Whitepapers
What does the new FDA accelerated regulatory pathway mean for gene therapies?
U.S. Food and Drug Administration (FDA) commissioner Scott Gottlieb has announced that the drug agency is developing a system by which innovative gene therapies could
Biopharma 2018 Success Kit: 11 months to go, 6 rare disease trends to watch!
For the rapidly developing orphan drug market, change is a constant. From new technologies to discussions over pricing, the estimated $137 billion market will witness
ICER finds Roche’s Hemlibra cost-effective despite high price tag
Roche’s new haemophilia treatment, Hemlibra, is has a high estimated price tag of $482,000 for the first year of treatment and $448,000 for every subsequent
Sanofi to buy Bioverativ in $11.6 billion deal
French pharmaceutical giant Sanofi have agreed to acquire Bioverativ, a U.S. hemophilia specialist for $11.6 billion. This deal is the biggest for Sanofi in seven
UK regulator attempts to ease fears of impending access abyss
The UK’s Medicines and Healthcare Products Regulatory Agency (MHRA) announced that there would be no sudden changes to the UK regulatory framework in the event
Generation Bio pioneers revolutionary gene therapy delivery system
The biotech venture capital firm Atlas Ventures has announced the creation of Generation Bio, a company that has developed a system by which the dosage
Spark Therapeutics Announces $850 000 price tag of gene therapy treatment Luxturna
Spark Therapeutics has announced its recently approved Luxturna (voretigene neparvovec-rzyl), a gene therapy treatment for biallelic RPE65 mutation-associated retinal dystrophy will cost $425 000 per
FDA approves gene therapy for rare form of vision loss
The U.S. Food and Drug Administration (FDA) has approved the use of Spark Therapeutics’ gene therapy Luxturna (voretigene neparvovec-rzyl) for the treatment of a rare
FDA issues new draft guidance for Orphan Drug development
The U.S. Food and Drug Administration (FDA) published its recommendation for a new approach to drug development for rare pediatric diseases. The new approach supports
