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Orphan Drugs And Rare Diseases: Global News (05.17.13)

May 17, 2013

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

I – Canada

Canada’s Health Minister Aglukkaq announces October 3, 2012, the “1st ever Canadian framework to increase access to new treatments and information and the launch of Orphanet-Canada”. The regulatory framework is a major step, opening the way for research and development of new drugs for rare disease indications and improving access to existing therapies. The announcement reverses a 16-year-old Health Canada policy denying the need for an Orphan Drug Policy in Canada. It comes nearly 30 years after the US passed the world’s first Orphan Drug Act and 12 years after the European Union did the same for its member countries.

Health Canada produces a draft consultation document on the new Canadian Orphan Drug Regulatory Framework. The goal is to submit it to the Canadian Parliament in the Fall of 2013. This first draft document combines the best from the Orphan Drug policies of the EU and the US. The Canadian Organization for Rare Disorders (CORD) in collaboration with Health Canada, is hosting 1-day sessions across Canada for the following upcoming Consultation meetings on Canada’s Orphan Drug Regulatory Framework :

•   05/31/13 in Edmonton
•   06/03/13 in Montreal
•   06/11/13 in Vancouver.

II – Italy

The open access Orphanet Journal of Rare Disease (OJRD) publishes on May 16, 2013, an interesting journal article that identifies the profile of Italian families of patients with rare diseases by using a web survey. The article concludes that “Italian parents of patients with rare diseases are active Internet users, engaged information search, and in online communities.” Some of the interesting statistics presented in this article about parents of children with rare diseases :

•   66% participate in an online forum on health
•   99% search for information on disease characteristics, 93% on therapy, 89% on diagnosis, 63% on alternative therapies, 62% on nutrition, and 54% on future pregnancies
•   82% feel that web information increases comprehension of the rare disease, 65% feel that it improves management of the disease.

The citation for the article is :

Alberto E Tozzi, Rita Mingarelli, Eleonora Agricola, et al. The Internet user profile of Italian families of patients with rare diseases: a web survey. Orphanet Journal of Rare Diseases. May 2013, 8:76 doi:10.1186/1750-1172-8-76.

III – Israel

Kamada, an Israeli therapeutics company focusing on orphan drugs and plasma-derived protein therapeutics, announces on May 15, 2013, the terms for the $60 million US IPO. Kamada’s inhaled AAT (AAT IH) product is in advanced stages of clinical trials, under FDA and EU Orphan Drug Designation (ODD) for several indications :

•   AAT Deficiency
•   Cystic Fibrosis
•   Bronchiectasis.


Callidus Biopharma, a drug discovery company located in Doylestown, Pennsylvania, announces on May 14, 2013, the company has closed on $4.6 million in Series A financing led by two strategic investors. The company will use the money to commercialize “its proprietary protein expression technology” and to accelerate the pre-clinical development of its drug discovery pipeline of therapies for Lysosomal Storage Disease (LSDs) for :

•   Pompe Disease
•   Gaucher Disease
•   Other IGF-2 related LSDs.

The FDA is on a roll with the granting of its 13th Breakthrough Therapy Designation to Genentech’s orphan drug Obinutuzumab for Chronic Lymphocytic Leukemia (CLL). The list of the FDA’s Breakthrough Therapy Designations is available.

V – Asia Pacific

The Department of Obstetrics and Gynaecology at The Chinese University of Hong Kong (CUHK) introduces technology to screen for Fragile X Syndrome. This technology will help to identify women who are carriers of the abnormal gene mutation causing Fragile X Syndrome.

The National Center of Neurology and Psychiatry (NCNP) and Nippon Shinyaku have been working together since 2009 to develop an antisense Oligonucleotide for the treatment of Duchenne Muscular Dystrophy (DMD). Plans are to start an investigator-initiated clinical trial in July 2013. “This product has been developed with the aim of skipping exon 53 of the dystrophin gene.”

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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