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KRX-0401 receives Orphan Drug designation from FDA for treatment of neuroblastoma

Keryx Biopharmaceuticals, Inc. today announced that KRX-0401 (perifosine) has received Orphan Drug designation from the U.S. Food and Drug Administration for the treatment of neuroblastoma, a cancer of the nervous system affecting mostly children and infants for which there are no FDA approved therapies.

15 Jul 2010

Aeterna Zentaris' partner Keryx receives FDA orphan-drug designation for perifosine in neuroblastoma

Aeterna Zentaris Inc., a late-stage drug development company specialized in oncology and endocrine therapy, today announced that its partner, Keryx Biopharmaceuticals, Inc., has been granted orphan-drug designation by the U.S. Food and Drug Administration for perifosine, Aeterna Zentaris' novel, potentially first-in-class, oral Akt inhibitor, for the treatment of neuroblastoma.

15 Jul 2010

Biogen Idec, Swedish Orphan Biovitrum present results from rFIXFc Phase 1/2a study in hemophilia B

Biogen Idec and Swedish Orphan Biovitrum AB today announced results from a Phase 1/2a open-label, dose-escalation, safety and pharmacokinetic study of the companies' long-lasting, fully-recombinant factor IX Fc fusion protein (rFIXFc) in hemophilia B patients. The data, which were presented at the World Federation of Hemophilia Congress in Buenos Aires, Argentina, on July 11, 2010, showed that rFIXFc was well tolerated and demonstrated an approximately three-fold increase in half-life compared to historical data for existing therapies.

12 Jul 2010

Biogen Idec, Swedish Orphan Biovitrum announce advancement of rFVIIIFc into registrational study

Biogen Idec and Swedish Orphan Biovitrum today announced that they plan to advance the companies' long-lasting, fully-recombinant factor VIII Fc fusion protein (rFVIIIFc) into a registrational clinical trial in people with hemophilia A. The decision to advance the program is based on promising data from a Phase 1/2a open-label, cross-over, multi-center, dose-escalation study that evaluated the safety and pharmacokinetics of an intravenous injection of rFVIIIFc in 16 previously-treated patients with severe hemophilia A.

12 Jul 2010

Pfizer to present results of hemophilia studies at WFH 2010 Congress

Pfizer Inc, the world's leading biopharmaceutical company, today announced that the results of a number of hemophilia studies will be presented at the World Federation of Hemophilia 2010 Congress taking place July 10-14, 2010, in Buenos Aires, Argentina. Key research includes a pre-clinical evaluation of recombinant factor Xa as a potential new approach to restoring hemostasis, as well as a study assessing the potential for an engineered recombinant factor VIIa molecule to improve therapeutic outcomes in mouse models of hemophilia.

9 Jul 2010

FDA grants orphan drug designation for Synageva BioPharma's SBC-102

Synageva BioPharma Corp., a privately held biopharmaceutical company, announced that the U.S. Food and Drug Administration has granted orphan drug designation for SBC-102, the Company's enzyme replacement therapy in development to treat Lysosomal Acid Lipase Deficiency, also known as Wolman Disease and Cholesteryl Ester Storage Disease, a condition for which there is currently no approved treatment.

7 Jul 2010

Researchers identify a set of molecular signatures for biliary atresia

Researchers have identified a set of "molecular signatures" for biliary atresia - the most common diagnosis leading to liver transplant in children - that can help identify the progression of disease at diagnosis and predict clinical outcomes.

1 Jul 2010

NORD urges FDA to reduce regulatory uncertainty in developing medicines for rare disorders

Noting that only about 200 of the nearly 7,000 diseases classified as rare currently have treatments, the National Organization for Rare Disorders (NORD) today called for a Food and Drug Administration (FDA) statement of policy on regulation of therapies for rare disorders.

30 Jun 2010

Recent, upcoming FDA efforts address rare, 'neglected' diseases

The FDA's new rare disease review group will hold its first public hearing on Tuesday and Wednesday to discuss how to expand efforts to develop treatments for rare diseases that affect fewer than 200,000 people, Reuters

30 Jun 2010

FDA grants t2cure orphan drug designation for t2c001 in Buerger's Disease

t2cure GmbH, a biopharmaceutical company developing and marketing stem cell-based regenerative therapies, announced today that the FDA has granted orphan drug designation to t2c001 for the treatment of Buerger's Disease. The Company also announced that in a concurrent application in Europe the EMA's Committee for Orphan Medicinal Products recently recommended t2c001 for orphan designation to the European Commission.

24 Jun 2010

FDA grants Tolera's TOL101 orphan drug designation for treatment of T1DM

Tolera Therapeutics, Inc., was granted orphan drug status by the United States Food and Drug Administration (FDA) for TOL101 (anti-TCR murine monoclonal antibody, type IgM), for treatment of recent onset immune-mediated Type 1 diabetes mellitus (T1DM). Designation is granted for treatment of patients sixteen years of age and younger with immune-mediated T1DM and preserved pancreatic beta-cell function, commonly referred to as juvenile diabetes.

23 Jun 2010

Teens to call upon Congress to fund drug research for rare diseases

Teens from across the United States whose siblings have cystic fibrosis will press their elected officials in Washington, D.C., June 24, to fund drug research and increase access to clinical trials for those with rare diseases.

22 Jun 2010

Dyax signs partnership agreement with Sigma-Tau to develop and commercialize subcutaneous DX-88

Dyax Corp. and Defiante Farmaceutica S.A., a subsidiary of the pharmaceutical company Sigma-Tau SpA, announced today a strategic partnership to develop and commercialize subcutaneous DX-88 (ecallantide) for the treatment of hereditary angioedema and other therapeutic indications throughout Europe, North Africa, Middle East and Russia.

21 Jun 2010

End Duchenne Day to be celebrated on June 26, 2010: PPMD

Patricia A. Furlong, Founding President and CEO of Parent Project Muscular Dystrophy, the largest non-profit organization in the United States focused on finding a cure for Duchenne muscular dystrophy, announced that the Mayor of Denver, Colorado, John W. Hickenlooper, has proclaimed June 26, 2010 "End Duchenne Day" in recognition of PPMD's 16th Annual Connect Conference. Denver is playing host to the country's largest Duchenne-specific, international conference, June 24 – 27, 2010.

19 Jun 2010

EC grants Allos Therapeutics' pralatrexate orphan medicinal product designation for CTCL treatment

Allos Therapeutics, Inc. announced today that the European Commission (EC) has granted orphan medicinal product designation for pralatrexate for the treatment of cutaneous T-cell lymphoma (CTCL). The Company is currently investigating pralatrexate in a Phase 1 clinical study in patients with relapsed or refractory CTCL.

18 Jun 2010

FDA seeks public input on regulation of laboratory-developed tests

The U.S. Food and Drug Administration today announced plans to hold a public meeting on July 19-20, 2010, to discuss how the agency will oversee laboratory-developed tests (LDTs).

17 Jun 2010

European Commission grants Orphan Medicinal Product Designation for QPI-1002

Quark Pharmaceuticals, Inc., a world leader in the discovery and development of RNAi-based therapeutics, today announced that the European Commission has granted Orphan Medicinal Product Designation for QPI-1002 (also referred to as "I5NP") for the prophylaxis of delayed graft function in kidney transplant patients.

16 Jun 2010

IMF concludes first-of-its-kind inaugural Myeloma Summit

The International Myeloma Foundation, the oldest and largest foundation dedicated to improving the life and care of myeloma patients, has concluded the first-of-its-kind inaugural Myeloma Summit meeting - nearly 70 of the world's leading experts in myeloma and related blood cancers charting the future of myeloma treatment and care.

14 Jun 2010

BIOTECanada congratulates CPHA on 100 years of leadership in public health

As a community partner of the Canadian Public Health Association, BIOTECanada, on behalf of Canada's biotechnology industry, congratulates the CPHA on 100 years of leadership in public health.

11 Jun 2010

FDA grants orphan drug designation for ICT-107

ImmunoCellular Therapeutics announced today that the U.S. Food and Drug Administration has granted orphan drug designation for ICT-107, the company's dendritic cell-based cancer vaccine candidate which targets glioblastoma multiforme. As a result of the orphan drug status, ImmunoCellular will be eligible to receive a number of benefits, including access to grant funding for clinical trials, tax credits, accelerated FDA approval and allowance for marketing exclusivity after drug approval for a period of up to seven years.