Orphan Drug

Five things for pharma marketers to know: Monday, April 6

Five things for pharma marketers to know: Monday, April 6

By

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 makes $1.1-billion rare-disease push

Horizon makes $1.1-billion rare-disease push

By

Horizon 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

Shire and Cincinnati Children's Hospital team up in rare diseases

By

The duo hope to begin testing a new treatment within three years.

Everyday Health buys rare-disease agency Cambridge BioMarketing

Everyday Health buys rare-disease agency Cambridge BioMarketing

By

The $38-million deal expands the reach of rare-disease campaigns.

Five things for pharma marketers to know: Tuesday, March 10

Five things for pharma marketers to know: Tuesday, March 10

By

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.

NPS drug approval bolsters Shire acquisition decision

NPS drug approval bolsters Shire acquisition decision

By

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.

Latest orphan-drug approval marks 15th this year

Latest orphan-drug approval marks 15th this year

By

Swiss 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

Five things for pharma marketers to know: Tuesday, December 16

By By

Bayer 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

Five things for pharma marketers to know: Wednesday, November 5

By By

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.

Sanofi prices Cerdelga

Sanofi prices Cerdelga

By

The $310,000 price for the Gaucher disease medication is typical of the rare-disease category.

Sanofi expands Gaucher disease portfolio

Sanofi expands Gaucher disease portfolio

By

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.

Amicus seeks to upset Fabry market

Amicus seeks to upset Fabry market

By

Phase-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

AbbVie makes inroads in orphan drugs

By

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.

Biotech Report: Ultra Man

Biotech Report: Ultra Man

By

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

BI drug gets priority review

Boehringer Ingelheim's experimental idiopathic pulmonary fibrosis treatment nintedanib is also an orphan drug.

Boehringer drug lands US, EU orphan tag

Boehringer drug lands US, EU orphan tag

By

The experimental Breakthrough Therapy treatment is for acute myeloid leukemia.

Amgen's PSCK9 shows rare disease potential

Amgen's PSCK9 shows rare disease potential

By

The Thousand Oaks, Calif. company says its cholesterol-lowering antibody hit its clinical trial endpoint among rare disease patients.

Sanofi seeks to revive Kynamro launch

Sanofi seeks to revive Kynamro launch

By

Kynamro's slow start, coupled with competition, has prompted Sanofi to add sales reps.

Rare diseases may be less so

Regulatory Focus discusses how the expanding US population may make it harder for a condition to qualify as rare.

Dept. of Justice subpoenas Aegerion

Dept. of Justice subpoenas Aegerion

By

The 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

The agency extended the review time for Biogen's Alprolix by three months.

IMS predicts $1T global drug market by 2017

IMS predicts $1T global drug market by 2017

By

The 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

By

The drug maker scored its third breakthrough designation label, this time for orphan drug bimagrumab, a possible treatment for a muscle-wasting disease.

Mixed results underscore orphan launch hurdles

By

Early sales of NPS's Gattex have soared, while those of Protalix's Elelyso have been sluggish, underscoring the challenges of rare-disease launches.

Approval clears Boehringer to enter oncology space

By

FDA's afatinib approval enables the drug maker set up shop in a new therapy space.

Business briefs: Lilly, Novartis, Activartis, plus policy news

Eli Lilly and Boehringer Ingelheim submit a glargine biosimilar to the EMA; Novartis pipeline claims an Enbrel-superior treatment; Activartis med gets orphan drug status; generics makers may have to pay more attention to labels; Administration tweaks healthcare reform implementation over the long weekend.

Culture of innovation has a sales value, study says

Culture of innovation has a sales value, study says

By

Why executives evaluating biotech acquisitions shouldn't overlook the cultural dimension: A new paper finds a link to revenue performance.

Drug for ultrarare lipid disorder OK'd by FDA

Drug for ultrarare lipid disorder OK'd by FDA

By

Aegerion's Juxtapid is first to market for treating the ultrarare inherited cholesterol condition HoFH, or homozygous familial hypercholesterolemia.

FDA committee set to review chronic fatigue drug

By

Notes to the advisory committee indicate that recycled data has perceived weak spots.

Company news: Burson-Marsteller, Corcept and Orexigen

PR firm Burson-Marsteller launched a new healthcare subsidiary, Orexigen's net loss widened in Q2, and Corcept closed out its first full quarter with orphan treatment Korlym.


A wave of more effective anti-cancer drugs has set the oncology world on fire with enthusiasm. While many hail this as a new era, an equally vocal faction questions the money spent for the value gained. This medical and commercial trend report for marketers of anti-cancer modalities touches on many of the latest shifts that have expedited product launches and otherwise impacted promotion and reimbursement of these drugs. Click here.

Email Newsletters


Pharma continues to struggle with its image. Exhibit A is the public debate around the price of, and access to, new and innovative meds. As countless transformative technologies hit market, has industry done enough to anchor its permission to innovate? To provide a closer look at trends affecting trust over the past year, MM&M presents this e-book, "The 2015 Edelman Trust Barometer." Click here.