Rare Disease News and Research

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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

First Edition: March 10, 2010

Protests, million-dollar ad campaigns, deepening partisan fault lines and president's stepped-up health reform end game dominate today's health care headlines.

10 Mar 2010

AMT initiates dosing in Phase I/II gene therapy clinical trial for hemophilia B

Amsterdam Molecular Therapeutics, a leader in the field of human gene therapy, announced today that the first patient has been dosed in the Phase I/II exploratory clinical trial with a gene therapy product for hemophilia B, a seriously debilitating and potentially lethal disease.

10 Mar 2010

Global Genes Project: World Rare Disease Day 2010, a tremendous success

The Global Genes Project, a worldwide rare disease awareness initiative, today announced the success from its World Rare Disease Day 2010 efforts and laid out plans for future rare disease awareness campaigns.

8 Mar 2010

Humanetics' BIO 300 medical radiation countermeasure drug candidate issued U.S. patent

In an announcement today, Humanetics Corporation reported that its BIO 300 medical radiation countermeasure drug candidate has been issued a U.S. patent. The patent is assigned to the United States of America with Humanetics as the designated licensee.

5 Mar 2010

FDA approves CSL Behring's Hizentra for PI

CSL Behring announced today that the U.S. Food and Drug Administration (FDA) has granted marketing approval for HizentraTM, Immune Globulin Subcutaneous (Human), 20% Liquid, for treating patients diagnosed with primary immunodeficiency (PI). A once weekly immunoglobulin (Ig) replacement therapy, Hizentra provides effective protection against infection by maintaining a steady and normal level of immunoglobulin in the body.

4 Mar 2010

Older women with suspected ovarian cancer 'face referral delays'

Older women with suspected ovarian cancer are often not referred for investigation as quickly as younger women, new research suggests.

4 Mar 2010

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