Drug Development Pharma - July 1, 2015
EC Approves Sobi Drug for HT-1
The European Commission has approved an oral suspension formulation of Swedish Orphan Biovitrum’s (Sobi) Orfadin (nitisinone). The orphan drug treats Hereditary Tyrosinaemia type-1 (HT-1), a rare genetic and potentially fatal condition that affects infants and children and can result in liver and kidney failure if untreated, according to PMLiVE. “The European Medicines Agency has called […]
Business article - June 2, 2015
Orphan Drugs Spur M&A Interest
Thanks to the potential for higher prices, limited competition and favorable regulations, orphan drugs continue to be popular to manufacture and also attractive to buy, and Moody’s predicts they will continue to generate merger and acquistion activity. Nearly half of recent acquisitons involved orphan drugs, or medications that treat serious conditions that affect fewer than […]
Drug Development Pharma - March 4, 2015
Progress on Drug for Orphan Disease
Orphazyme, a Danish biotech company working on the development of orphan drugs, recently completed a new series B capital round of 20 million Euro. Kurma Partners and Idinvest Partners joined the existing investor syndicate of Novo A/S, Sunstone Capital and Aescap Venture. The company is working on Arimoclomol, the leading drug candidate for the treatment […]
Drug Development Pharma - January 12, 2015
2014 Year of Orphan Drugs
The European Medicines Agency (EMA) recommended the highest number of orphan designated medicines for marketing authorization in a single year in 2014. Out of the 82 medicines for human use recommended last year, 17 are meant for the treatment of a rare disease, serving patients who frequently have a few or no treatment options. Among […]
Drug Development Pharma - November 20, 2014
BerGenBio Drug Gets Orphan-Drug Status
BerGenBio AS, a Norwegian oncology biopharmaceutical company, announced Wednesday that the US Food and Drug Administration (FDA) granted orphan-drug designation for BGB324 for treatment of acute myeloid leukemia (AML). Earlier this month BerGenBio announced that the first patient was dosed in its multi-center Phase 1b trial of BGB324, a selective inhibitor of Axl, in patients […]
Pharma Business - November 4, 2014
Orphan Drugs’ Market Share to Increase
Interest in developing drugs for orphan diseases is expected to grow into the next decade, leading to orphan drugs making up 19 percent of all prescription sales by 2020, according to some estimates. Sales also could increase at a rate of almost 11 percent per year through the end of the decade, almost three times […]
