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Advanced Cell Technology’s MA09-hRPE cells for treatment of SMD: FDA grants orphan drug designation

Advanced Cell Technology, Inc., a biotechnology company applying cellular technology in the field of regenerative medicine, announced today that the U.S. Food and Drug Administration (FDA) has granted orphan drug designation for the company’s MA09-hRPE cells for use in the treatment of Stargardt’s Macular Dystrophy (SMD). As a result, the company is eligible to receive a number of benefits, including tax credits, access to grant funding for clinical trials, accelerated FDA approval and allowance for marketing exclusivity after drug approval for a period of as long as seven years.

3 Mar 2010

Determining personal risk for genetic disorders essential

Genetic diseases affect an estimated 12 million Americans, yet according to a survey of 1,000 people conducted by the Genetic Disease Foundation (GDF), while two-thirds of those surveyed were willing to and saw the benefits of undergoing genetic testing, close to 80 percent had never talked to their physician about genetic screening – an inconsistency that can have serious implications on a person's overall health and the health of their immediate family members.

1 Mar 2010

NPS Pharmaceuticals supports second annual U.S. Rare Disease Day observance

NPS Pharmaceuticals, a specialty pharmaceutical company developing innovative therapeutics for rare gastrointestinal and endocrine disorders, announced today its support for the second annual U.S. Rare Disease Day observance on February 28.

1 Mar 2010

Single submission of orphan drug designation annual report for both FDA and EMA

In recognition of World Rare Disease Day, the U.S. Food and Drug Administration and the European Medicines Agency (EMA) today announced a more streamlined process to help regulators better identify and share information throughout the development process of orphan drug and biologic products, which are developed specifically to treat rare medical conditions. Both agencies have agreed to accept the submission of a single annual report from sponsors of orphan drug and biologic products designated by both the United States and the European Union.

27 Feb 2010

CHI lauds California state Assembly's proclamation of Feb. 28, as Rare Disease Day

CHI-California Healthcare Institute, a non-profit public policy research organization, applauds the state Assembly’s proclamation of Feb. 28, as Rare Disease Day. This day helps bring awareness of the biomedical companies that are dedicated to researching and developing treatments for patients suffering from rare diseases.

27 Feb 2010

Shire receives marketing approval from FDA for VPRIV

Shire plc, the global specialty biopharmaceutical company, announced today that the U.S. Food and Drug Administration has granted marketing approval for VPRIV, a human cell line derived enzyme replacement therapy (ERT) for the long-term treatment of Type 1 Gaucher disease in pediatric and adult patients.

27 Feb 2010

Rare Disease Day 2010: Alexion Pharmaceuticals joins NORD and EURORDIS to raise awareness

Alexion Pharmaceuticals, Inc., has joined the National Organization for Rare Disorders (NORD) and the European Organization for Rare Diseases (EURORDIS) in supporting the goals of the third annual Rare Disease Day, February 28, 2010.

26 Feb 2010

NORD lauds FDA's establishment of new position within CDER

Peter L. Saltonstall, president and CEO of the National Organization for Rare Disorders (NORD), today said the Food and Drug Administration's (FDA) announcement that it has created a new position—Associate Director for Rare Diseases—in the agency's Center for Drug Evaluation and Research's (CDER) Office of New Drugs (OND) represents an important advance for patients with rare diseases.

26 Feb 2010

"Hope – It's In Our Genes" rare disease campaign slogan translated into multiple international languages and Braille

The Global Genes Project (www.globalgenesproject.org), today announced that its rare disease campaign slogan, "Hope – It's In Our Genes™," has been translated into multiple international languages and Braille by rare disease patient advocates and supporters from around the world. The translation of this slogan unifies the rare disease community and global network under one banner.

25 Feb 2010

ESMO conference on sarcoma and gastrointestinal stromal tumour

More should be done across Europe to ensure that people with rare forms of cancer are not denied access to the best possible treatment, say the organizers of a major European cancer conference to be held in Milan on 9 and 10 March 2010.

25 Feb 2010

ESMO Conference to address issues relating to sarcoma and GIST

25 Feb 2010

FDA approves 53 new cancer indications between July 2005 and end of 2007

The U.S. Food and Drug Administration's Office of Oncology Drug Products approved more than 50 new indications for the use of oncology and hematology drugs and biologics between July 2005, when the office began reviewing marketing applications, and the end of 2007, according to a new agency study.

19 Feb 2010

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