Dutch firm UniQure finally brings to market their ground breaking lipoprotein lipase deficiency gene therapy in Germany with partner Chiesi After receiving approval for their …
Extra 6 Months Market Exclusivity for Drugs Re-purposed for Orphan Indications
A bill submitted to the US House of Representatives would grant an extra six months of market exclusivity to products that are re-purposed to treat …
EMA Orphan Drug Designations – November 2014
See the FDA Orphan Drug designation for October here. EMA Drug: Mucopolysaccharidosis type IIIA (Sanfilippo A syndrome Orphan Designation: Adeno-associated viral vector serotype 10 carrying …
Rare Disease Lobbying Works, but Is It Worth it?
View PostShould We Be Worried About Pricing, Pipeline, or Policy?
If pricing isn’t going to be an issue in the next decade, then should we not be concerned with our development pipeline? Pricing, and the sustainability of high cost …
FDA Orphan Drug Designations – October 2014
Find the EMA’s orphan designation and approvals for October here. FDA – 10/01/2014 Generic Name: PEGylated recombinant human hyaluronidase PH20 Trade Name: N/A Orphan Designation: Treatment of pancreatic …
EMA Orphan Drug Designations – October 2014
Find the EMA’s orphan designation and approvals for September here. EMA – 09/10/2014 Drug: 1-(6-benzothiazolylsulfonyl)-5-chloro-1H-indole-2-butanoic acid Orphan Designation: Systemic Sclerosis Sponsor: Inventiva EMA – 09/10/2014 Drug: …
A Note To Techdirt and The Street: Read Up on the Orphan Drug Act
Catalyst Pharma Accused of ‘Abusing’ the Orphan Drug System Abuse is the improper usage or treatment of an entity. Techdirt put out an article this week which highlights what …