Henri: N of 1

How the late Genzyme CEO Henri Termeer created the Orphan drug industry

Gaucher’s disease had swollen Brian Berman’s spleen to the size of a basketball and physicians at NIH told the three-year-old’s parents that it had to be removed.

It was 1984, there was no effective treatment for the rare genetic disorder, and the surgery would do nothing to change a prognosis that was grim and certain: years of deformity and unremitting pain relieved only by death.

Brian’s mother knew NIH was collaborating with a tiny biotech company called Genzyme to develop an experimental medicine for Gaucher’s disease. She pleaded with the research team to enroll her son as the first patient in a trial of the experimental enzyme replacement therapy.

Within a few months, the formerly listless boy was racing around the halls of NIH’s clinical center. Brian’s response to the treatment astonished his doctors and delighted his mother. It also transformed the life of Henri Termeer, Genzyme’s president.

Witnessing Brian’s recovery turned Termeer from a man determined to succeed at business into one dedicated to creating life-altering drugs. He needed a successful company do this, and was relentless -- some would say ruthless -- about creating that company. But from the moment he saw an experimental drug bring a child back to life, business became the means and creating life-saving medicines his goal.

“We wanted to make a big difference, not a small difference.”

Henri Termeer

Over the following seven years Termeer pursued the commercialization of what became Ceredase alglucerase with a persistence that at the time looked to many people like irrational stubbornness. His conviction that the therapy could and must be developed -- in the face of clinical evidence that it didn’t work, and despite daunting scientific, financial and regulatory challenges -- was fueled by images of Brian Berman’s exuberance.

In retrospect, the path that led from witnessing a small dying boy regain his life to building a multibillion dollar biotech company looks logical and premeditated. In fact, Termeer had no grand plan, many of his best moves were improvisations, and there were many instances where good fortune intervened to save his company from failure.

As news spread that Termeer had died suddenly on the night of May 12, Berman, now 37 and president and CEO of the National Gaucher Foundation, was among the patients and colleagues who remembered him as a visionary who overcame immense obstacles to launch the Orphan drug industry.

Termeer’s fundamental insight was that drugs for extraordinarily rare conditions had to be priced at levels that made developing them a viable, financially sustainable business. The way he did this was immensely controversial in the 1990s, and that controversy has exploded beyond the boundaries of markets for very rare conditions and has become the biggest challenge facing the biopharma industry.

Termeer was a member of a small group of entrepreneurs who grew a disparate group of fledgling businesses into a biotech industry that has become the world’s biomedical innovation engine. Along the way he pioneered patient-centered drug development, helped forge biotech’s public policy agenda, and inspired a generation of entrepreneurs to pursue the toughest, most important challenges.

A ‘Baxter boy’

Termeer was born in 1946 in Tilburg, the Netherlands, which he described in oral history interviews conducted by the Life Sciences Foundation as the “eighth-largest town in a very small country.”

After setting aside dreams of becoming a chess grandmaster, Termeer’s ambition starting as a teenager was to become a business leader. While studying economics at Erasmus University Rotterdam he traveled to a shoe company in the U.K. to write a thesis about how computers could be applied to inventory control. The company liked the plan so much that it hired him to implement it, so Termeer didn’t graduate.

Termeer learned that American business schools were recruiting international students, enrolled in the University of Virginia’s Darden School of Business and graduated with an MBA in 1973.

He caught the attention of a recruiter from Baxter Travenol Laboratories Inc. (now Baxter International Inc.). The company had created a program to recruit MBAs from leading business schools and fast-track them into senior management positions. In doing so, Baxter inadvertently provided the biotech industry with a cohort of early leaders.

A Harvard Business School study has noted that a disproportionate number of the CEOs of early biotech companies were former Baxter employees. More than one in five biotechs that went public between 1979 and 1996 had what the study dubbed a “Baxter boy” on the IPO team.

Baxter alumni included Gabriel Schmergel, who became CEO of Genetics Institute Inc.; Ted Greene, who when on to lead Hybritech Inc. and later founded Amylin Pharmaceuticals Inc. and Robert

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