Orphan Drug
No slowing down: Orphan drug designations skyrocket in 2017
March 19, 2018The pace may even pick up again this year.
Did Congress just kill the Orphan Drug Act?
February 13, 2018By 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.
Five things for pharma marketers to know: Wednesday, January 3, 2018
January 03, 2018Utah 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
Five things for pharma marketers to know: Thursday, September 8, 2016
September 08, 2016A new study found that orphan drug spending will slow through 2018; senators open inquiry into Mylan; Shires launches branded campaign
My Orphan Drug Awakening
February 29, 2016One 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.
Top 25 orphan drugs, by 2015 global sales
February 22, 2016Roche's Rituxan generated $7.3 billion in sales in 2015, making it the top-selling orphan drug.
Five things for pharma marketers to know: Wednesday, July 8
July 08, 2015CDC 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
Five things for pharma marketers to know: Monday, April 6
April 06, 2015Pfizer 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 makes $1.1-billion rare-disease push
March 30, 2015Horizon Pharma's purchase of Hyperion Therapeutics will add two rare-disease drugs to its portfolio.
Shire and Cincinnati Children's Hospital team up in rare diseases
March 26, 2015The duo hope to begin testing a new treatment within three years.
Everyday Health buys rare-disease agency Cambridge BioMarketing
March 23, 2015The $38-million deal expands the reach of rare-disease campaigns.
Five things for pharma marketers to know: Tuesday, March 10
March 10, 2015HCV 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.
NPS drug approval bolsters Shire acquisition decision
January 26, 2015The 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.
Latest orphan-drug approval marks 15th this year
December 16, 2014Swiss drugmaker Novartis received an FDA nod for rare disease treatment Signifor LAR Tuesday, as orphan drug approvals reach new heights.
Five things for pharma marketers to know: Tuesday, December 16
December 16, 2014Bayer consolidates OTC creative portfolio; Genentech R&D head steps down; Novartis receives approval for orphan drug.
Five things for pharma marketers to know: Wednesday, November 5
November 05, 2014The 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.
Sanofi prices Cerdelga
September 03, 2014The $310,000 price for the Gaucher disease medication is typical of the rare-disease category.
Sanofi expands Gaucher disease portfolio
August 20, 2014The 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.
Amicus seeks to upset Fabry market
August 20, 2014Phase-III tests indicate patients may be able to switch from injectable enzyme-replacement therapies, like Fabrazyme, to the firm's oral drug.
AbbVie makes inroads in orphan drugs
August 13, 2014Not long after acquiring Shire to add to its orphan-drug portfolio, the drugmaker scored an orphan designation from both FDA and EMA last week.
Biotech Report: Ultra Man
August 01, 2014Innovation 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
BI drug gets priority review
July 02, 2014Boehringer Ingelheim's experimental idiopathic pulmonary fibrosis treatment nintedanib is also an orphan drug.
Boehringer drug lands US, EU orphan tag
April 17, 2014The experimental Breakthrough Therapy treatment is for acute myeloid leukemia.
Amgen's PSCK9 shows rare disease potential
March 17, 2014The Thousand Oaks, Calif. company says its cholesterol-lowering antibody hit its clinical trial endpoint among rare disease patients.
Sanofi seeks to revive Kynamro launch
March 03, 2014Kynamro's slow start, coupled with competition, has prompted Sanofi to add sales reps.
Rare diseases may be less so
January 16, 2014Regulatory Focus discusses how the expanding US population may make it harder for a condition to qualify as rare.
Dept. of Justice subpoenas Aegerion
January 13, 2014The drugmaker announced Friday that the DOJ wants documents regarding sales and marketing of Juxtapid, its drug for an ultra-rare lipid disorder.
FDA pushes review date for Biogen blood drug
December 02, 2013The agency extended the review time for Biogen's Alprolix by three months.
IMS predicts $1T global drug market by 2017
November 19, 2013The catch is the difference between sales and revenues. Generics will continue to be a force, and small-audience drugs will become increasingly important.
Novartis breakthough designation hat trick
August 20, 2013The drug maker scored its third breakthrough designation label, this time for orphan drug bimagrumab, a possible treatment for a muscle-wasting disease.
