Martine Rothblatt is an idealist and the CEO of a very successful biotech firm focused on treatments for so-called "orphan diseases" - those illnesses that affect too few to be of interest to big pharma.
When her seven-year-old daughter developed pulmonary arterial hypertension (PAH), a life-threatening disease that restricts blood flow between the heart and lungs, Rothblatt found that the best treatment was sitting on a shelf at GlaxoSmithKline (GSK). So she bought the patent, promising Glaxo 10% of revenues and founded United Therapeutics (UTHR), which marketed the drug, known as Remodulin, as well as two other PAH mediciations. And late last year the FDA approved a pill form of the drug called Orenitram. (Remodulin is injected).
Related: 4 picks in biotech
Rothblatt’s daughter, who suffers from PAH, is now 30 years old, and United Therapeutics has a market value topping $6 billion. Its shares have gained more than 2,000% since the company’s IPO in 1999.
Rothblatt says government support is key for the future of the biotech industry. Legislation currently provides manufacturers of orphan drugs seven years of market exclusivity, plus tax credits. The legislation, known as the Orphan Drug Act, which was amended in the latest version of the Drug Price Competition Act, “has helped many new biotech companies grow up and get some revenues,” says Rothblatt.
Related: Yes, biotech is a bubble
But she tells Yahoo Finance in the video above that more government help is needed for the National Institutes of Health, where research is done, and for the National Organization of Rare Disorders, and any medicines that serve small populations.
The future of medicine, says Rothblatt, will be personalized - because “each person’s disease is a thing unto itself.”
Martine Rothblatt is the author of Virtually Human, out this month.
More from Yahoo FInance