Rare Diseases And Orphan Drugs: Global News (10.31.13)
Recent orphan drug and rare disease global developments are in the news. Here is a list and description of recent developments:
I – Japan
On October 30, an online article in The Japan News, discusses how Japan’s Health, Labor and Welfare Ministry submits a proposal to review the country’s public assistance system for patients with rare or intractable diseases. Currently, the Japanese government subsidizes medical expenses of patients who suffer from 56 rare diseases. If the disease is severe, the government covers all of the medical expenses. Under the new proposed plan:
• Patients with rare diseases will be responsible for 20% of their medical expenses
• There is a cap on the amount patients have to pay, depending on household income
• Number of rare diseases will increase from 56 to 300.
After reaching a conclusion next month on how to handle rare diseases financially, the Ministry will introduce a new law that becomes effective in January 2015.
II – Australia
On October 25, Australia’s PBAC (Pharmaceutical Benefits Advisory Committee), following a decision in July 2013 to defer a recommendation on the reimbursement of Vertex Pharmaceutical’s orphan drug Kalydeco (Ivacaftor) for Cystic Fibrosis (CF), has decided again to defer a decision – until March 2014. Vertex Pharmaceuticals needs to submit their submission by November 6. About 200 CF patients who have the G551D mutation qualify for Kalydeco.
The alphabetical list of designated orphan drugs in Australia, from the Australian Government’s Department of Health & Ageing’s Therapeutic Goods Administration (TGA), is updated as of October 25, 2013.
III – United Kingdom
Earlier in October, the University of Cambridge, Illumina Inc., and Genomics England, announce the start of a 3-year project that will sequence 10,000 whole genomes of children and adults with rare genetic diseases. This is a pilot project for Genomics England’s national 100,000 Genome Project, which will sequence the genome or personal DNA code of 100,000 patients in the UK’s National Health Service (NHS).
IV – Canada
Bellus Health, a Canadian drug development company focusing on rare diseases, announces on October 28 a partnership with AmorChem Holdings to develop drug candidates for the treatment of rare disease AL Amyloidosis. AL Amyloidosis patients accumulate proteins in different parts of their bodies, mainly the kidneys, heart, liver, and peripheral nerves. When these proteins build up, they disrupt the function and structure of the affected tissues and organs leading to organ failure and eventually death. Median survival time is 1 – 2 years after diagnosis.
V – United States
Children’s Hospital of Philadelphia (CHOP) invests $50 million in Spark Therapeutics, a new biotech startup, that “seeks to be the nation’s first commercial provider of gene therapy”. Spark Therapeutics will assume control over two clinical trials that started at CHOP:
• A rare inherited form of blindness caused by mutation in the RPE65 gene
• Hemophilia B.
Spark Therapeutics plans on providing treatment for more genetic diseases in the future – other rare forms of blindness, blood disorders, and neurodegenerative diseases. Several gene therapies have received FDA orphan designation, but none has yet been approved. In Europe, a gene therapy called Glybera receives approval for the treatment of Lipoprotein Lipase Deficiency in 2012.
Bluebird bio, a Cambridge, Massachusetts biotechnology company developing gene therapies for severe genetic and rare diseases, announces October 28 that the first patient undergoes infusion with the company’s orphan product Lenti-D in its Phase II/III CCALD (Childhood Cerebral Adrenoleukodystrophy) study, Starbeam (ALD-102), in an “autologous hematopoietic stem cell transplantation”. Adrenoleukodystrophy (ALD), a rare X-linked, inherited neurological disorder, is made famous in the movie “Lorenzo’s Oil”.
GlycoMimetics, an orphan disease biotech in Gaithersburg, Maryland, announces on October 28 the terms for its Initial Public Offering (IPO). The company hopes to raise $60 million by offering 4 million shares at a price range of $14 – $16. The company plans to list on the NASDAQ under the symbol GLYC.
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