Drug Approvals Reach 15-Year High on Smoother FDA Reviews

By Anna Edney & Catherine Larkin

Blood smear of the final blast crisis phase of chronic myelogenous lleukemia. Photograph: CDC

U.S. drug approvals in 2012 reached their highest level in 15 years, led by 11 new cancer therapies, including Ariad Pharmaceuticals Inc. (ARIA)’s Iclusig and Pfizer Inc.’s Bosulif, both aimed at forms of leukemia.

The Food and Drug Administration cleared 39 novel medicines in 2012, nine more than a year earlier, agency data show. Among the non-cancer products were Arena Pharmaceuticals Inc. (ARNA)’s Belviq, the first new prescription weight-loss pill in 13 years, and Johnson & Johnson (JNJ)’s Sirturo, aimed at patients with tuberculosis who are resistant to multiple-drug therapies.

Approvals of innovative drugs have averaged about 23 a year over a decade. The FDA tightened its safety standards after the high-profile 2004 failure of Merck & Co.’s painkiller Vioxx and other drugs. Since then, companies have included more safety studies as part of their development strategies, and shifted their focus to specialty drugs with higher chances of approval because they target the patients they’re most likely to work on.

“The sheer number I think supports the correctness in some of the strategy shift of the pharmaceutical companies over the last number of years,” Rick Edmunds, a senior partner at Booz & Co. who leads the consulting firm’s global health-care practice in Washington, said in a telephone interview. The rate “implies pharma growth potential to 2015 and beyond.”

The FDA signed off on 53 novel drugs in 1996, the most since it began sharing aggregate data, and 39 in 1997. There were 36 approvals in 2004, the year the agency began counting therapeutic biologic medicines, made from living organisms.

Cancer Drugs

Oncology drugs were high on the FDA’s approval list last year and will likely remain a good area for investors in 2013 as the FDA division that reviews those treatments seems more willing to clear new medicines, Ira Loss, an analyst at Washington Analysis LLC, said in a telephone interview.

The agency approved three drugs to treat a rare blood and bone marrow disease — chronic myeloid leukemia — including Cambridge, Massachusetts-based Ariad’s Iclusig, three months ahead of schedule. Roche Holding AG (ROG)’s Perjeta also won approval to work with the Basel, Switzerland-based company’s Herceptin and chemotherapy in patients with a gene mutation associated with aggressive breast cancer.

Diabetes drugs still face hurdles, Loss said.

AstraZeneca Plc (AZN) and Bristol-Myers Squibb Co. (BMY) as well as Johnson & Johnson are attempting to bring diabetes treatments to market that excretes excess blood sugar into patients’ urine. London-based AstraZeneca and Bristol-Myers have been working for a year to answer FDA questions on dapagliflozin. The FDA is scheduled to decide on J&J’s canagliflozin by the end of March.

J&J’s Sirturo

The FDA also is weighing the risks and benefits of Novo Nordisk A/S (NOVOB)’s once-a-day insulin Tresiba after advisers to the agency determined in November the Bagsvaerd, Denmark-based company provided sufficient efficacy and safety data to support marketing of the drug. They also said Novo should conduct a cardiovascular safety trial because the drug may have higher heart risks than other diabetes treatments.

J&J’s tablet Sirturo was one of two FDA approvals made on the final day of 2012. The drug is the first new way in 40 years to treat the contagious lung infection and the first therapy specifically for patients with the disease who are resistant to multiple drugs.

Salix Pharmaceuticals Ltd. (SLXP), a maker of gastrointestinal drugs, also won clearance on Dec. 31 to sell Fulyzaq, the first approved medicine to treat HIV-associated diarrhea. On Dec. 28, Pfizer and Bristol-Myers’s blood thinner Eliquis was approved after more than a year of review, giving the two New York-based companies a new potential blockbuster heart drug.

User Fees

“The strong number of approvals demonstrates the continuing innovation by biopharmaceutical research companies and commitment to help improve patients’ lives,” said Matt Bennett, a spokesman in Washington for the Pharmaceutical Research and Manufacturers of America, or PhRMA.

The FDA also credited congressional passage of a new five- year agreement that raises the so-called user fees drugmakers pay the agency for safety and efficacy reviews.

The Prescription Drug User Fee Act “has provided critical resources for improving the quality and timeliness of premarket review of drugs,” Sandy Walsh, an FDA spokeswoman, said in an e-mail. “These accomplishments could not have been achieved without the innovations of the biopharmaceutical industry and the dedication and skill of FDA’s drug review staff.”

‘Breakthrough’ Therapies

The FDA may be able to speed the approval of some “breakthrough” drugs under a new proposal that was part of legislation that became law in July. Pharmaceutical companies can request the agency designate their experimental treatments for serious or life-threatening diseases as breakthrough therapies, which affords them advice and guidance from FDA staff to ensure development is on the right track.

The FDA received seven breakthrough requests, of which staff granted two, denied one and four were still pending, John Jenkins, director of the FDA’s Office of New Drugs, said at a conference Dec. 10. No drugmaker has revealed its breakthrough status.

On the other hand, the FDA has 60 additional days to review drugs as part of the legislation, a provision that took effect in October. Ten and six month reviews are now 12 and eight months in an effort to make drug decisions based on better planning and communication that may eliminate the need for additional questions that reset the review clock, Jenkins said.

To contact the reporters on this story: Anna Edney in Washington at aedney@bloomberg.net; Catherine Larkin in Indianapolis at clarkin4@bloomberg.net

To contact the editor responsible for this story: Reg Gale at rgale5@bloomberg.net

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