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AMT's MAA for LPLD gene therapy product Glybera on schedule

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, today announced that the Marketing Authorisation Application (MAA) for Glybera(R) remains on schedule following meetings with the European Medicine Agency (EMA) concerning the Day 120 List of Questions. The company is confident that Glybera(R), a gene therapy product for lipoprotein lipase deficiency (LPLD), remains on track for a regulatory decision by mid-2011.

18 Aug 2010

Researchers discover genetic alterations responsible for Kabuki syndrome

Using a new, rapid and less expensive DNA sequencing strategy, scientists have discovered genetic alterations that account for most cases of Kabuki syndrome, a rare disorder that causes multiple birth defects and mental retardation. Instead of sequencing the entire human genome, the new approach sequences just the exome, the 1-2 percent of the human genome that contains protein-coding genes.

16 Aug 2010

FDA grants BioMimetic Therapeutics orphan drug designation for rhPDGF-BB in treatment of OCD

BioMimetic Therapeutics, Inc. today announced that it has received orphan drug designation from the Food and Drug Administration for its protein therapeutic, recombinant human platelet derived growth factor, to be used in conjunction with autograft and/or commercially available osteochondral allograft for the treatment of osteochondritis dissecans of the knee, elbow or ankle.

12 Aug 2010

U.S. Senators introduce legislation to encourage R&D aimed at neglected pediatric diseases

"New bipartisan legislation was introduced in the U.S. Senate last week which seeks to encourage innovative [research and development] R&D by drugmakers aimed at treating rare and neglected pediatric diseases," PharmaTimes reports.

11 Aug 2010

FDA grants Orphan Drug Designation to YM BioSciences' JAK1/2 inhibitor

YM BioSciences Inc., announced that the Office of Orphan Products Development of the U.S. Food and Drug Administration has granted Orphan Drug Designation to the Company's highly-selective JAK1/2 inhibitor, CYT387, for the treatment of myelofibrosis, a chronic debilitating unmet medical need, in which a patient's bone marrow is replaced by scar tissue, and for which treatment options are limited or unsatisfactory.

10 Aug 2010

ASEBIO to host Biospain 2010

The Spanish Bioindustry Association (ASEBIO), in collaboration with the Navarre Society of Development (SODENA), and the government of Navarre, will host BioSpain 2010 the biennial international gathering of experts in the biotechnology sector, in Pamplona from September 29th to October 1st. BioSpain 2010 is honoured to welcome three lecturers of international prestige: Mr. Carlos Cordon-Cardo, Vice-Chair of Pathology & Professor, Columbia University; Mr. Larry Fritz, President & CEO, Covella Pharmaceuticals Inc. and Life Science Venture Partner, Westfield Capital Management Company LP; and Mr. Conny Bogentoft, CEO Karolinska Development AB.

8 Aug 2010

U.S. Senate passes I-ACT to enable patients with rare disease to participate in clinical trials

The U.S. Senate last night passed the "Improving Access to Clinical Trials Act" (I-ACT), a bipartisan piece of legislation championed by the Cystic Fibrosis Foundation, its advocates and 120 other health advocacy organizations.The legislation enables patients with rare diseases to participate in clinical trials without losing eligibility for public healthcare benefits.

7 Aug 2010

Kids v Cancer applauds Senators for Creating Hope Act of 2010

Kids v Cancer applauds Senators Sam Brownback (R-KS), Sherrod Brown (D-OH), and Al Franken (D-MN) for their dedication to curing childhood diseases and supporting the bipartisan "Creating Hope Act of 2010."

7 Aug 2010

PFF announces introduction of Pulmonary Fibrosis Research Enhancement Act in US Senate

The Pulmonary Fibrosis Foundation (PFF) today announced that the Pulmonary Fibrosis Research Enhancement Act (PFREA) was introduced in the United States Senate. The PFREA was introduced by Senators Patty Murray (D-WA) and Mike Crapo (R-ID), and the PFF has offered its unconditional endorsement to pass this important legislation on behalf of the pulmonary fibrosis community.

5 Aug 2010

Carfilzomib could become new option for patients with relapsed myeloma, IMF says

The International Myeloma Foundation, the oldest and largest foundation dedicated to improving the life and care of myeloma patients, today said promising data suggest a new drug called carfilzomib could become an important new option for patients whose myeloma stops responding to other therapies.

4 Aug 2010

Acorda second-quarter net loss decreases to $6.8 million

Acorda Therapeutics, Inc. today announced its financial results for the second quarter of 2010.

3 Aug 2010

Octapharma USA receives confirmation of orphan drug exclusivity from FDA for wilate

Octapharma USA has received confirmation of orphan drug exclusivity from the U.S. Food and Drug Administration for wilate (von Willebrand Factor/Factor VIII Concentrate, Human), the replacement therapy developed specifically for von Willebrand Disease. The approval comes from the FDA Office of Orphan Products Development, which helps to advance the development of products that demonstrate promise for the treatment of rare diseases.

2 Aug 2010

Taligen Therapeutics to highlight three pipeline candidates at XXIII ICW

Taligen Therapeutics, a biotechnology company developing therapies that regulate the complement system to treat inflammatory and immune diseases, today announced that preclinical data on three of its pipeline candidates will be highlighted at the XXIII International Complement Workshop taking place August 1-5, 2010, in New York City.

2 Aug 2010

Researchers identify genetic mutation for Uner Tan Syndrome

A team of researchers from leading universities in Turkey and the United States have reported that they have identified the genetic mutation responsible for the Uner Tan Syndrome (UTS) using advanced genetic capture and sequencing technologies from Roche. The combined genomic technologies enabled the team of scientists to identify the causative mutation of this debilitating disease after years of mystery.

30 Jul 2010

Researchers discover enzyme crucial for DNA repair

Researchers have discovered an enzyme crucial to a type of DNA repair that also causes resistance to a class of cancer drugs most commonly used against ovarian cancer.Scientists from The University of Texas MD Anderson Cancer Center and the Life Sciences Institute of Zhejiang University in China report the discovery of the enzyme and its role in repairing DNA damage called cross-linking in the Science Express advance online publication of Science.

30 Jul 2010

Isis Pharmaceuticals receives $5M payment from GSK for dug development

Isis Pharmaceuticals, Inc. announced today that it has earned a $5 million milestone payment from GlaxoSmithKline related to the designation as a development candidate of a drug to treat an undisclosed rare and serious disease. Isis will develop the drug to Phase 2 proof-of-concept, at which time GSK has the option to license it.

29 Jul 2010

First patient enrolled in Phase 3 SyNAPSe study of BHR-100 for TBI

BHR Pharma, LLC announced today that the first patient has been enrolled in its SyNAPSe study, a global, Phase 3, pivotal trial to evaluate the safety and effectiveness of its intravenous progesterone infusion product (BHR-100) as a neuroprotective agent for treating severe (Glasgow Coma Scale scores of 4-8) traumatic brain injury patients.

23 Jul 2010

Prevalence of HD substantially underestimated in the UK

The prevalence of Huntington's disease is substantially underestimated in the UK, with significant implications for those affected, the healthcare system, and research. New estimates of prevalence, and their implications, are discussed in a comment published in an upcoming Lancet, written by Professor Sir Michael Rawlins, who is the Chairman of the UK National Institute for Health and Clinical Excellence, but writes in his capacity as an Honorary Professor of the London School of Hygiene and Tropical Medicine, UK.