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Synageva BioPharma seeks FDA orphan drug designation for SBC-102 enzyme replacement therapy

Synageva BioPharma Corp., a privately held biopharmaceutical company, today announced its filing with the U.S. Food and Drug Administration (FDA) requesting orphan drug designation for its program, SBC-102. This program is an enzyme replacement therapy in preclinical development to treat lysosomal acid lipase (LAL) deficiency, also known as Wolman disease or cholesteryl ester storage disease (CESD), a condition for which there is currently no effective treatment.

4 May 2010

Neural stem cell transplantation promising for Batten disease

Neuronal Ceroid Liposfuscinosis (NCL), often referred to as Batten disease, is a rare and fatal neurodegenerative disorder that afflicts infants and young children. NCL is caused by a deficiency of a lysosomal enzyme, and is a relatively rare disease, affecting an estimated 2 to 4 of every 100,000 babies born in the U.S. Although NCLs are classified as rare diseases, they often affect more than one child in families that carry the defective gene, and tragically, are always fatal.

4 May 2010

ImmunoCellular Therapeutics seeks FDA orphan drug status for ICT-107 dendritic-cell based vaccine for GBM

ImmunoCellular Therapeutics announced today it has filed an orphan drug application with the U.S. Food and Drug Administration (FDA) for its cancer treatment candidate ICT-107. ICT-107 is a dendritic-cell based cancer vaccine product which targets glioblastoma multiforme (GBM), the most prevalent, aggressive and malignant form of primary brain tumors.

3 May 2010

DARA Therapeutics collaborates to advance clinical study of KRN5500 for CIPN in cancer patients

DARA BioSciences, Inc. announced today that on April 26, 2010, DARA Therapeutics, a subsidiary of DARA BioSciences, and the Division of Cancer Prevention, National Cancer Institute (NCI), National Institutes of Health (NIH), entered into a Clinical Trials Agreement to advance the clinical study of KRN5500 for the treatment of Chemotherapy Induced Peripheral Neuropathy (CIPN) in patients with cancer. Under the terms of the collaboration, NCI will fund the studies and DARA will supply KRN5500 and placebo.

28 Apr 2010

Alexion Pharmaceuticals reports total revenues of $117.6M from net product sales of Soliris

Alexion Pharmaceuticals, Inc. today announced financial results for the quarter ended March 31, 2010.

22 Apr 2010

Lotus Pharmaceuticals' asthma drug Laevo-Bambutero to receive Chinese SFDA Fast Track Approval

Lotus Pharmaceuticals, Inc., a growing developer, manufacturer and seller of medicine and drugs in the People's Republic of China reported that its asthma drug Laevo-Bambutero will receive the China's State Food & Drug Administration's (SFDA) Fast Track Approval to enter clinical trials. The SFDA's Drug Review and Evaluation Center told the Company during recent consultations at the end of the Company's IND that formal approval would be granted by the Beijing office of the SFDA.

21 Apr 2010

CSL Behring calls for grant requests through LEAD program

CSL Behring, a global leader in the plasma protein biotherapeutics industry, is calling for grant requests through its Local Empowerment for Advocacy Development program. The LEAD program awards funds to United States patient organizations that assist people with rare diseases in advocating for continued access to health care and life-saving plasma protein therapies.

13 Apr 2010

Broken heart disease affects women in post menopause period

It seems an infarction, but it's not. It's called Tako-Tsubo syndrome, or stress-induced cardiomyopathy, and it's a rare disease which at first used to be confused with the far more common (and dangerous) cardiac infarction. Patients arrive to the emergency room with the characteristic heart attack symptoms: acute pain in the chest, an electrocardiogram with the typical changes and the release of those enzymes associated with the usual heart disease.

9 Apr 2010

Scientists use non-viral, synthetic nanoparticle carrier to restores vision

Take a look at this: Scientists from Buffalo, Cleveland, and Oklahoma City made a huge step toward making the blind see, and they did it by using a form of gene therapy that does not involve the use of modified viruses.

1 Apr 2010

National Doctors' Day: St. Jude Children's Research Hospital honors physicians and staff

As physicians nationwide are acknowledged for their talent, sacrifice and courage on National Doctors' Day, St. Jude Children's Research Hospital honors its more than 230 staff and consulting physicians who strive to fulfill the hospital's mission each day.

31 Mar 2010

FDA grants Orphan Drug Designation for anti-diabetic drug

The FDA has granted Orphan Drug Designation of Diamyd Medical´s lead drug candidate Diamyd®. Orphan drugs qualify for seven years of market exclusivity from the date of US marketing approval, tax credits for clinical research and a waiver for FDA user fees.

24 Mar 2010

BioSante Pharmaceuticals receives FDA Orphan Drug designation for GVAX AML Vaccine

BioSante Pharmaceuticals, Inc. today announced positive GVAX AML Vaccine results and BioSante’s receipt of Orphan Drug designation from the FDA’s Office of Orphan Products Development for GVAX AML Vaccine in the treatment of acute myeloid leukemia. This Orphan Drug designation is the second GVAX cancer vaccine from BioSante to secure FDA orphan drug designation.

23 Mar 2010

ViroPharma to present Cinryze data at Southern European Allergy Society congress

ViroPharma Incorporated today announced the presentation of two abstracts relating to Cinryze™ (C1 esterase inhibitor [human]) therapy at the First International Congress of the Southern European Allergy Society in Florence, Italy.

19 Mar 2010

PhRMA President honors Ron and Raychel Bartek for driving awareness of Friedreich's ataxia

Pharmaceutical Research and Manufacturers of America President and CEO Billy Tauzin today honored Ron and Raychel Bartek for their dedicated work driving awareness of Friedreich's ataxia, a debilitating, life-threatening, rare disease.

19 Mar 2010

Call for European Cystic Fibrosis healthcare gap to be closed

A healthcare gap amounting to a ‘death sentence’ for Cystic Fibrosis (CF) children born in Eastern Europe must be closed say researchers from the EuroCareCF Coordination Action for Cystic Fibrosis.

18 Mar 2010

BioSante Pharmaceuticals receives receipt of Orphan Drug designation for GVAX Pancreas Vaccine

BioSante Pharmaceuticals, Inc. today announced BioSante’s receipt of Orphan Drug designation from the FDA’s Office of Orphan Products Development for GVAX Pancreas Vaccine in the treatment of pancreatic cancer.

15 Mar 2010

20.2% women in New Jersey receive delayed or no prenatal care

In releasing a new report on maternal health nationwide, Amnesty International today revealed that flaws and shocking disparities in maternal health care that the government is ignoring lead to two to three women dying daily in the United States from pregnancy-related complications, with half of these deaths believed preventable, according to the Centers for Disease Control. A state-by-state examination shows that New Jersey is 35th on a maternal mortality ranking, with 11.3 deaths per 100,000 live births.

12 Mar 2010

Amnesty International: New York stands 47th on maternal mortality ranking

12 Mar 2010

FDA sponsoring workshops to increase rare disease treatment options

The Wall Street Journal: FDA is putting on workshops for pharmaceutical manufacturers to increase the pool of applicants for approved "orphan drugs," that treat rare diseases. Currently, there are about 7,000 so-called orphan diseases in the U.S. that have few or no FDA-approved treatments.

11 Mar 2010

NeoPharm seeks Orphan Drug Designation for IL13-PE38QQR in treatment of IPF

NeoPharm, Inc., announced today that it has filed an orphan drug application with the Office of Orphan Products Development of the United States Food and Drug Administration to have Orphan Drug Designation granted to IL13-PE38QQR (IL13-PE) for the treatment of Idiopathic Pulmonary Fibrosis.

10 Mar 2010