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Thursday 21 March 2019
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Topic: orphan drug

October 20, 2016
Orphan Drug Designation for Abeona's lead gene therapy program ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA)

Abeona Therapeutics Inc. a clinical- stage biopharmaceutical company focused on developing gene therapies for life-threatening rare diseases, announced that the European Medicines Agency (EMA) Committee for Orphan Medicinal Products has granted Orphan Drug Designation for Abeona's lead gene therapy program ABO-102 for the treatment of patients with Sanfilippo syndrome type A (MPS IIIA), a rare autosomal recessive disease that causes neurocognitive decline, speech loss, loss of mobility, and premature death in children.

 

September 12, 2016
Lung involvement, such as interstitial lung disease, is the leading cause of death among people with systemic sclerosis

Boehringer Ingelheim has announced that the European Commission (EC) and US Food and Drug Administration (FDA) have granted Orphan Drug Designation to nintedanib for the treatment of systemic sclerosis (SSc, also known as scleroderma), including the associated interstitial lung disease (SSc-ILD).

 

December 18, 2015
Pentosan polysulphate has been awarded the status of an orphan drug from the EMA for the indication of interstitial cystitis

In European and US medical guidelines, pentosan polysulphate is established as a standard drug for the treatment of the severe chronic bladder disease interstitial cystitis (IC). Now the drug has been awarded the status of an orphan drug from the European Medicines Agency, EMA, for the indication of IC.  

December 3, 2015
The drug, discovered and developed in Italy, is being studied in patients with neurotrophic keratitis, a pathology still lacking an effective treatment that affects fewer than five out of 10,000 individuals
April 16, 2008
The FDA grants Graft versus Host Disease drug orphan drug status

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February 29, 2008
Drug firm EpiCept has been dealt a blow in its efforts to get a marketing authorisation application (MAA) for Ceplene

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