Rare Disease News and Research

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Biogen Idec, Swedish Orphan Biovitrum restructure collaboration agreement for rFVIIIFc and rFIXFc programs

Biogen Idec and Swedish Orphan Biovitrum today announced that they have restructured the collaboration agreement for the companies’ long-acting, recombinant Factor VIII Fc fusion protein (rFVIIIFc) in hemophilia A patients and the recombinant Factor IX Fc fusion protein (rFIXFc) in hemophilia B patients.

18 Feb 2010

Quark Pharmaceuticals' QPI-1002 receives orphan drug designation from FDA

Quark Pharmaceuticals, Inc., a world leader in the discovery and development of RNAi-based therapeutics, announced today that the US FDA has granted orphan drug designation for QPI-1002 (also referred to as "I5NP"), a synthetic siRNA targeting p53 mRNA for the prophylaxis of delayed graft function (DGF) in kidney transplant patients.

17 Feb 2010

Lundbeck launches web-based initiative to support people with rare diseases

In support of Rare Disease Day 2010, set for February 28, Lundbeck Inc. today launched the Raise Your Hand to Fight Rare Diseases campaign. The web-based initiative is designed to generate support for people with rare diseases and is being conducted in partnership with the National Organization for Rare Disorders (NORD), the sponsor of Rare Disease Day in the United States.

9 Feb 2010

FDA grants Talecris Biotherapeutics orphan drug designation for Alpha1-Proteinase Inhibitor to treat AAT deficiency

Talecris Biotherapeutics, Inc. announced today that it was granted orphan drug designation by the U.S. Food and Drug Administration (FDA) for the development of an aerosol formulation of Alpha1-Proteinase Inhibitor (Human, A1PI) to treat congenital alpha1-antitrypsin (AAT) deficiency. AAT deficiency is a chronic, hereditary condition that increases the risk of certain diseases, especially emphysema, which typically emerges in the fourth decade of life. Currently, there are no approved, inhaled treatments available for the treatment of AAT deficiency.

5 Feb 2010

Discovery Health, NORD partner for Rare Disease Day

The National Organization for Rare Disorders (NORD) announced that Discovery Health and its new program, DISEASE DETECTIVES, will serve as media and program partners for Rare Disease Day on February 28, 2010.

4 Feb 2010

Denim blue jeans ribbon campaign: an initiative to raise awareness about children with rare diseases

Hope – It's In Our Genes.™ The Children's Rare Disease Network (http://www.crdnetwork.org) today announced the Global Genes Project (http://www.globalgenesproject.org), an initiative designed to raise awareness about the prevalence of rare diseases that afflict millions of children worldwide. Rare disease advocates organizing the campaign selected a blue jeans denim ribbon as the international symbol of hope for children suffering from life threatening and chronically debilitating rare diseases.

1 Feb 2010

Tufts CSDD: Orphan product designations increased up to 425 in 2006-08

The number of orphan product designations in the U.S. more than doubled during the last decade, reflecting growing interest by pharmaceutical and biotech companies in developing products to treat orphan diseases, according to a study recently completed by the Tufts Center for the Study of Drug Development.

21 Jan 2010

A novel approach for funding children's genetic disease research

Parent Project Muscular Dystrophy together with Trisomy 18 Foundation are among 100 charities that earned a $25,000 grant during Round 1 of the Chase Community Giving Challenge on Facebook.

19 Jan 2010

GAO: Some meds had 'extraordinary' price hikes over past eight years

A report from the Government Accountability Office (GAO) issued Monday finds that "[p]rices on a growing number of prescription medications have ballooned in recent years as consolidation in the drug industry leaves fewer companies manufacturing niche medications," The Associated Press reports.

12 Jan 2010

Opinions: U.S. medical emergency response; micronutrients; rotavirus vaccine; antibiotic development

"[D]espite the tireless efforts of public health and health-care workers, America's experience with H1N1 shows that the nation is not prepared to deal with a flu pandemic," former Democratic Senator Bob Graham and former Republican Senator Jim Talent write in Washington Post opinion piece that examines U.S. preparedness for medical emergencies. The writers chaired the Commission on the Prevention of Weapons of Mass Destruction Proliferation and Terrorism.

6 Jan 2010

Senate health care bill will increase patient access and help create high-wage, high-value jobs

“These provisions mean increased access for the patients of today and renewed hope for the patients of tomorrow. In addition, they will help create high-wage, high-value jobs in our nation’s burgeoning biotech sector and help maintain U.S. global leadership in innovation.”

28 Dec 2009

FDA provides guidance on applying for orphan drug designation

As part of its continuing effort to make the agency more transparent and accessible, the FDA has scheduled a series of workshops about orphan drug designation for academics, biotechnology companies, and those unfamiliar with the process. The

24 Dec 2009

University of Florida obtains stimulus grant to establish ataxia consortium

University of Florida neurologists are trying to change that with the help of a $1 million Challenge grant from the National Institute of Neurological Disorders and Stroke to establish a nationwide network of physician-scientists with expertise in clinical ataxia research.

16 Dec 2009

Manchester Pharmaceuticals' Chenodal now available for patients suffering from gallstones

Manchester Pharmaceuticals, Inc.(TM) announced today the availability of Chenodal(TM) 250 mg (chenodiol tablets). On October 22, 2009, the US Food and Drug Administration (FDA) approved Chenodal, an important therapy for patients suffering from gallstones in whom surgery poses an unacceptable health risk due to disease or advanced age.

15 Dec 2009

EC grants orphan drug designation for MP4CO for treating sickle cell disease

Sangart, Inc., a global biopharmaceutical company dedicated to developing life-saving medicines specifically designed to enhance the perfusion and oxygenation of ischemic (oxygen deprived) tissues through targeted oxygen and other gas delivery, today announced that the European Commission (EC) has granted orphan medicinal product designation for MP4CO for the treatment of sickle cell disease.

10 Dec 2009

FDA launches workshop to simplify orphan drug development

The U.S. Food and Drug Administration (FDA) has announced the launch of an orphan drug workshop series--an opportunity for biotechnology companies, pharmaceutical firms, academics and other interested parties to spend two days creating applications for orphan status designation, under the guidance of staff from FDA's Office of Orphan Products Development (OOPD).

9 Dec 2009

BHR Pharma files an IND for its BHR-100 intravenous progesterone infusion product

BHR Pharma, LLC (BHR) announced today that it has filed an Investigational New Drug (IND) application with the U.S. Food and Drug Administration (FDA) for its proprietary BHR-100 intravenous progesterone infusion product.

8 Dec 2009

Stem cell treatment: A new hope for Multiple Sclerosis

RNL Bio Co., Ltd, (www.rnl.co.kr) a leading biopharmaceutical company specialized in adult stem cell therapeutics announced today that a 46-year-old female, Kang Sook Park's Multiple Sclerosis improved tremendously after receiving stem cell treatment. Park was suffering from MS (Multiple Sclerosis), an autoimmune disease that affects the central nervous system (brain and spinal cord).

6 Dec 2009

Allos Therapeutics to launch the COMPLETE registry for patients with PTCL

Allos Therapeutics, Inc. today announced the launch of an international registry designed to address the urgent need to better understand treatment patterns and outcomes for patients with peripheral T-cell lymphoma (PTCL). The registry, known as COMPLETE (Comprehensive Oncology Measures for Peripheral T-cell Lymphoma Treatment), is a global observational study that will enroll patients with newly-diagnosed PTCL and obtain data regarding longitudinal treatment patterns and outcomes.

3 Dec 2009

Pfizer, Protalix team up to develop and market plant-based enzyme for Gaucher’s disease treatment

Pfizer and Protalix (NYSE-Amex: PLX) today announced that they have entered into an agreement to develop and commercialize taliglucerase alfa, a plant-cell expressed form of glucocerebrosidase (GCD) in development for the potential treatment of Gaucher’s disease.

1 Dec 2009