Washington Editor

WASHINGTON – Instead of running dry, the drug pipeline for rare diseases may be flowing with hope. As of last month, more than 450 drugs were in clinical trials for rare diseases or awaiting FDA approval.

More than a third of the drugs are being developed to treat rare cancers, and nearly 70 are targeting genetic disorders such as cystic fibrosis, according to a new report from Pharmaceutical Research and Manufacturers of America (PhRMA). Drugs also are being developed to treat infectious diseases, help with organ transplants and treat autoimmune, blood, cardiovascular, eye, gastrointestinal, growth, neurological and respiratory disorders.

The bulk of the drug candidates are in Phase II and III trials, but nearly 30 have applications pending at the FDA. Altogether, 2,300 drugs, in all stages of development, had been granted orphan drug status as of Jan. 24.

"This is . . . a time for hope," PhRMA President and CEO John Castellani said in the report. "Biopharmaceutical research is entering an exciting new era with a growing understanding of the genome" and new research tools to study complex diseases.

Since the Orphan Drug Act was passed in 1983, the FDA has approved more than 350 drugs to treat rare diseases. Fewer than 10 were approved in the 1970s, Castellani said.

The report gives the odds in getting a drug from the lab to approval, a process that can take 15 years in the U.S. One in 5,000 compounds in preclinical testing will make it to human testing; therapies tested in humans have a 20 percent chance of being approved.

The National Institutes of Health (NIH) is opening a National Center for Advancing Translation Science (NCATS) to help reduce those odds by taking some of the risk out of the early stages of drug development. In announcing the Oct. 1 opening of NCATS, NIH Director Francis Collins noted Wednesday that of 6,000 identified rare diseases, only 200 have therapies. Rare disease are defined as those that affect fewer than 200,000 people. (See BioWorld Today, Feb. 24, 2011.)

House Wants Answers About Heparin

The FDA is once again under scrutiny for the way it investigated contaminated heparin imported from China in 2008.

Noting that it's been nearly three years since the FDA linked 81 deaths and serious allergic-type reactions to imported heparin that was adulterated with overly sulfated chondroitin sulfate, the House Energy and Commerce Committee is demanding answers. (See BioWorld Today, April 23, 2008.)

"Neither the Chinese government nor the FDA has identified those responsible for the contamination or described how the heparin actually came to be contaminated," committee Chairman Fred Upton (R-Mich.) said in a Feb. 23 letter to FDA Commissioner Margaret Hamburg.

With more than 80 percent of the U.S. unfractionated heparin supply and 16 percent of pharmaceutical ingredients coming from China, "there is reason to believe all or some of the individuals responsible for the adulteration are still actively engaged in the Chinese pharmaceutical supply chain," the letter said.

As part of its investigation into how the FDA handled the contaminated blood-thinner, the committee is asking the agency to provide it, by March 9, with all documents regarding:

• heparin-related inspections conducted by the FDA in China;

• possible sources and methods of the adulteration;

• testing result information on heparin lots submitted by companies to the FDA for screening.

The Government Accountability Office (GAO) also has been critical of the FDA's investigation. In an October report, the GAO said the agency had inspected several Chinese heparin companies, but some of them refused to give the inspectors full access to records or to conduct a complete inspection. Despite these refusals, the FDA allowed the companies to continue importing products into the U.S. (See BioWorld Today, Nov. 11, 2010.)

Vaccine Preemption Upheld

The U.S. Supreme Court handed vaccine manufacturers a victory this week when it ruled that federal law preempts vaccine design defect claims.

In the 6-2 ruling in Bruesewitz v. Wyeth LLC, the court said the National Childhood Vaccine Injury Act of 1986 expressly eliminates liability for unavoidable adverse side effects of a vaccine that's manufactured and labeled in accordance with its FDA-approved license.

"The ruling confirms that expert federal agencies . . . are best suited to determine the optimal design of life-saving childhood vaccines," noted the Biotechnology Industry Organization. "In addition, the ruling recognizes the critical balance struck by the Vaccine Act to promote the development of advanced and even safer vaccines while providing a system for the fair resolution of injury claims."

In a dissent, Justices Sonia Sotomayor and Ruth Bader Ginsburg said the "decision leaves a regulatory vacuum in which no one ensures that vaccine manufacturers adequately take account of scientific and technological advancements when designing or distributing their products."

They added that manufacturers have a "legal duty, rooted in basic principles of products liability law, to improve the designs of their vaccines in light of advances in science and technology."

The lawsuit involved a diphtheria, tetanus and pertussis vaccine made by Lederle Laboratories, which became part of Wyeth. (See BioWorld Today, Oct. 18, 2010.)

FTC Appeals Denial of LabCorp Injunction

The Federal Trade Commission (FTC) isn't taking no for an answer in its quest to enjoin Laboratory Corp. of America's $57.5 million acquisition of Westcliff Medical Laboratories, of Santa Ana, Calif. The FTC is appealing a federal district court's rejection of a preliminary injunction to keep LabCorp, of Burlington, N.C., from integrating the Westcliff assets. It also is asking the U.S. District Court for the Central District of California to grant an injunction pending the appeal. The FTC challenged LabCorp's acquisition of the rival clinical laboratory testing company in December, alleging that it would harm competition.