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NPS Pharmaceuticals' Natpara Receives Orphan Drug Designation in Europe

NPS Pharmaceuticalas

NPS Pharmaceuticals, Inc., a biopharmaceutical company focused on treatments for rare diseases, recently announced that the European Commission (EC) has granted its Natpara (recombinant human parathyroid hormone (rhPTH[1-84]) orphan drug designation.

The company’s Natpara received orphan drug designation in Europe for treatment of hypoparathyroidism, a rare endocrine disorder in which the body does not produce sufficient levels of parathyroid hormone (PTH). Natpara is a bioengineered replacement for endogenous PTH, a principal regulatory of the body’s mineral homeostasis.

A product is granted orphan drug designation in the European Union (EU) if the product is designed for the diagnosis, prevention or treatment of rare diseases that are life-threatening or chronically debilitating. A rare disease in the EU is defined as affecting no more than five to ten thousand people.

“We are very pleased that Natpara has received orphan drug status in the EU,” Francois Nader, MD, president and chief executive officer of NPS Pharmaceuticals, stated. “As a global rare disease biopharmaceutical company, this aligns with our commitment to deliver innovative therapies that transform the lives of patients around the world. Hypoparathyroidism patients face a significant burden of a disease given the multitude of physical, cognitive, and emotional symptoms associated with this disorder. Natpara could be the first PTH replacement therapy to treat this condition.”

Insufficient levels of PTH lead to low serum calcium, high serum phosphate, increased urinary calcium excretion, and decreased urinary phosphorus excretion. Additionally, PTH deficiency can disrupt skeletal homeostasis, causing bone abnormalities. Patients with insufficient levels of PTH are also unable to convert native vitamin D into its active state to properly absorb dietary calcium.

Patients with hypoparathyroidism experience acute symptoms largely due to hypocalcemia. These include fatigue, muscle spasms and cramps, tingling, tetany, seizures, brain fog/mental lethargy, anxiety and depression.

The US Food and Drug Administration (FDA) granted the drug orphan drug designation in 2007. In October 2013, the company submitted its US Biologic License Application (BLA) to the FDA.

Source: NPS Pharmaceuticals, Inc.

IDF Commends Washington for Screening Newborns for Deadly Immune Disease


The state of Washington has added Severe Combined Immune Deficiency (SCID) to the list of conditions that all newborns in the state are screened for at birth.

The Immune Deficiency Foundation (IDF) applauded the state of Washington for this decision, effective as of January 1, 2014. SCID is a treatable but serious primary immunodeficiency disease, in which babies fail to develop a functioning immune system. SCID is commonly known as the “bubble boy disease” after a boy who lived in a germ-free bubble for 12 years. Generally, children with SCID are born without T-cells, specialized white blood cells made in bone marrow to fight infections, leading to extreme susceptibility to illness. Although babies born with SCID appear healthy at birth, lack of treatment will cause most children with SCID to die of infections before two years of age.

Typically, treatment for SCID is bone marrow transplantation. If treated within the first months of life, babies have more than a 90 percent survival rate. Screening for SCID is important due to the success of treatment when diagnosed early. In the US, over 50 percent of babies born are being screened for SCID and dozens of infants have been diagnosed in states that already perform screening for this disease, allowing these babies with a chance of a normal, healthy life.

“We are so happy that babies in Washington will be screened for SCID,” Marcia Boyle, President and Founder of IDF, stated. “We must continue to work to ensure that newborn screening for SCID is established in all 50 states so that all babies, no matter where they are born, have the chance at a healthy life.”

Screening for SCID in newborns is done by using the same dried blood spots currently collected from all babies for screening of a variety of inborn conditions. Kathleen Sebelius, Secretary of Health and Human Services recommended in 2010 that SCID be added to the uniform newborn screening panels in all 50 states and territories.

Source: Immune Deficiency Foundation

Last updated: 1/3/13; 1:50PM EST

AstraZeneca Obtains CDK9 Cancer Drug Portfolio from Probiodrug


AstraZeneca bought a portfolio of new cancer therapy from German Biotech Probiodrug for an undisclosed sum.

Today, Probiodrug AG announced the transfer of its experimental cyclin-dependent kinase 9 (CDK9) inhibitor program to AstraZeneca. The transaction will provide AstraZeneca with Probiodrug’s lead molecule and back-up compounds with the associated intellectual property. CDK9 has been implicated in the transcriptional regulation of genes involved in proliferation and inflammation. It is a promising target for treatments of both cancer and inflammatory diseases.

The transaction will allow Probiodrug to focus on development for treatments in Alzheimer’s disease. Probiodrug’s core capabilities are based on its long-standing expertise in the elucidation of the structure and function of enzymes which play a central role in the maturation of hormones. Probiodrug’s PQ912 is currently being evaluated in an extended Phase 1 clinical trial for the treatment of patients with Alzheimer’s disease.

“AstraZeneca is an excellent party to take over our promising CDK9 inhibitor program,” said Hendrik Liebers, chief financial officer of Probiodrug. “This transaction is consistent with our strategy to focus our resources exclusively on Alzheimer’s disease.”

Cyclin-dependent kinases (CDKs) are critical regulators of cell cycle progression and RNA transcription. A number of various genetic events cause over-activity of the cell cycle CDKs in cancer. Inhibition of CDKs can lead to both cell cycle arrest and cell death (apoptosis). Until now, the bulk of investigators’ attention have been in CDK 4/6 therapies at Pfizer and Novartis.

“We are particularly interested in the development of targeted therapies for oncology, such as CDK9. We believe that Probiodrug’s CDK9 inhibitor program, while early stage, shows potential and we look forward to continuing its development,” said Susan Galbraith, vice president and head of the Oncology iMed unit at AstraZeneca.

Source: Probiodrug AG

Last updated: 1/3/14; 12:15PM EST

Amgen and UCB's Osteoporosis Drug Shows Promising Results in its Phase II Trial

UCB Amgen

According to results from a mid-stage study, a new medication for osteoporosis stimulates the body to rebuild bone and could potentially strengthen the skeleton against fractures.

Amgen and UCB recently announced results from a Phase II trial evaluating romosozumab (AMG785/CDP7851) in postmenopausal women with low bone mineral density (BMD), which was published in the New England Journal of Medicine (NEJM).

In the study, treatment with romosozumab for 12-months demonstrated significant increases in BMD at the lumbar spine, total hip and femoral neck compared with alternative treatments. Additionally, significant increases in BMD were observed at three months, which was the first BMD assessment. The study compared romosozumab with Fosamax (alendronate sodium) and Forteo/Forsteo (teriparatide), currently available treatments. According to results, romosozumab is one-and-a-half to three times more effective than current osteoporosis drugs in rebuilding BDM at the lumbar spine.

“Results of the study demonstrate significantly increased BMD and stimulation of bone formation with romosozumab treatment in women with postmenopausal osteoporosis,” Michael McClung, MD, director of the Oregon Osteoporosis Center and lead study investigator, stated. “Additionally, romosozumab treatment resulted in greater increase in bone mineral density than those seen with both placebo and the active comparators. These data provide important insight into this medicine being developed for women with postmenopausal osteoporosis at high risk for fractures.”

At month 12, treatment with romosozumab achieved a mean increase of 11.3 percent at the lumbar spine compared to increases of 4.1 percent and 7.1 percent at the same region achieved with Fosamax and Forteo, respectively. At the total hip, treatment with romosozumab increased BMD 4.1 percent compared to increases of 1.9 percent with Fosamax and 1.3 percent with Forteo.

The investigational drug is in Phase III clinical development for postmenopausal women with osteoporosis. The drug is not approved by any regulatory authority to date.

Osteoporosis is common in women after menopause since their ability to form new bone cannot counter balance the rate at which bone is being removed. Over time, bone loss leads to weakened bones, increasing the potential for a break. According to the National Osteoporosis Foundation, roughly half of women over the age of 50 will experience an osteoporosis-related fracture and only 24 percent of these women receive treatment during the following year.

“Broken bones due to osteoporosis are common and can have a significant impact on the patient, her family and the healthcare system, yet the seriousness of this health event remains underappreciated, with only two-in-10 women receiving follow-up testing or treatment after they have broken a bone,” Sean E. Harper, MD, executive vice president of Research and Development at Amgen, stated. “With its bone-forming ability, romosozumab may result in new treatment strategies to help manage this disease.”

Romosozumab is designed to inhibit the protein sclerostin in order to increase bone formation and decrease bone breakdown. The drug’s adverse events were similar with those associated with Fosamax and Forteo, however patients receiving romosozumab experienced mild, generally non-recurring injection site reactions more frequently. No adverse reactions led to discontinuation of the treatment or study withdrawal.

“There remains a significant need for additional treatment options that form new bone. Romosozumab is designed to stimulate bone formation, which makes it different from most available treatments that reduce bone resorption,” said Prof. Dr. Iris Loew-Friedrich, chief medical officer, UCB. “We are encouraged by the emerging efficacy and safety profile, and look forward to further investigating its potential in the ongoing global Phase 3 clinical program.”

Sources: Amgen

Michael McClung, M.D., founding director, Oregon Osteoporosis Center, Portland, Ore.; Robert Recker, M.D., president, National Osteoporosis Foundation and director, Osteoporosis Research Center, Creighton University, Omaha, Neb.; Jan. 1, 2014, New England Journal of Medicine, online

Last updated: 1/3/14; 11:35AM EST

Specialty Drugs Account for More than Half of Approved Drugs in 2013


In 2013, the US Food and Drug Administration (FDA) approved 27 new drugs, of which more than half were specialty.

The number of FDA-approved drugs fell more than 30 percent in 2013 compared to the 39 therapies approved in 2012, a 15-year high. Of the newly approved drugs in 2013, 14 were considered specialty drugs for therapeutic areas, such as chronic viral infections, cancers, autoimmune diseases, genetic disorders, and rare diseases.

Despite the decline, the tally of innovative medications approved in 2013 is in line with the historical trend of the agency, which has on average approved 28 new drugs annually over the past five years.  According to experts, the decline in drugs approved is due to fewer drug applications submitted to the agency.  The FDA received at least 32 applications for innovative medicines in 2013, down from 41 in 2012, according to a presentation by the FDA last month. John K. Jenkins, director of the office of new drugs at the FDA’s Center for Drug Evaluation and Research said that nearly three quarters of the novel drugs approved last year would be going to market first in the US.

Three therapies that were granted Breakthrough Therapy Designation by the FDA received approval last year. These include Roche’s Gazyva, approved for treatment of previously untreated chronic lymphocytic leukemia (CLL); Janssen and Pharmacyclics’ Imbruvica, approved for treatment of mantle cell lymphoma; and Gilead’s Sovaldi, approved for treatment of chronic hepatitis C virus.

Additional approved drugs approved for treatment of specialty conditions include Janssen’s Olysio for hepatitis C, Actelion’s Opsumit for pulmonary arterial hypertension (PAH), ViiV’s Tivicay for HIV, Boehringer Ingelheim’s Gilotrif for non-small cell lung cancer, GlaxoSmithKline’s (GSK) Mekinist for melanoma with BRAF V600E or V600K mutations, GSK’s Tafinlar for melamona with BRAF V600E mutation, Bayer’s Xofigo for prostate cancer, Biogen’s Tecfidera for multiple sclerosis (MS), Genentech’s Kadcyla for HER2-positive breast cancer,  Celgene’s Pomalyst for multiple myeloma, and Navidea’s Lymphoseek for breast cancer or melanoma.

The FDA is scheduled to determine whether or not to approve several innovative medications, such as Biogen’s long-lasting injectable drug for hemophilia.

Source: US Food and Drug Administration

Last updated: 1/3/14; 10:30AM EST

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