusion-independent after treatment with CTX001, with follow-up ranging from four to 26 months. TDT patients typically need regular blood transfusions.

Permanently ridding this patient population of this need would be a significant win for the patients and their families. It could also reduce healthcare costs associated with the illness. In other words, CTX001 would be a big deal if approved. Vertex Pharmaceuticals expects this medicine to be its next commercial launch.

Just getting started 

Vertex Pharmaceuticals has outperformed the market this year, in the trailing-12-month period, and over the past 10 years. But it isn't too late to get in on this biotech giant. Vertex Pharmaceuticals still has miles of room to grow within its main CF market, and it is developing several promising medicines. Vertex Pharmaceuticals will likely prove to have been a market-beating investment again in another decade.