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Orphan Drugs And Rare Diseases: Global News (06.20.13)

June 20, 2013

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

I – Europe

Celgene is notified that the European Commission (EC) amends the marketing authorization for Revlimid (Lenalidomide), resulting in the approval to treat patients with transfusion-dependent anemia due to low or intermediate-1 risk myelodysplastic syndromes (MDS) associated with an isolated deletion 5q cytogenetic abnormality when other therapeutic options are insufficient or inadequate. Germany is expected to be the first country to bring the therapy to patients with MDS del(5q), while other European countries will launch the drug according to local requirements. Revlimid is approved in the US, Canada, Switzerland, Australia, New Zealand, and several Latin American countries, as well as Malaysia and Israel, for the same indication.

uniQure, a gene therapy company, announces in May 2013, that with its consortium partners it is to receive EUR 2.5 million Eurostars grant to develop an RNA interference (RNAi) gene therapy for Huntington’s Disease. uniQure’s Glybera, a gene therapy for the treatment of rare disease Lipoprotein Lipase Deficiency, is the first approved gene therapy in the Western world.

At a June 2013 meeting, the European Union Committee of Experts on Rare Diseases (EUCERD) adopts a set of Core Recommendations on Rare Disease Patient Registration and Data Collection. The recommendations call for:

“ … international operability of registries and data bases in order for knowledge and data to be pooled and shared. Rare diseases suffer from fragmented knowledge, scattered expertise and patients, and research duplication. Interoperability on a global scale would enable data to be sufficiently pooled to “reach sufficient statistically significant numbers for clinical research and public health purposes”. Using international coding and nomenclature, minimum common data sets, and good practice guidelines would enhance interoperability and maximize the utility of registries and databases.

The involvement of all stakeholders, including patients, policymakers, researchers, clinicians, and industry in designing, maintaining and governing registries is also called for. Collected data needs to be available to the benefit of public health and research and adaptable for regulatory purposes, including post-marketing surveillance of treatments and observational real life studies. Finally, registries and databases must be financially sustainable …”.

II – Australia

Prana Biotechnology presents a world first, patient-reported outcomes study for Huntington’s Disease (HD) in a poster presentation at the 17th Annual Congress of Parkinson’s Disease and Movement Disorders being held in Sydney. The poster presentation details a new benchmarking system to assess the problems experienced by people with HD. The full poster is available here.

Pharmaxis announces on June 20, 2013, that it has enrolled the first subject into its European pediatric clinical trial evaluating Bronchitol in Cystic Fibrosis. It is a Phase II trial being conducted in Europe and Canada.

The list of designated orphan drugs in Australia is updated as of June 3, 2013, on the Therapeutic Goods Administration (TGA).

III – United States

Raptor Pharmaceutical, a California-based biopharmaceutical company, announces on June 19, 2013, that orphan drug Procysbi (Cysteamine Bitartrate) is now available in the US. The FDA approves Procysbi in April 2013 for the management of nephropathic cystinosis in adults and children ages 6 and older. Nephropathic Cystinosis makes up “95% of Cystinosis cases and is a rare, life-threatening metabolic Lysosomal Storage Disorder (LSD) that causes toxic accumulation of cystine in all cells, tissues, and organs in the body.” Raptor Pharmaceutical anticipates receiving a determination from the European Medicines Agency (EMA) regarding its Procysbi marketing application in the European Union (EU) in the second half of 2013. There is approximately 500 patients in the US and 2,000 worldwide.

IV – Brazil

In March 2013, Brazilian regulators grant approval to Protalix BioTherapeutics’ Uplyso for treatment of Gaucher Disease. Protalix BioTherapeutics, an Israel-based company, announces on June 19, 2013, that the company reaches a supply and technology transfer deal with Fundação Oswaldo Cruz (commonly referred to as Fiocruz), an arm of the Brazilian Ministry of Health, for Uplyso, an Enzyme Replacement Therapy (ERT) for Gaucher Disease. Fiocruz must purchase at least $280 million of Uplyso before Protalix BioTherapeutics is required to complete the final stage of a technology transfer. The transfer is intended to enable the Brazilian government to construct its own manufacturing facility, which it will fund on its own, so it can produce its own supply of Uplyso. Under the agreement, which is expected to span seven years, Fiocruz has committed to purchase at least $40 million of Uplyso during the first two years of the pact. In later years, Fiocruz is required to make purchases of $40 million a year.

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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