With a total R&D spend of USD 2.66 billion in 2022 and a pipeline that features cutting-edge technologies such as mRNA, cell therapy and gene editing, Vertex Pharmaceuticals is looking to deliver “serial innovation” by investing most of its resources in research. And while some other biopharma companies depend on external innovation through acquisitions, 40 percent of Vertex’s pipeline stems from its internal programmes, backed up by a solid revenue stream from its cystic fibrosis medicines.


In seven years, we went from being able to treat a handful of cystic fibrosis patients to treating 90 percent of them

Jeffrey Leiden, executive chairman

Failed Hepatitis C Beginnings and Cystic Fibrosis Success

The Boston-based biotech, founded in 1989, has had a distinctively innovative spirit since its beginnings when it was one of the first biopharma companies to use an explicit strategy of rational drug design instead of combinatorial chemistry. The company has continued to investigate new treatment avenues with novel approaches ever since.

Vertex’s first success came from Incivek, a hepatitis C medicine approved in 2011 that soon became a commercial blockbuster with sales peaking at USD 1.1 billion in 2012. Unfortunately, this promising start turned into a flop as steep competition from more effective hepatitis C drugs came from companies like Gilead.

But Vertex bounced back by deciding to focus on the genetic condition, cystic fibrosis (CF), a strategy that paid off with a string of ground-breaking therapies. In 2012, the FDA approved its Kalydeco, which was the first drug to treat the underlying cause of CF. While Kalydeco brought in sales of USD 371 million by 2013, it was only capable of treating about 4 percent of CF patients, still leaving many patients without treatment options.

The biotech forged ahead in the CF area, looking to expand the number of CF patients that could be effectively treated and its second CF drug, Orkambi, was approved in 2015, followed by Symdeko in 2018. Both products —two-drug combinations backboned by Kalydeco— dramatically increased the percentage of patients that could be treated, a number that increased further with the approval of the company’s fourth CF drug, Trikafta, in 2019.

“In seven years, we went from being able to treat a handful of cystic fibrosis patients to treating 90 percent of them now, with Trikafta. That’s historic. It’s never been done in the industry before,” said then CEO Jeffery Leiden at the time.


Beyond CF: The World’s First CRISPR Gene Editing Therapy

Vertex has proved the success of its aggressively innovative approach in CF and continues its research in the area, having initiated clinical trials for an mRNA candidate in partnership with Moderna that has received an FDA Fast Track designation. At the same time, the company has continued to look to ground-breaking technologies such as gene editing to diversify its efforts beyond CF and address other therapeutic areas. Vertex’s diversified R&D efforts have proven to be especially productive in six other disease areas, with positive results in five of its programs in Phase 2 development.

Perhaps the most promising among Vertex’s candidates is the exa-cel gene therapy for sickle cell disease and beta thalassemia it has developed in collaboration with CRISPR Therapeutics. Exa-cel uses CRISPR/Cas9, a revolutionary gene editing method, and will potentially provide a one-time functional cure. Having concluded submissions in Europe, the UK and the US, the company anticipates a commercial launch in 2023.

“The completion of our exa-cel global regulatory filings is a historic milestone,” said Carmen Bozic, M.D., Executive Vice President, Global Medicines Development and Medical Affairs, and Chief Medical Officer at Vertex Pharmaceuticals.  “Within a decade, we have progressed from the discovery of the CRISPR platform to the first regulatory filings for a CRISPR-based therapy,” agreed Phuong Khanh (P.K.) Morrow, M.D., FACP, Chief Medical Officer at CRISPR Therapeutics in a release.


Plans for the Future

Vertex has also made breakthroughs in other therapeutic areas such as pain management, APOL1-mediated kidney disease and Type 1 Diabetes and says that it aims to bring new products to five disease areas within the next five years. This goal is based not only on the firm’s promising pipeline, but also on its strong financial footing. The company reported full year product revenues of USD 8.93 billion for 2022, which represented an 18 percent increase compared to 2021.

“Outstanding execution across the company resulted in another year of strong revenue growth as well as acceleration of both the research and clinical-stage pipeline,” said Reshma Kewalramani, M.D., Chief Executive Officer and President of Vertex. “Our progress in 2022 lays the foundation for treating more people with cystic fibrosis, launching multiple new therapies in the near term, achieving important clinical milestones, and driving continued significant growth for many years to come.”

(Image Source: Vertex)