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Rare Diseases And Orphan Drugs: Global News (10.11.13)

October 11, 2013

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

I – Scotland

The purpose of the Scottish Medicines Consortium (SMC) is to accept the use of newly licensed drugs that represent good value for the money and to make available the drugs quickly to patients. SMC analyzes information supplied by the drug company on the health benefits of the drug and the justification of its price. Scottish Health Secretary Alex Neil recently announces that the drug approval system is to be overhauled to improve patient access to new drugs. The changes include examining the criteria that SMC uses for deciding if expensive drugs for treating rare diseases or extending a patient’s life are good value for the money.

The Scottish Government has also extended the new Rare Medicines Drug Fund from the original end date of April 2014 to 2016.

II – Canada

The National Gaucher Foundation of Canada announces the release of a short children’s film, “Emma’s Garden: Growing with Gaucher” on October 1, the start of Gaucher Awareness Month. The digital story shows Emma’s journey from first diagnosis at age 3 through age 13, when she starts treatment for her condition. The film is produced with funding from Genzyme’s Patient Advocacy Leadership (PAL) Award. To view the film, please visit the website.

III – United States

GlycoMimetics, is a clinical-stage, Maryland-based biotech developing treatments for orphan diseases. The company files at the beginning of October, a registration statement on Form S-1, with the U.S. Securities and Exchange Commission (SEC) for the proposed Initial Public Offering (IPO) of its common stock. The company plans to list on the NASDAQ under the symbol “GLYC”. In September, the European Medicines Agency (EMA), grants orphan drug designation to GMI-1070 (Rivipansel Sodium) for the treatment of Vaso-Occlusive Crisis (VOC) in patients with Sickle Cell Disease.

The FDA announces on October 8, 2013, the approval of Bayer’s orphan drug Adempas (Riociguat) to treat adults for two forms of Pulmonary Hypertension (PH):

•   Pulmonary Arterial Hypertension (PAH)
•   Chronic Thromboembolic Pulmonary Hypertension (CTEPH).

This is the 10th treatment for PH that the FDA approves. PH is a rare and life-threatening lung disease affecting approximately 20,000 – 30,000 patients.

IV – Taiwan

According to an online October article in the Taipei Times, a study by the local Mackay Memorial Hospital shows that newborn screening and early treatment of the rare genetic disease, Mucopolysaccharidosis Type I (MPS I), is possible. MPS I is the result of a genetic defect that effects the production of the enzyme, Alpha-L-Iduronidase (IDUA). The study is a four and a half-year newborn screening pilot program for MPS I, that measures IDUA activity in 35,000 newborns. The screening program detects two newborns with MPS I before the onset of the disease, and then initiates subsequent checking to ensure early medical intervention. The study identifies the incidence of MPS I in Taiwan as 1/18,000 births.

V – Germany

According to an online EURORDIS article, Germany adopts a rare disease strategy after a three-year process. The plan, “Measures to Improve the Health Situation of Persons with Rare Diseases in Germany”, has 52 unique action proposals.

Please Note: “Earth6391” courtesy of THEBLITZ1 at en.wikipedia [public domain].

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

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