Better Buy: Vertex Pharmaceuticals vs. Alexion Pharmaceuticals

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Alexion Pharmaceuticals (NASDAQ: ALXN) and Vertex Pharmaceuticals (NASDAQ: VRTX) rank as two of the most successful biotechs focused on rare diseases on the planet. The stock performances for both companies underscore that success: Both Alexion's and Vertex's share prices have more than quintupled over the last 10 years.

But which of these rare-disease winners is the better pick for long-term investors now? Here's how Alexion and Vertex compare in three key areas.

Three scientists with image of DNA helix
Three scientists with image of DNA helix

Image source: Getty Images.

Current lineups

Alexion's Soliris generated nearly $3.6 billion in 2018 treating rare diseases blood disorder paroxysmal nocturnal hemoglobinuria (PNH), atypical hemolytic uremic syndrome (aHUS), and generalized myasthenia gravis (gMG). Sales for Soliris continue to grow, rising 9% year over year in the second quarter of 2019. The drug also picked up a new approved indication earlier this year for treating neuromyelitis optica spectrum disorder (NMOSD).

In addition to Soliris, Alexion has three other approved drugs: Strensiq, Kanuma, and Ultomiris. Strensiq, which is approved as a treatment for hypophosphatasia (a genetic disease that affects the development of bones and teeth), is currently the biggest seller of the three with 2018 sales totaling $475 million. PNH drug Ultomiris, however, is likely to be an even bigger winner. Market researcher EvaluatePharma ranked Ultomiris as its top new drug launch of 2019 and projects sales of $3.5 billion by 2024.

Vertex's current lineup includes three cystic fibrosis (CF) drugs: Kalydeco, Orkambi, and Symdeko. Kalydeco and Orkambi were blockbusters in 2018, racking up net sales of $1 billion and $1.3 billion, respectively. Symdeko is on track, though, to be the biggest winner this year.

Although Alexion's drugs have delivered impressive sales growth over the last three years, Vertex's trailing-12-month revenue during the period has more than doubled that of Alexion. I think the advantage in this category goes to Vertex based on this strong performance and continued excellent growth prospects for its current CF lineup.

Pipeline prospects

Alexion has several late-stage clinical studies for Ultomiris. It's evaluating the intravenous version of Ultomiris in treating aHUS, gMG, and PNH in children and adolescents under the age of 18, as well as a subcutaneous version of the drug in treating PNH and aHUS. The biotech plans to soon begin late-stage studies of the drug in treating NMOSD and hematopoietic stem cell transplant-associated thrombotic microangiopathy (HSCT-TMA). Alexion is also evaluating experimental drug ALXN1840 in treating Wilson disease, a rare genetic liver disorder.