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Joan didn’t want to lose her voice, to have to talk through a squeak box. But that would be her fate if she agreed to a permanent tracheotomy.
Her doctors feared for her life. For the second time in six years, the 48-year-old Pennsylvania woman’s airway had swelled shut, due to a rare inflammatory disease called hereditary angioedema, or HAE. They wanted to keep the tube in so it wouldn’t happen again.
Joan’s husband, desperate, sought out the HAE association, and found there was a trial underway for a potential treatment for the disease. It was 2007, the drug target was then called Ecallantide, and it was being developed by Dyax Corp. in Cambridge.
The drug provided vital relief to Joan and others in the trial. This week, the drug, now called Kalbitor, launched in the United States after it secured FDA approval in December.
It is the end of what Dyax officials call an 18-year roller-coaster ride to get the company’s first drug approved, and it is the beginning of its new life as a commercial specialty biopharmaceutical company. With this approval of a rare-disease drug, the company joins the ranks of success stories like Cambridge-based Genzyme Corp. and U.K.-based Shire plc. But with just over 100 employees, a year’s worth of cash and only 6,500 known cases of HAE, the company still faces many hurdles. But Dyax, which went public in 2000, is used to challenges.
Last year, Dyax was near the finish line. It’s VP of Medical Affairs Patrick Horn sat in front of an FDA advisory committee in Washington, D.C., last February with 600 slides defending Kalbitor’s safety and efficacy at the ready. The committee only asked to see two of them, in a five-hour session that made Horn feel more like a lawyer presenting to the U.S. Supreme Court. Then came the vote. Five committee members had voted no, two abstained, and six voted to recommend approval.
Kalbitor had just squeaked by. The FDA wasn’t required to follow the committee’s lead. But it was a very good sign. However, the subsequent elation at the Kendall Square office was short-lived; a month later, Dyax received a dreaded “complete response” letter from the FDA. The agency wanted more data.
This meant a delay of at least six months. It was time — and money — the company didn’t have. Dyax had $52.2 million in cash, cash equivalents and short-term investments as of March 30, and the company was burning through $15 million per quarter.
“It was so terrible. These people had been with the company for so long. It was the lowest point,” Robert Ladner, the inventor of Kalbitor, said about the day the company laid off 60 workers, or 36 percent of its staff. In the next nine months the company would make several other painful strategic decisions, including halting early-stage research and selling shares for as low as $2.02 apiece through follow-on offerings. Dyax’s (Nasdaq: DYAX) stock hovered around $3.50 a share this week. The stock has traded between $1.55 and $4.69 over the past year.
Approval means that revenue from the drug will slowly start rolling in. But the firm also faces new expenses related to marketing and selling the drug. Those costs will likely be intensified because Kalbitor has two new competitors. A year ago, a drug made by Exton, Pa.-based ViroPharma Inc. was approved to be used as a prophylactic treatment for HAE.
A drug made by CSL Behring, a subsidiary of Australia-based CSL Ltd. to treat HAE for acute attacks, was approved six weeks before Kalbitor.
Dyax officials say neither drug is a direct competitor because the ViroPharma drug is not approved for acute attacks and the Behring drug is not approved for the life-threatening attacks that affect the larynx. Dyax refused to disclose the price of Kalbitor, but it will have to share a total market of between $515 million to $810,000 million per year.
Dyax CEO Gustav Christensen is unfazed.
“Behring had a six-week headstart on us with their drug. But we are launching just a week after them. A small company is a better fit for this kind of drug because we will focus all our attention on it,” he said.
The company has hired 18 salespeople, five medical liaisons and two reimbursement specialists to ensure patients’ insurance will pay for Kalbitor.
Joan said it looks like her insurance will pay for the drug in full. And instead of traveling an hour to the clinical trial site, she’ll be able to receive the under-the-skin infusion at a nearby hospital whenever she gets an attack. Joan said she hopes this means she can work again, after 13 years of disability.
“This is going to change my life,” she said.
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