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Orphan Drugs And Rare Diseases: Global News (05.08.13)

May 8, 2013

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

I – Europe

An European Medicines Agency’s (EMA) panel recommends orphan drug Revlimid for a new indication – anemia in patients with blood disorders called Myelodysplastic Syndromes (MDS). The EU is expected to make a final decision within three months. Revlimid is already approved in the US for this indication.

EURORDIS (Rare Diseases Europe) announces that RareConnect, an online global patient network, welcomes its 40th rare disease community member. RareConnect “brings together thousands of patients, families, and groups who might otherwise be isolated …. Can communicate, sharing experiences and information in a safe, moderated online forum.” The 40th rare disease community added to RareConnect, is for people caring for patients with rare undiagnosed brain disorders.

PharmaMar, headquartered in Madrid, Spain, announces the completion of enrollment for a Phase IIb trial for orphan drug Lurbinectedin (PM01183), for patients with resistant/refractory ovarian cancer. The trial results will be presented at a major medical conference in 2013, “potentially leading to the launch of a Phase III trial, upon agreement after discussions with the EMA and the FDA.”  PharmaMar in August 2012, receives FDA Orphan Drug Designation (ODD) and in October 2012, EMA grants orphan designation.

Eisai’s orphan drug Lenvatinib receives orphan drug designation from the EU for follicular and papillary thyroid cancer.

II – Mexico/Latin America

Protalix BioTherapeutics’ orphan drug Uplyso (Elelyso outside of Latin America) receives regulatory approval for Gaucher Disease from the :

•   Mexican Federal Commission for the Protection against Sanitary Risk (COFEPRIS)
•   Public Health Institute of Chile.


It has been a busy week for FDA regulatory actions.

First, the FDA approves Raptor Pharmaceutical’s orphan drug Procysbi (Cysteamine Bitartrate) for rare disease Nephropathic Cystinosis.

Second, the FDA grants several FDA Breakthrough Designations to :

•   Bristol-Meyers Squibb for an experimental, non-orphan triple Direct-Acting Antiviral (DAA) combo for Hepatitis C
•   Scioderm for an investigational topical cream, SD-101, for inherited Epidermolysis Bullosa (EB)
•   Janssen Pharmaceutical for non-orphan drug Daratumumab for Multiple Myeloma (MM)
•   AbbVie for non-orphan, all-oral triple Direct-Acting Antiviral (DAA) combination for Hepatitis C.

IV – India

An article from, a New Delhi Television news station, has a fascinating article about a Mumbai teen, Ashley Vanristell, who has a rare disease called Primary Immuno Deficiency (PID). For the past five years, Ashley Vanristell has been running a foundation, Ashley Hypogammaglobulinemia Foundation, that he established to support children with PID. His foundation raises money for the treatment of kids whose families cannot afford the treatment IVIG (IntraVenous ImmunoGlobulin).

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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