orphan drugs
Global orphan and rare dermatology drugs market to grow rapidly, notes GBI Research
Rapid growth from $1.64 billion in 2017 to $6.07 billion in 2024 is expected in the global orphan and rare dermatology premium products market, according to business intelligence provider, GBI Research. more
A booming market — looking at the growth of the orphan drug market and its drivers
With worldwide orphan drug sales predicted to grow at a significant rate and each drug fetching a hefty price tag, I look at this market and its drivers in a bit more detail… more
CHMP adopts positive opinion for orphan drug to treat rare hereditary disease
Akcea Therapeutics, has announced that the Committee for Medicinal Products for Human Use (CHMP) has adopted a positive opinion, recommending the use of its orphan drug for the treatment of Stage 1 or Stage 2 polyneuropathy in adults with hereditary more
The top five tweets: From FutureLink to stem cell therapy — what did EPM readers click on this week?
Here we count down the top five tweets from @EPM_magazine that have gained the most impressions this week, taken from Twitter analytics. more
The availability of orphan medicines varies from country-to-country, says new European study
A new study from the University of Eastern Finland, published in the European Journal of Clinical Pharmacology, has highlighted that there are differences in the availability of orphan medicines between different European countries. more
Orphan drug designation received for acute myeloid leukaemia treatment
The US Food and Drug Administration (FDA) has granted orphan drug designation to SY-1425, which is an oral first-in-class selective retinoic acid receptor alpha (RARα) agonist for the treatment of acute myeloid leukaemia (AML) from Syros more
Brain cancer vaccine awarded orphan status by FDA
An immunotherapy developed at Roswell Park Cancer Institute has been granted orphan drug status by the US Food and Drug Administration (FDA). more
Plans unveiled to rid FDA of orphan designation backlog
The US Food and Drug Administration (FDA) has revealed its strategic plan to eliminate its orphan designation request backlog and to ensure there is continued timely response to new requests. more
PRO 140 potential too broad for orphan designation
The FDA has not granted orphan drug designation (ODD) to CytoDyn for its PRO 140 therapy as it appears to have the potential to treat more than just the subset of multi-drug resistant HIV patients for which the designation was requested. more
Yeliva receives FDA orphan drug designation
RedHill Biopharma has announced that its treatment for cholangiocarcinoma, Yeliva (ABC294640), has been granted orphan drug designation by the FDA. more
Six medicines recommended for approval at CHMP
During its March 2017 meeting, the European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) recommended six medicines for approval, including three orphans. more
Worldwide orphan drugs market set to reach $209bn by 2022
A new report by research group EvaluatePharma has highlighted the strength of the orphan drugs market. more
The good, the bad, and the Donald: Trump's implications on pharma
Orphan drugs, CMO continuous manufacturing and developing world sales offer biggest revenue opportunities, say the experts. more
Boyds joins team to develop orphan drug Trappsol Cyclo
Boyds has joined CTD Holdings’ International Clinical Program team, along with Aptus Clinical and KJC Statistics, to develop orphan drug Trappsol Cyclo with Accenture. more
