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Orphan Drugs: In The News (03.19.13)

March 19, 2013

The following are recent orphan drug and orphan drug company developments in the news.


In January 2013, Pharmaxis receives a unanimous negative recommendation from the FDA Pulmonary-Allergy Drugs Advisory Committee (PADAC) Meeting for orphan drug Bronchitol (Mannitol) for the treatment of Cystic Fibrosis (CF).

The FDA on March 18, 2013, concludes its review of the Bronchitol New Drug Application (NDA) and recommends that Pharmaxis should conduct an additional clinical trial to obtain approval. Gary Phillips, Pharmaxis CEO, says that “We are clearly disappointed that Bronchitol cannot yet be made available to patients in the US. The FDA has provided guidance on the necessary measures to gain approval and Pharmaxis will now have a follow-up meeting with the FDA. This will be a Type A meeting which I expect will take place next quarter ….”.

Bronchitol has Orphan Drug Designation in the US, Europe, and Australia. It is approved for marketing in Australia and throughout the European Union. Bronchitol was discovered and developed in Australia and is currently on the list of Australia’s Pharmaceutical Benefits Scheme (PBS) for CF.

Protalix BioTherapeutics

Protalix BioTherapeutics and Pfizer announces March 18, 2013, that Brazil’s National Health Surveillance Agency, ANVISA (Agencia Nacional de Vigilancia Sanitaria), gives regulatory approval to Uplyso (known as Elelyso outside of Latin America) for Type I Gaucher Disease in adults.  

Uplyso/Elelyso (Taliglucerase Alfa) has the following regulatory history :

  1. FDA approval in May 2012
  2. Israel’s Ministry of Health approval in September 2012
  3. Uruguay’s Ministry of Public Health approval in November 2012
  4. EC adopts EMA’s recommendation against approval in 2012 due to Shire plc’s orphan drug exclusivity for Vpriv.

NPS Pharmaceuticals

On March 19, 2013, NPS Pharmaceuticals and Takeda Pharmaceutical announces that NPS Pharmaceuticals has re-gained the full worldwide rights to orphan drug Gattex (Revestive in Europe) and recombinant human ParaThyroid Hormone 1-84 (PTH 1-84). NPS Pharmaceuticals will now have worldwide rights to develop and commercialize both products.

Takeda Pharmaceutical will assign assets related to both products (active pharmaceutical ingredient inventory and all information related to the drug development life cycle). Takeda Pharmaceutical will receive NPS Pharmaceuticals common stock valued at $50 million. Also, Takeda Pharmaceutical will receive a milestone payment, if combined worldwide net sales of both products exceed $750 million in the first year.

Francois Nader, M.D., President and CEO of NPS Pharmaceuticals, says that, “This transaction establishes NPS as a global commercial rare disease company. Our primary focus is to secure reimbursement for Revestive in Europe … 2013 priorities remain the successful US launch of Gattex and the submission of our US marketing application for Natpara.”

This new agreement will give NPS Pharmaceuticals an opportunity to establish a worldwide orphan drug franchise.

Please Note: “RSS” by Abzt at de.wikipedia (Transferred from de.wikipedia) [GPL] | Wikimedia Commons.

Copyright © 2012-2013, Orphan Druganaut Blog. All rights reserved.

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