By allowing companies to claim a 50% tax credit for qualified clinical testing expenses, developing drugs for rare diseases became economically feasible for the first time.
Utah Senator who backed Orphan Drug Act will not seek reelection; medicines more expensive in 2018; Spark's genetic therapy for rare blindness costs $850,000
A new study found that orphan drug spending will slow through 2018; senators open inquiry into Mylan; Shires launches branded campaign
One conversation with a real patient—followed by many more conversations with more patients—helped me see how important our work can be in the eyes of patients.
Roche's Rituxan generated $7.3 billion in sales in 2015, making it the top-selling orphan drug.
CDC report finds prescription painkiller abuse contributed to an increase in heroin use; Novartis heart-failure drug gets FDA approval; Isis Pharmaceuticals obtains orphan-drug designation for volanesorsen
Pfizer exec said biosimilar interchangeability definition not happening anytime soon; AARP launches digital health tech studies with Pfizer and UnitedHealthcare; BMS inks gene therapy collaboration deal
Horizon Pharma's purchase of Hyperion Therapeutics will add two rare-disease drugs to its portfolio.
The duo hope to begin testing a new treatment within three years.
The $38-million deal expands the reach of rare-disease campaigns.
HCV drugs and other specialty medications drove a 13% increase in last year's prescription spending, the CBO overestimated just how much healthcare reform would cost, and an FDA panel voted 17-0 in favor of Kythera's drug that can reduce double chins.
The FDA approved NPS Pharmaceuticals's rare-disease drug Friday. The company, which Shire shelled out $5.2 billion to buy, expects it to hit the market during the second-quarter.
Swiss drugmaker Novartis received an FDA nod for rare disease treatment Signifor LAR Tuesday, as orphan drug approvals reach new heights.
Bayer consolidates OTC creative portfolio; Genentech R&D head steps down; Novartis receives approval for orphan drug.
The Hill outlines its healthcare expectations with a GOP Congress, Sanofi's executive shakeup may have an impact on MannKind's Afrezza launch, AstraZeneca's olaparib may have prostate cancer prospects, the FDA granted orphan status to an experimental Merrimack pancreatic cancer drug, and researchers are struggling to get Ebola virus samples.
The $310,000 price for the Gaucher disease medication is typical of the rare-disease category.
The oral medication Cerdelga joins Cerezyme in its Gaucher disease arsenal. Sanofi expects the drug will be priced "on par" with Cerezyme, which goes for around $300,000 a year in the US.
Phase-III tests indicate patients may be able to switch from injectable enzyme-replacement therapies, like Fabrazyme, to the firm's oral drug.
Not long after acquiring Shire to add to its orphan-drug portfolio, the drugmaker scored an orphan designation from both FDA and EMA last week.
Innovation is still valued in the orphan-drug space, but the reimbursement bar is rising. NPS Pharma's Eric Pauwels, who's launched five ultra-rare disease products, explains how these biotech brands can demonstrate their value. Marc Iskowitz reports
Boehringer Ingelheim's experimental idiopathic pulmonary fibrosis treatment nintedanib is also an orphan drug.
The experimental Breakthrough Therapy treatment is for acute myeloid leukemia.
The Thousand Oaks, Calif. company says its cholesterol-lowering antibody hit its clinical trial endpoint among rare disease patients.
Kynamro's slow start, coupled with competition, has prompted Sanofi to add sales reps.
Regulatory Focus discusses how the expanding US population may make it harder for a condition to qualify as rare.
The drugmaker announced Friday that the DOJ wants documents regarding sales and marketing of Juxtapid, its drug for an ultra-rare lipid disorder.
The agency extended the review time for Biogen's Alprolix by three months.
The catch is the difference between sales and revenues. Generics will continue to be a force, and small-audience drugs will become increasingly important.
The drug maker scored its third breakthrough designation label, this time for orphan drug bimagrumab, a possible treatment for a muscle-wasting disease.
Early sales of NPS's Gattex have soared, while those of Protalix's Elelyso have been sluggish, underscoring the challenges of rare-disease launches.