Creating Opportunity Cassie Hodges  | March 20, 2017

Meet the World’s 5 Most Innovative, Disease-Destroying Pharmaceutical Companies

These days, when someone says the word “innovation,” we tend to think airborne drones and mobile apps, wearable technologies and driverless cars. However, some of the most impactful and cutting-edge innovation continues to occur in one of our country’s legacy industries: pharmaceuticals.

In fact, five of the 10 most innovative companies in the world, according to the latest rankings by Forbes, hail from America’s pharmaceutical sector. Every one of them is pushing medicine and health care treatments to previously unimaginable heights, and in the process, they’re forever improving the lives of patients with some of the world’s most serious ailments and diseases.

According to Forbes’ stated methodology, the companies were ranked by their innovation premium; that is, the difference between their market capitalization and the net present value of cash flows from existing businesses. Here’s a closer look at the five innovative pharmaceutical players that made the list.


Regeneron Pharmaceuticals

Regeneron Pharmaceuticals, based in Tarrytown, New York, lands at No. 3 on Forbes’ list. With sales eclipsing $4 billion, Regeneron specializes in inventing, developing, and commercializing medicines for treating serious medical conditions, including various types of cancer, arthritis, dermatitis, asthma, and infectious diseases. The 28-year-old company is responsible for more than 5,000 jobs, and it has been ranked No. 1 in Science magazine’s global Top Employer survey in four of the past six years.

On the innovation front, Regeneron has produced three FDA-approved medicines, including EYLEA Injection, a prescription medication that treats diseases of the retina such as diabetic retinopathy. Over the past two years, Regeneron Pharmaceuticals also entered into an agreement with the federal government to develop a new antibody treatment for the Ebola virus and launched a new immuno-oncology collaborative relationship with global healthcare giant, Sanofi.



Coming in at No. 4 on Forbes list is Incyte, a biopharmaceutical company based in Wilmington, Delaware, and focused on the development of proprietary therapeutics to treat serious unmet medical needs, primarily in the area of oncology. The company is most well known for a product called Jakafi, which is currently approved in the U.S. to treat intermediate or high-risk myelofibrosis – a serious bone marrow disorder – and is in development as a potential treatment for other cancers.

Incyte has announced a number of clinical trials currently underway in the oncology space, and the company has released a list of their sponsored studies and investigator-sponsored trials. In addition, Incyte emphasizes community engagement through its philanthropy-focused initiatives like the Incyte Charitable Giving Foundation, Community Service Program and the Matching Gifts Program.


Alexion Pharmaceuticals

Alexion Pharmaceuticals, based in New Haven, Connecticut, was also tapped as one of the five most innovative companies in the world. A global company that focuses on therapies for patients with rare disorders, Alexion is responsible for a world-renowned product, Soliris, the only approved complement inhibitor that treats patients with two life-threatening and ultra-rare disorders: paroxysmal nocturnal hemoglobinuria and atypical hemolytic uremic syndrome.

Alexion employs over 3,000 individuals serving patients in 50 countries. Recently, it has introduced two innovative enzyme replacement therapies for patients with hypophosphatasia and lysosomal acid lipase deficiency, both extremely rare and life threatening disorders.


Vertex Pharmaceuticals

Vertex Pharmaceuticals, a Boston-based company that landed at No. 9 on the list, has research and development sites and commercial offices in the United States, Europe, Canada and Australia. Vertex specializes in clinical development programs with a focus on cystic fibrosis. Vertex employs around 2,000 people has seven times been named one of Boston’s Top Places to Work.

The company, which was founded in 1989, has two approved medicines on the market – ORKAMBI and KALYDECO used to treat specific types of cystic fibrosis – and currently has more than a dozen ongoing research programs aimed at life-threatening diseases. Vertex employs approximately 2,000 individuals, and for the seventh time, Vertex has been named one of Boston’s Top Places to Work.


BioMarin Pharmaceuticals

Rounding out the list, BioMarin Pharmaceuticals, based in San Rafael, California, has five products on the market and multiple clinical and pre-clinical products in its pipeline. BioMarin provides innovative therapeutics to those with rare and ultra-rare unmet medical needs driven by genetic causes, including Lambert-Eaton Syndrome, an autoimmune disease in which the immune system attacks the body’s own tissues.

Chairman and CEO Jean-Jacques Bienaimé has attributed the company’s recently surging growth in commercialized products to the global launch of its most recent product, Vimizim, which is used to treat Morquio A Syndrome, which is a progressive inherited disease of the metabolism. BioMarin is also working through an early phase trial with a gene therapy product intended to treat patients with hemophilia A (a genetic disorder that affects blood clotting).

If successful, Bienaimé told Forbes that, “Gene therapy could represent not only a medical first for hemophilia A patients, but a new technology platform that could change the way we think about treating rare genetic diseases.”