1) Degarelix – Ferring Pharmaceuticals – USA & Europe

2) Rilonacept – Regeneron Pharmaceuticals – USA

Additional NDA (1) and PMA (1) approvals are expected in 2009 and an NDA will be submitted by Target Health in December 2009.

CDER New Molecular Entity (NME) and New Biologic License Application (BLA) Approvals for 2008

Drug Name  
FDA Appl. #






(NDA # 022187)





(BLA # 125249)



P, O 



(NDA # 021992)





(NDA # 022249)



P, O 



(NDA # 022161)





(BLA # 125160)






(NDA # 021964)





(NDA # 021775)






(NDA # 022212)





(NDA # 022090)





(NDA # 022156)





(NDA # 021894)



P, O 



(NDA # 125268)






(NDA # 022290)



P, O 



(NDA # 022206)





(NDA # 022253)






(NDA # 022030)





(NDA # 021911)





(NDA # 022291)



P, O 



(NDA # 022304)





(NDA # 022244)






(NDA # 022311)



P, O 



(NDA # 021711)





(NDA # 022201)





(NDA # 022304)





Associated Press

Steve Jobs, Apple Inc.’s CEO, explained his noticeably thinner appearance in recent weeks as the result of a hormone imbalance.

WebMD.com, January 6, 2009 — Steve Jobs today posted a letter on the Apple web site stating that he has a “hormonal imbalance” that caused him to lose weight throughout 2008, and that he’s being treated and staying on the job as Apple’s CEO.

In 2004, Jobs had surgery to remove a pancreatic tumor, which he called a very rare form of pancreatic cancer called islet cell neuroendocrine tumor.

But in today’s statement, Jobs doesn’t mention cancer or his 2004 cancer treatment. Instead, Jobs says he has a “hormonal imbalance that has been ‘robbing’ me of the proteins that my body needs to be healthy.”

Jobs adds that “the remedy for this nutritional problem is relatively simple and straightforward, and I’ve already begun treatment,” and that it will take him until spring to regain the weight.

What is a hormonal imbalance and what might it mean about Jobs’ health? Here’s what four doctors — who aren’t treating Jobs — told WebMD today.

All four doctors said there isn’t a lot of medical information in the statement — for instance, Jobs doesn’t say what hormone or hormones are out of balance. But they read between the lines and offered some perspective.

What are hormones?

“Basically, the definition of a hormone is very simple. It’s a chemical your body makes in one place that works in another. It has to be something your body makes and has to be made in one cell and work in a different cell,” says Robert Lustig, MD, an endocrinologist at the University of California, San Francisco. Lustig adds that the body makes hundreds of hormones.

The pancreas — which is where Jobs had his 2004 tumor — makes hormones including insulin, which regulates blood sugar, and also releases enzymes into the digestive system, says Bernard A. Roos, MD, director of the Geriatric Research Education and Clinical Center at the Miami VA and the University of Miami Miller School of Medicine.

What is a hormonal imbalance?

“The imbalance is a manifestation of something wrong with a gland. But then you have to figure out what’s wrong with the gland, and unfortunately we can’t tell from [Jobs’ statement],” Lustig says.

Could Jobs’ previous pancreatic tumor be related to his hormonal imbalance?

Maybe, but not necessarily.

“Certainly, the fact that he had a pancreatic neuroendocrine tumor back in 2004 puts him at higher risk for another endocrine tumor, of the pancreas or elsewhere. That’s true, but that doesn’t mean what’s going on here, because then it would not be a ‘nutritional problem’ and certainly would not have a simple treatment,” says Lustig.

Could Jobs’ hormonal imbalance be related to his 2004 surgery to remove the pancreatic tumor?

It’s possible. But exactly what that surgery involved — such as how much of the pancreas was removed — hasn’t been made public; all Jobs has said about it in the past is that he was “fine” after the operation.

“The more of the pancreas that’s taken out in surgery, the more likelihood of developing a hormonal problem,” says Jay Marks, MD, an associate clinical professor of medicine at UCLA who contributes to MedicineNet, which is part of the WebMD network.

Because of his previous pancreatic cancer and treatment, Jobs may be low on pancreatic enzymes, and fixing that would mean taking pancreatic enzyme supplements with meals. And if he doesn’t do that or is on a dose that’s too low, that could hinder his digestion and lead to weight loss, explains Otis Brawley, MD, chief medical officer at the American Cancer Society.

What else could be causing Jobs’ hormonal imbalance?

He could have a thyroid problem, note Brawley and Roos. Roos also says it’s possible that Jobs has low levels of growth hormone due to a chronic illness. “There are three possibilities: He has growth hormone deficiency, he has pancreatic enzyme deficiency, or he has both,” says Roos.

But that’s speculation.

“I definitely encourage folks to take him at his word and realize that the weight loss could be due to his disease progressing, but it’s also very, very likely due to some nonmalignant process,” says Brawley.


Robert Lustig, MD, professor of clinical pediatrics, division of endocrinology, University of California, San Francisco.

Bernard A. Roos, MD, director, Geriatric Research, Education, and Clinical Center, Miami VA and University of Miami Miller School of Medicine.

Jay Marks, MD, associate clinical professor of medicine, University of California at Los Angeles.

Otis Brawley, MD, chief medical officer, American Cancer Society.


The prognosis for Apple’s Steve Jobs: continuing conjecture

The cancer survivor tells worried fans and investors that a hormone imbalance has caused his weight loss.

By Dawn C. Chmielewski and Jessica Guynn

January 6, 2009

Reporting from San Francisco and Los Angeles — Apple Inc. Chief Executive Steve Jobs broke with his usual code of secrecy Monday to explain his health problems, but the disclosure that a hormone imbalance was causing his noticeable weight loss will probably do little to tamp down concerns.

Medical experts said a hormone imbalance in a pancreatic cancer survivor raises red flags about a possible recurrence. Jobs said in 2004 that he had undergone surgery to treat a rare form of the deadly disease.

Although Jobs is known as one of the nation’s most intensely private corporate leaders, he issued an open letter Monday in which he tried to assure Apple investors and customers that he was healthy enough to lead the pioneering technology company he co-founded.

The 53-year-old CEO said his doctors discovered his condition had been “robbing” his body of proteins needed for good nutrition. He is undergoing treatment, which he described as simple and straightforward.

He did not mention cancer in his letter.

“I’ve said more than I wanted to say, and all that I am going to say, about this,” Jobs wrote.

Several medical experts, who had no access to Jobs’ health records, said problems other than cancer could have caused a hormone imbalance. For example, the surgery to remove his tumor could have left Jobs with a pancreas too small to produce the necessary enzymes.

Yet hormone imbalances are common in people who have an active neuroendocrine tumor, not in people who have been cured of the cancer, said Dr. Selwyn M. Vickers, chairman of the surgery department at the University of Minnesota.

An Apple spokesman declined to comment. The board said it would give Jobs its “complete and unwavering support” while he recovered.

Jobs noted that he spent this holiday season in an atypical way: with his family, rather than preparing for today’s keynote address at the annual Macworld Conference & Expo in San Francisco. He’ll miss delivering the speech for the first time since 1997. In years past, he used it to announce such industry-changing products as the feature-rich iPhone and iTunes music software.

The CEO has been inextricably linked to his company’s fortunes since he helped revive the Mac computer and spurred a digital renaissance with the iPod media player and the iPhone. Jobs didn’t do it alone, but continued anxiety over his health suggests the company has yet to persuade investors or Macolytes that Apple could survive without him.

“Why is everybody obsessed with Steve? I think the answer is very simple,” said Paul Saffo, a technology forecaster who teaches at Stanford University. “Apple has not yet convinced the world that Apple without Steve will continue with the same powerful vision.”

Last month, the Cupertino, Calif., company announced that Jobs would not give the Macworld presentation, triggering speculation that he was gravely ill. At the time, Apple, which is pulling out of the trade show after this year, said his participation “doesn’t make sense.”

In his letter, Jobs made light of the “stories of me on my deathbed” and said he would remain CEO while he recuperated. He said he expected to regain his lost weight by late spring.

“I will be the first one to step up and tell our Board of Directors if I can no longer continue to fulfill my duties as Apple’s CEO,” Jobs wrote. “I hope the Apple community will support me in my recovery and know that I will always put what is best for Apple first.”

Jobs began looking unusually thin and drawn in June, when he introduced the latest generation of iPhones. Investors called for him to come clean about his health.

In the absence of any reliable information from the company, the blogosphere filled in the blanks with wildly speculative reports that whipsawed the company’s stock. Bloomberg, the financial news service, mistakenly published Jobs’ obituary in August. And a false account on a CNN website claiming Jobs had suffered a heart attack caused Apple stock to plunge.

As recently as last week, the popular technology blog Gizmodo cited a single source who said Jobs’ health was “rapidly declining.” The account knocked Apple share prices down for part of a day.

Internet paparazzi have staked out Jobs’ known Palo Alto haunts and broadcast information about his appearance. Self-described geek blogger Robert Scoble caused a stir last week, when he recounted his visit to a Palo Alto yogurt shop that Jobs patronizes. “They said he was in a couple of days ago and is in great health,” Scoble wrote.

Patrick McGurn, special counsel at shareholder advisory firm RiskMetrics Group, faulted Apple for responding only when it was “clear there would be a feeding frenzy” at the trade show that would overshadow product news.

“They can argue that they didn’t have a final diagnosis to point to and a treatment regimen in place, and I can buy that to a degree,” McGurn said. “But the fact of the matter is that there was a prior history here. This is not the first time Apple has gone through this drill. It should have just bitten the bullet and announced it would provide regular updates on Steve Jobs’ health because the company knows the issue is important to investors.”

Wall Street heaved a sigh of relief Monday. Apple’s shares gained $3.83 to $94.58.

“Lack of information is the worst thing for investors,” said Michael Obuchowski, chief investment officer of First Empire Asset Management Inc., which holds Apple stock. “This made Apple investors very happy.”

Adam Harter, an analyst at Financial Enhancement Group, which owns more than 30,000 shares, reacted more cautiously.

“I am not a doctor, but it certainly doesn’t give you the comfort that nothing is wrong with him.”




Times staff writer Shari Roan contributed to this report.

© AP Photo

Electronic Medical Records

Electronic medical records promise to end costly and inefficient paper shuffling at the doctor’s office. The best programs securely put medical health information online, including data like lab test results and prescriptions. They also allow patients to get reminders to schedule appointments with a physician. While the technology has already gone mainstream with Microsoft and Google, versions of EMR programs, it’s not yet in widespread use.

“We are developing what I call digital penicillin”

Forbes.com, by Rebecca Ruiz — It’s not just automakers, finance firms and state coffers that stand to gain from government loans and stimulus spending. If President-elect Obama is able to pass a stimulus package early next year, rumored to cost $850 billion, it could be a boon for the health care industry as well.

In addition to funding an expansion of state Medicaid and the State Children’s Health Insurance Program, the Washington Post has reported there are plans to allocate upward of $10 billion to implement a nationwide, interoperable electronic medical records (EMR) system. The investment could jump-start a flurry of spending on health information technology (IT).

If well funded and adopted widely, many different technologies–from electronic records to algorithms to remote monitoring devices–promise to streamline the health care system, saving money and improving services.

“I think we’ll look back and see the real reflection of change,” says Paul Keckley, executive director of the Deloitte Center for Health Solutions, of these technologies’ potential. “We will not be having the discussion about electronic medical records. [That] will be standard.”

The end result, he says, is a future in which health care is more personalized–and frequently delivered outside of the doctor’s office and hospital.

A Costly Proposition
This sounds ideal, but it’s an expensive proposition. Campaign for America’s Future, a liberal think tank based in Washington, D.C., expects a nationwide electronic medical records system to cost $7.6 billion a year over 15 years.

The high cost is due partly to the fact that few providers currently use health IT. By 2006, only 12% of America’s physicians and 11% of its hospitals had adopted computer applications to help modernize their record keeping, according to the Congressional Budget Office.

But there’s a potential return on the high cost. Last week, the CBO released a study estimating that the implementation of health information technology, including EMRs, could save $7 billion in the first five years.

Cost and savings aside, EMRs have the ability to radically change the way patients interact with health professionals.

Dr. Brian Klepper, principal of Health 2.0 Advisors in San Francisco, says that patients have the ability to fully engage in their health care through an EMR. Klepper also owns WeCare TLC, a small business that provides employers with on-site health care clinics and access to EMRs. The bare-bones interface allows a patient to schedule a visit, get reminders about annual check-ups and special exams or review lab test results–explanation of medical terminology included.

A spokesman for the company says that its clients save 15% to 25% of their annual health care costs by paying a flat yearly fee for clinic services. It currently serves 9,000 individuals and operates four clinics in Florida and Georgia.

By scale, WeCare TLC is a tiny player in an industry that includes Google and Microsoft, both of which have launched their own EMR programs. In fact, the breadth of the market raises the question about how patients will have access to uniform and interoperable EMRs–a question that will likely be resolved through financial incentives to health providers to adopt health IT.

Such incentives, Klepper says, must be paired with new technologies in order to see real savings. Otherwise, the health care system will continue to reward service-based treatment instead of superior performance. Given new technological innovations available in certain markets, the level of performance could reach far greater heights.

Technological Wonders
Algorithms, for example, harness advanced computational power to give physicians information about how best to treat each individual patient.

Predictive modeling, a type of algorithm, can draw on billions of specific health indicators and outcomes culled from clinical and claims data while also taking into consideration an individual patient’s health status. Using the rules of probability, a computer can weigh the data against a patient’s particular needs and determine which treatment option is most likely to work.

Another algorithm can take a set of rules for how to treat a disease or condition and translate them into an if-then formula. Such algorithms are derived by a peer-reviewed process in which an expert on a particular health concern–diabetes, for example–develops a comprehensive list of treatment options. Using a computer or handheld device, the physician can then use the algorithm to get a treatment plan based on best practices and the patient’s unique needs.

Unfortunately, says Klepper, doctor offices are reluctant to invest in these devices, which cost about $1 per patient, since the technology doesn’t generate income for the physician. But there are other tools that are more accessible to consumers.

Keckley, of the Deloitte Center for Health Solutions, points to Medtronic and Intel, two companies that create remote monitoring devices designed to cut down on trips to the doctor’s office and hospital. Some of Medtronic’s cardiac devices, for example, can send data about a patient’s vital signs via the Internet to his or her EMR, which is then accessed by a physician–thus saving a trip to the doctor’s office.

Intel’s HealthGuide, a small box-shaped device for at-home use, is equipped with a video camera for two-way calls between you and your doctor. It also can be connected to devices like blood-pressure monitors and glucose meters; the results are sent to your physician automatically.

Diagnostic tools like these can make a substantial difference in providing high-quality health care, but some companies are also developing technology to lessen administrative demands, which can monopolize a considerable amount of a physician’s time.

Dr. Louis Cornacchia, a physician and president of the company Doctations, has created a suite of Web applications that attempt to seamlessly integrate health records, dictation and transcription services, billing information and communication between doctors and patients. To Cornacchia, tech-savvy health care can certainly save money and time. But more important, it might save lives.

“We are developing what I call digital penicillin,” he says.


© AP Photo


E-prescribing has already been rolled out in some clinical settings–and with great success. The ability to send prescriptions electronically to a pharmacy increases efficiency, but with systems that allow doctors to select lower-cost or generic drugs, the savings can be considerable. For example, when that type of e-prescribing was used recently in a study at two hospitals in Boston, researchers found that it had the potential to save $845,000 per 100,000 patients.


© Getty Images

Remote Device Monitoring

Most people would skip a trip to the doctor’s office if they had a choice. New technology might help facilitate this by wirelessly transmitting vital signs of patients with chronic diseases and certain implantable devices. Medtronic is on the cutting edge of such innovations. The company produces cardiac devices that transmit data via the Internet to a patient’s electronic health record. Some of these automatically alert a clinician when the patient experiences a sudden or dangerous change to his or her condition.


Courtesy Intel

In-Home Devices

An in-home device connects you wirelessly with a physician or health care professional. Intel has pioneered such technology with HealthGuide, a small box-shaped device that is equipped with a video camera for two-way calls between you and your doctor. It can also be connected to devices like blood-pressure monitors and glucose meters. Those results are made available to your physician. To learn about pricing and purchasing, visit Intel.


© iStock

Algorithm-Driven Health Care

Ensuring physicians adhere to the same best practices is a near-impossible task. But some believe it can be done using algorithms. With this model, an expert on a particular health topic lays out a set of rules for how to treat related diseases, which are then judged by a panel of his or her peers. Algorithms are then created to help physicians determine the best treatment using if-then formulas. Companies working on versions of this technology include mTuitive and Oncology Metrics.


© iStock

Predictive Modeling

What if your physician could quickly come up with a personalized treatment plan based on probability? That might sound risky, but computers using advanced computational power to draw on billions of specific health indicators and outcomes have the potential to make it sound science. Predictive modeling, a type of algorithm, gives physicians access to clinical and claims data via a computer that also takes into consideration the patient’s health status and needs. With this information, physicians can calculate the probability of which treatment will work best for you.



Social Networking

Trying to communicate effectively with a health care provider can be an exhausting process. But some researchers are looking at how interactions between similar patients belonging to the same social network can actually produce better outcomes and improve communication skills. Something similar already exists online with list serves and blogs, but Paul Keckley, executive director of the Deloitte Center for Health Solutions, sees potential for such groups to become a standard in care.

Asia Medical eNewsletter

Volume 9, Number 1 – January 2009

PacificBridgeMedical.com — In November 2008, Japan’s Ministry of Health, Labor and Welfare (MHLW) released its slated 2009 revisions to the Good Clinical Practice (GCP) standards governing clinical trials for medical devices in Japan. Almost identical revisions to GCP for drugs were implemented in April 2008.

Currently, clinical trials in Japan are extremely expensive, partly because Japan is a high-cost country and partly because of burdensome regulations. For example, the qualifications medical institutions must meet to conduct clinical trials are very high. While clinical trials throughout Asia usually cost less than they would in the US or Europe, in Japan they often cost more. The new device and drug GCP rules have two measures allowing more subcontracting in clinical trials. These measures may reduce clinical trial costs in Japan somewhat.

The first change is regarding Institutional Review Boards (IRBs). Previously, medical institutions conducting clinical trials were obligated to set up and maintain their own IRBs internally, which can be expensive. With the new changes, a medical institution’s director can designate an outside IRB to perform this task instead. This IRB must be part of a university, nonprofit company, or independent government agency, not a for-profit company. To preserve IRB transparency while making this change, the rules also add new reporting requirements. IRBs must make publicly available their internal procedures, members’ names, and summaries of meeting minutes.

The second change is regarding the physical handling of investigational products. Previously, the shipping of investigational devices and drugs (including confirmation of receipt and quality management) had to be performed directly by those running the clinical trial. Now, the trial sponsor can designate a third party to perform these tasks.

The new medical device GCP standards will take effect on April 1, 2009. However, the part adding new reporting requirements for IRBs will not take effect until April 1, 2010.

Indonesia Orders Foreign Drugmakers to Manufacture In-Country

In a surprise move, Indonesia’s Ministry of Health issued a new regulation in November 2008 designed to force foreign companies to manufacture drugs in Indonesia. The regulation states that foreign-owned companies will not be allowed to register imported drugs for sale unless they have a drug production facility in Indonesia.

This regulation is not being implemented yet; drugmakers are being given two years to comply. It would affect about 13 foreign drug companies whose Indonesian offices sell imported drugs but do not manufacture there, mostly large multinationals like Merck, Wyeth, and Sanofi-Aventis.

Indonesia has the third largest population in Asia (237 million people), and its drug market of nearly $500 million is growing quickly, making it an important destination for future growth. However, Indonesia is one of Asia’s poorer countries in terms of healthcare consumption. By comparison, India, with about four times the population, has a drug market of over $10 billion.

The affected drugmakers are weighing whether it would be easier to build pharmaceutical plants in Indonesia, or simply exit the country. It is harder for foreign companies to do business in Indonesia than in most other Asian countries, due to corruption, infrastructure deficits, and political uncertainty. This abrupt government action adds to that political uncertainty, impacting Indonesia’s attractiveness for drug companies of all sizes.

It is unclear if the government’s threat will actually be carried out. Despite protests by foreign drug companies in Indonesia as well as the US Chamber of Commerce, the Minister of Health, Siti Fadilah Supari, has remained adamant. The ministry also announced that if multinationals choose to leave Indonesia, the government will directly import drugs that become unavailable.

Diabetes Increasing Rapidly in China

In November 2008, the Chinese Diabetes Society (CDS) released the results of its study into the epidemiology of diabetes in China. The results suggested that diabetes is becoming prevalent in China more rapidly than previously estimated, increasing the market prospects for Western drugs and devices treating diabetes.

The CDS found that the prevalence of diabetes among Chinese between 20 and 70 years old is now 10.5%, or 9.5% for women and 12% for men. This is about double the prevalence in 2001, and four times the prevalence in 1994. The study estimated that there are about 40 million Chinese suffering from diabetes in urban areas, and 20 million in rural areas.

Diabetes is one of the chronic diseases that are becoming increasingly prevalent in China in response to changing lifestyles. As incomes rise with economic development, and people move from agriculture to more sedentary occupations, more and more Chinese are now overweight or obese, both risk factors for diabetes. Chinese people living in cities are much more likely to have diabetes. About 55% of the Chinese population is rural, but two-thirds of diabetes sufferers (as estimated by the study) were urban.

However, for diabetes treatment to improve in response to this rapid rise, popular awareness of the disease will need to increase. Many Chinese do not know they have diabetes. Also, maintenance practices like blood glucose self-monitoring are low in China compared to other Asian countries.

As part of the effort to increase awareness, Eli Lilly, Roche, and Becton Dickinson are collaboratively funding the China Diabetes Education Program, which has given comprehensive diabetes training to over 37,000 medical professionals and 170,000 patients. With longer experience of diabetes in their own countries, as well as higher R&D levels, western firms should have a competitive advantage in developing and marketing diabetes products in China.

The CDS is a member of the International Diabetes Federation. In 2007, it entered into a partnership with Eli Lilly and two European diabetes societies to fund and conduct collaborative European-Chinese diabetes research. Its current study took over a year to conduct and surveyed 40,000 people in 14 provinces.

India Proposes Quality Certification for Medical Devices

On December 15, 2008, India’s Minister of Science and Technology, Kapil Sibal, discussed coming medical device regulations at a conference in New Delhi. These regulations are to be submitted to India’s cabinet soon by the Department of Science and Technology, which oversees the medical device industry (though it does not register medical devices).

The outlined measures would require ISO certification of medical devices and their manufacturers before they could be sold in India. Currently, only ten specified types of high-risk medical devices (and a few smaller items) need registration in India before marketing. These new standards, likely to be ISO 13485, would apply to medical devices of all or most types. The Bureau of Indian Standards currently manages ISO certification in India through notified bodies, but overseas certifications may also be accepted.

The conference, “Optimizing Healthcare Delivery in India,” was on increasing the affordability of medical treatment to Indians, and the measures described by Mr. Sibal focused on this goal. He described the measures as designed to promote domestic Indian production of medical devices, creating more competition to imported devices and thereby lowering prices.

Other measures Mr. Sibal highlighted at the conference included increasing R&D in medical technology, genomics, and vaccines, as well as improving the supply and quality of Indian healthcare professionals, who are currently in high demand.