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Orphan Drugs And Rare Diseases: Global News (06.12.13)

June 12, 2013

Recent orphan drug and rare disease global developments are in the news. Here is a list and description of recent developments:

I – Europe

ViroPharma, an international biopharmaceutical company, announces on June 11, 2013, that the European Commission (EC) has granted Orphan Drug Designation (ODD) for Maribavir for the treatment of Cytomegaloviral (CMV) Disease in patients with impaired cell mediated immunity. The company previously receives ODD for Maribavir in the US in June 2011, for treatment of clinically significant Cytomegalovirus Viremia and disease in at-risk patients. CMV is a member of the herpes virus group, which includes the viruses that cause chicken pox, mononucleosis, and herpes labialis (cold sores). Like other herpes viruses, CMV has the ability to remain dormant in the body for long periods of time. CMV can lead to serious disease or death in people with compromised immune systems.

According to a June 11, 2013 article from the Genzyme Rare Community, more than 100 participants from patient associations from several Eastern European countries, came together last month in Sarajevo to host the first Lysosomal Storage Disorders (LSDs) meeting in the Alpe Adria region. The countries participating in the meeting are :

·   Bosnia & Hercegovina
·   Slovenia
·   Macedonia
·   Croatia
·   Montenegro
·   Serbia.

The meeting topics included lectures on LSDs, presentations on the status of LSDs in each country, and personal stories about living with or caring for a patient with LSD.

EU-LIFE, a new alliance of ten European leading life-science institutes, officially launches in Barcelona, Spain, in late May 2013. The purpose of the new alliance is to raise “the international profile of European research and to develop coordinated responses to the challenges facing the sector.”

II – Canada

According to the Cystic Fibrosis Canada organization, the Institute National D’excellence en Sante et en Services Sociaux (INESSS) recommends not including orphan drug Kalydeco (Ivacaftor), used for the treatment of Cystic Fibrosis (CF), in Quebec’s drug formulary. INESSS recognizes the clinical benefits of Kalydeco, but recommends not including it in the formulary due to the high cost. There are less than 12 people with CF in Quebec who have the G551D mutation that the drug is indicated for. Some of the patients are covered through private insurance or can be covered under the Patient d’exception program. Other Canadian provinces continue to negotiate the price with Vertex Pharmaceuticals.

III – United States

According to the Regulatory Affairs Professionals Society (RAPS), the US FDA has finalized a regulation that makes “some relatively minor changes to the orphan drug regulations.” In October 2011, the FDA announces that it wants to make a number of minor revisions to bring its definitions up to date. A consultation process followed with the FDA issuing its final rule on the changes:

•   Clarification of the definition of “orphan subset”
•   Even if a single drug is used to treat multiple indications that would cause it to treat > 200,000 patients, the FDA will “still see the drug as an orphan treatment for the purposes of the Orphan Drug Act, so long as each patient population is < 200,000”.

For further details on the FDA final changes to the Orphan Drug Regulation, please reference the article on the RAPS website.

PatientsLikeMe, a 200,000+ patient network that allows patients with the same conditions to connect with one another, appoints its first CEO, Martin Coulter.

IV – Israel

According to a recent article in The Jerusalem Post, an international group of researchers headed by Israelis, have discovered among Arab children, a rare genetic disease that weakens the immune system of children. The research:

… led to the discovery of an unusual bone marrow malfunction due to mutations in a gene named VPS45 (Vacuolar Protein Sorting 45) as the cause of the disease, which they called Congenital Neutrophil Defect Syndrome … all (7) of the children are the result of consanguinity – inbreeding of first cousins …

V – International

On June 5, 2013,  the formation of a new initiative, the Global Alliance to Enable Responsible Sharing of Genomic and Clinical Data is announced. The aim of the new alliance is to “break down barriers to data sharing in genetic research in the hope of speeding up the discovery of new ways to treat everything from common killers like cancer to rare disorders.” The consortium consists of more than 70 public and non-profit organizations, including:

•   US National Institutes of Health
•   Wellcome Trust Sanger Institute in the UK
•   Beijing Genomics Institute in China.

Please Note: “Globe” by Augiasstallputzer at Wikimedia Commons [Public domain] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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