With fewer new marketed drugs than previous years, the pharmaceutical market in 2008 showed signs of greater regulation and cautiousness, but still unveiled significant breakthroughs according to University of the Sciences in Philadelphia’s Daniel A. Hussar, PhD. Dr. Hussar, Remington professor of pharmacy at the University’s Philadelphia College of Pharmacy, selected the most noteworthy new drugs and trends leading into the New Year.
“With just 16 drugs, 2008 continued the trend of a low number of new drugs being marketed,” said Dr. Hussar. “Last year, there were only 17, which was then the lowest number in years, whereas a typical number of new drugs going to market is between 20 to 30.”
Dr. Hussar attributes the decrease in numbers to greater precautionary measures by the Food and Drug Administration (FDA) and shifting goals of pharmaceutical companies. “The FDA is taking an even closer look at the studies that are supporting the applications of new drugs,” said Dr. Hussar. “Additionally, pharmaceutical companies are looking more at drugs that they hope will be blockbusters, and less attentively at drugs that might not compete that well.”
While it is typical to see a considerable number of drugs marketed to treat common conditions, there are also incentives for pharmaceutical companies to develop therapies for less-common ailments. “What we’re really seeing this year is a larger than usual number of drugs for rare disorders,” said Dr. Hussar. “Approximately one third of the new drugs that were marketed are used to provide treatment for rare disorders.”
To provide an unbiased and comparative analysis of the new drugs marketed each year, Dr. Hussar uses the New Drug Comparison Rating (NDCR), a one-to-five point scale system he created to succinctly rate a drug’s importance. A designation of “5” denotes the drug represents an important advance, while a designation of “1” signifies important disadvantages.
Six out of the 16 new drugs marketed earned a NDCR of three or lower, meaning they failed to introduce any significant advantages to patients or the pharmaceutical marketplace. With NDCR ratings of four or five, the following six drugs are the most significant of 2008.
Generic Name/Trade Name, Manufacturer, NDCR
Tetrabenazine/Xenazine®, Ovation Pharmaceuticals, 5
As the first drug to be approved for the treatment of chorea in Huntington’s disease, Xenazine® earns a NDCR of 5. Chorea is jerky, involuntary movement that occurs in people with Huntington’s disease, a rare, inherited neurological disorder affecting roughly 1 in 10,000 people in the United States. Xenazine® reversibly inhibits the human vesicular monoamine transporter type 2 (VMAT2), resulting in depletion of monoamine stores.
C1 Inhibitor (Human)/Cinryze®, ViroPharma, 5
Cinryze® is administered intravenously for routine prophylaxis against angioedema attacks in adolescent and adult patients with hereditary angioedema (HAE), a condition caused by C1 inhibitor deficiency. While Cinryze® is not the only drug used for the prevention of HAE attacks, other treatments have been of limited effectiveness and have been associated with the occurrence of serious adverse events. Cinryze® is manufactured using a sequence of steps to reduce the risk of viral transmission, and possesses a unique mechanism of action and is less likely to cause serious adverse events.
Etravirine/Intelence®, Tibotec, 4
Intelence® is administered in combination with other antiretroviral agents for the treatment of HIV-1 infection in treatment-experienced adult patients who have evidence of viral replication and HIV-1 strains resistant to a non-nucleoside reverse transcriptase inhibitor (NNRTI), and other retroviral agents. Intelence® is the fourth antiretroviral agent to be classified as a NNRTI, however, it is effective in some patients with HIV-1 strains that are resistant to other NNRTIs. While the drug is administered more frequently than a comparable treatment, and is not indicated for pediatric use, Intelence®0 is not likely to cause central nervous system, psychiatric, and/or hepatic adverse events and is less likely to interact with other drugs.
Methylnaltrexone bromide/Relistor®1, Wyeth, 4
Opioid anagesics, such as morphine, are often used on a continuous basis to relieve pain in patients with incurable cancer or other advanced illnesses, and who are receiving palliative care. Relistor®2 is administered subcutaneously for the treatment of opioid-induced constipation and is often effective in patients whose response to the use of a bowel regimen (e.g., laxatives, stool softeners) has not been sufficient. Usually administered every other day as needed, the drug has a unique mechanism of action for the treatment of opioid-induced constipation and serves as an effective alternative to a bowel regimen.
Eltrombopag olamine/Promacta®3, GlaxoSmithKline, 4
Romiplostim/Nplate®4, Amgen, 5
Both Promacta®5 and Nplate®6 are used for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins, or splenectomy. Chronic ITP is a rare autoimmune disorder characterized by low platelet counts, which may result in serious bleeding events. Earning a NDCR of 5, Nplate®7 was approved as the the first treatment that directly stimulates the production of needed platelets. Promacta®8, which also increases platelet production, was approved as the second drug for the treatment of thrombocytopenia in patients with chronic ITP, following Nplate®9 by several months. Both drugs are the first of their kind.
Several orphan drugs, including Cinryze®0, Xenazine®1, Nplate®2, and Promacta®3, to treat rare conditions affecting less than 200,000 people were introduced this year. “If a drug is designated by the FDA as an orphan drug, the company that develops it is granted a seven-year period in which they have exclusive marketing rights, so that provides an incentive for some companies to develop those,” explained Dr. Hussar. He further noted that these specialized drugs are usually very expensive.
A new book written by Dr. Hussar, titled NDCR 2009 New Drugs: 2002-2008: New Drug Comparison Ratings, is now available. The book details the 158 drugs that have been marketed in the 2002-2008 period, including comparisons to previously marketed drugs, specific advantages and disadvantages of each, and ratings for each new drug based on comparisons with related agents. To order this valuable reference, please visit www.newdrugsndcr.com
In March 2009, Dr. Hussar will present New Drugs of 2008 at the 30th Annual Symposium on Advances in Pharmacy Practice, a continuing pharmacy educational program presented by the Philadelphia College of Pharmacy at University of the Sciences. Developed for pharmacists, the symposium provides the most up-to-date continuing education on newly approved drugs and updates in pharmacy law presented by the foremost authorities in the field. For more information, or to register, please visit http://www.usp.edu/AdvancesCE/