Orphan Drugs And Rare Diseases: Global News (04.29.13)

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

I – Australia

In January 2013, the FDA Advisory Committee did not recommend Pharmaxis’ orphan drug Bronchitol (Mannitol) for approval for Cystic Fibrosis (CF). The FDA in March 2013, concludes its review of the Bronchitol New Drug Application (NDA) and recommends that Pharmaxis should conduct an additional clinical trial to obtain approval. Bronchitol has Orphan Drug Designation in the US, Europe, and Australia, and is approved for marketing in Australia and the EU.

Pharmaxis announces April 24, 2013, that a Phase III clinical trial of Bronchitol for Bronchiectasis did not show a significant difference in the rates of pulmonary exacerbation in patients treated over a one year period. As a result of the negative study results, Pharmaxis shares plunge 52%.

II – US

Ipsen, a French drug maker, announces April 25, 2013, that the supplier of orphan drug Increlex (Mecasermin)’s active ingredient, Lonza, is facing manufacturing issues at its site in Hopkinton, Massachusetts. Supply interruptions are to start appearing in the US in the 2^nd quarter 2013 and in Europe and the rest of the world in the 3^rd quarter 2013. “Re-supply before the end of 2013 is not currently anticipated.” Increlex treats a rare disease in children called IGF-1 deficiency, which results in children being very short for their age.

Bristol-Myers Squibb during their first quarter 2013 financial results meeting on April 25, 2013, announces that the FDA has given the Breakthrough Therapy Designation for a triple Direct-Acting Antiviral (DAA) treatment regimen for chronic Hepatitis C. The non-orphan triple-DAA includes :

•   Daclatisvir
•   Asunaprevir
•   BMS-791325.

III – Japan

A recent article from the online JapanDailyPress.com discusses that by age 20, patients with rare diseases stop receiving medical care subsidies. Japan’s Health, Labor, and Welfare Ministry conducts national research revealing that children receiving government medical subsidies for rare or intractable diseases have high medical expenses after turning 20 because the funding ends by then. The government program for subsidizing children’s treatments is launched in 1974. The following are the 2010 program numbers :

•   Approximately 110,000 children receive subsidies
•   Expenses reach $250 million
•   514 diseases are covered.

Under the government program for subsidizing adult treatments, only 56 diseases are covered, with only 15 diseases in both programs overlapping. To help with the financial burden of adults with rare or intractable diseases, the recommendation is to develop a program that will cover patients when they get past the age of 20.

IV – Egypt

The Inherited Kidney Diseases Workshop will be held May 9-10, 2013, in Cairo, Egypt. The Workshop is organized by ERA (European Renal Association), EDTA (European Dialysis & Transplant Association), Egyptian Group for Orphan Renal Diseases (EGORD), and the Egyptian Society of Pediatric Nephrology & Transplantation. EGORD is a national health group of healthcare professionals dedicated to the care of rare genetic kidney diseases. EGORD’s Facebook page.

V – Iran

Per the Tehran Times, there was a groundbreaking ceremony for the building of a medical and research center for rare diseases in the city of Urmia. The center will have “diagnostic and treatment departments and will be free of charge for those who suffer from such diseases.”

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

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