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Orphan Drugs And Rare Diseases: Global News (05.28.13)

May 28, 2013

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

I – Australia

A May 25, 2013, article in the Australian Herald Sun, discusses how Pompe Disease patients are appealing directly to the Health Minister so that they can have access to the drug Myozyme for the treatment of their rare disease. Access to Myozyme has been refused by Australia’s Pharmaceutical Benefits Scheme (PBS) for the seventh time. The drug company that makes Myozyme has decided not to re-apply. Some of the Pompe Disease patients are receiving compassionate access to Myozyme from the drug company, but there are five Australians who currently have no access to the treatment.

ARIAD Pharmaceuticals’ drug Ponatinib (Iclusig in the US), is designated as an orphan drug on May 14, 2013. Ponatinib is for the treatment of Acute Lymphoblastic Leukemia and adult patients with chronic phase, accelerated phase, or blast phase Chronic Myeloid Leukemia (CML) that is resistant or intolerant to prior Tyrosine Kinase Inhibitor (TKI) therapy.

The Australian reports that Pharmaxis will slash 45 jobs and cut costs as the company restructures after failing to achieve regulatory milestones. The company says that it will seek “partnership opportunities” for Bronchitol in the US for Cystic Fibrosis and globally for Bronchiectasis. Pharmaxis will retain direct commercial interest in Bronchitol in Europe.

II – France

On May 22, 2013, it is announced that the not-for-profit AFM-Telethon, the French Muscular Dystrophy Association, and the Fonds National d’Amorcage (FNA), a fund set up by the French government to provide capital to companies developing innovative technologies, launches the first seed capital fund dedicated to “innovative biotherapeutics and rare diseases”. The new fund, managed by CDC Enterprises, launches with EUR 50 million (approximately $64.2 million US). According to, the plan is for the fund to invest EUR 3 million to EUR 10 million ($3.9 – $12.8 million US) each in 12-15 companies, with the majority of investments to be in French companies.  The AFM-Telethon’s mission is to “initiate innovative actions and a strategy of general interest that benefits all rare diseases and all persons with disabilities.”


A May 2013 article from Pharmatimes Magazine, “Rare drugs – raw deal ?”, assesses ultra-orphan drugs. The article discusses the new NICE (National Institute for Health and Care Excellence) function to “calculate the cost effectiveness of expensive drugs for rare diseases”. NICE is taking over this evaluation from the Advisory Group for National Specialized Services. The first test according to the article, will be NICE’s evaluation of Alexion Pharmaceuticals’ orphan drug Soliris (Eculizumab), currently the world’s most expensive drug. Soliris is approved for the treatment of Atypical Haemolytic Uremic Syndrome (aHUS), a rare kidney disease. Less than 300 people in the UK have aHUS. According to the article, “It is too soon to say whether NICE’s assessment of ultra-orphan drugs will benefit rare disease patients, but its decisions will undoubtedly be scrutinised.”

According to a May 2013 article, Shire is found guilty of 10 breaches of the ABPI (Association of the British Pharmaceutical Industry) Code of Conduct in the UK, after using a press release for its Gaucher Disease drug Vpriv, to knock a rival product, Genzyme’s Cerezyme for Gaucher Disease. A complaint was brought by Genzyme. “Genzyme contends that a press release from Shire, sent out last year in the UK, was not accurate, balanced, fair or based on up-to-date information.” Shire’s breaches of the Code are all related “to how the rare disease specialist has used clinical trial data in a press release when comparing its drug to Cerezyme.” Interesting details are provided in the article.

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

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