WASHINGTON – Fiscal 2011 produced a bumper crop of new drug approvals with historic breakthroughs in a number of areas. But the yield on biologics actually dropped.

The FDA's Center for Drug Evaluation and Research (CDER) approved 30 new molecular entities (NME) and five biologic license applications (BLA) in fiscal 2011. However, two of the BLAs were for the same biologic – Seattle Genetics Inc.'s Adcetris (brentuximab vedotin), which was approved for two different lymphoma indications.

On a calendar year basis, CDER approved six new biologics in 2009 and six in 2010, agency spokeswoman Karen Mahoney told BioWorld Today. Only three were approved in 2008.

Although the agency generally tallies its approvals on a calendar year basis, it released a report last week lauding the approval of the 35 NMEs and new biologics in fiscal 2011, which ended Sept. 30, as an example of its flexibility in approving important new drugs in a timely manner.

The agency said it accelerated the review and approval of many of the drugs by using "expedited approval" pathways and streamlining clinical trial requirements to permit smaller, shorter, or fewer studies when possible.

Those tools only help, though, when there are strong applications to review. Seven new BLAs were submitted in 2008, seven in 2009 and only four in 2010, Mahoney said. The number of BLAs submitted in 2011 was not available.

Because the review time takes several months, BLAs submitted in one year may not be approved until the following year.

Regardless of the number of BLAs approved, the agency's flexibility was apparent. Adcetris, for instance, was approved in less than six months on the strength of one single-arm trial in a limited number of patients for each indication. The accelerated approval made Adcetris the first new Hodgkin's drug in more than 30 years and the first drug approved in the U.S. to treat systemic anaplastic large cell lymphoma. (See BioWorld Today, Aug. 22, 2011.)

Offering another breakthrough, Human Genome Sciences Inc.'s Benlysta (belimumab) also raced through the approval process in less than six months, becoming the first new systemic lupus drug in more than half a century. (See BioWorld Today, March 11, 2011.)

The FDA showed a different kind of flexibility with Bristol-Myers Squibbs Co.'s Yervoy (ipilimumab), which was approved to treat melanoma despite a risk of serious side effects. The first in a class of immune-boosting drugs, Yervoy "was the first drug approved by the FDA that was clearly demonstrated to prolong the life of patients with late-stage melanoma," according to the report. (See BioWorld Today, March 28, 2011.)

Although it was granted priority review, Yervoy took nine months to get through the review process as BMS had to submit a late major amendment. The biologic's approval was based on a single, three-arm international trial that evaluated 628 patients. Because of the risks, the FDA required a risk evaluation and mitigation strategy.

The other new biologic approved in fiscal 2011, BMS' Nulojix (belatacept), was granted fast-track designation, but it took nearly two years and two review cycles to get the FDA's blessing. Approved to prevent rejection of transplanted kidneys, Nulojix represents the first new class of drugs for rejection of organ transplants in more than a decade, the FDA said.

Going Public Could Get a Lot Easier

With only one dissenting vote, the House approved a bill last week that would allow more small biotechs to go public without having to register with the SEC.

The Small Company Capital Formation Act, H.R. 1070, would reform the SEC's Regulation A, which currently requires companies to register with the SEC if they issue more than $5 million in shares in a public offering. The bill would raise that cap to $50 million.

The legislation has been sent to the Senate, where a companion bill, S. 1544, was introduced in September.

If passed, the act would "help ease the burden on cash-strapped small biotechnology companies short on resources by streamlining requirements and supporting their efforts in raising much-needed capital," Jim Greenwood, president and CEO of the Biotechnology Industry Organization, said in a press release.

"Raising the exemption to $50 million would give struggling companies another avenue to raise capital through direct public offerings without a lengthy paperwork process," he added.

Ethics of MCM Testing in Children Questioned

A proposal to test anthrax vaccines in children is putting the Department of Health and Human Services (HHS) between the proverbial rock and a hard place.

On the one hand, the National Biodefense Science Board recently recommended that HHS proceed with an anthrax vaccine trial in children – if ethical issues can be worked out.

That recommendation came on the heels of a report from the Bipartisan WMD Terrorism Research Center, which gave the nation a D last month for its medical countermeasure (MCM) development and production process. One of the concerns the research center voiced was the lack of provisions for developing MCMs for children and pregnant women. (See BioWorld Today, Oct. 17, 2011.)

On the other hand, groups like Public Citizen are urging HHS to ignore the science board's advice. Such trials "would be unethical," the group said in a letter last week to HHS Secretary Kathleen Sebelius, "because the research does not present any prospect of direct benefit to the children who would be the subjects of the research, and the vaccine poses significant known risks of potentially serious harm."

The letter questioned whether truly informed parents would allow their children to participate in such a trial.

"Anthrax currently is not 'a serious health problem affecting the health or welfare of children' in the U.S., and the extremely remote chance of children being exposed to anthrax is not sufficient justification for testing the anthrax vaccine in children, particularly since there are antibiotics approved by the FDA for use in children to treat post-exposure cutaneous or inhalation anthrax," the advocacy group said.