Orphan Drug

Latest orphan-drug approval marks 15th this year

Latest orphan-drug approval marks 15th this year

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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

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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

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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

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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

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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

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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

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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

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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

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The experimental Breakthrough Therapy treatment is for acute myeloid leukemia.

Amgen's PSCK9 shows rare disease potential

Amgen's PSCK9 shows rare disease potential

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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

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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

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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

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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

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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

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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

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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

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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

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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

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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.

Company news: Corcept Therapeutics and Daiichi Sankyo

Corcept Therapeutics said its Cushing's syndrome drug will be launched three weeks earlier than expected.

Shire exits Fabry's market, citing costs as FDA weighs more research

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Shire has pulled its drug, Replagal, from FDA review. The company was set to go before the agency next month, but preliminary talks indicated the FDA was going to require more testing.

Company news: ViroPharma and DIME

Specialty drug company ViroPharma teamed with several medical societies on the launch of a program designed to unite specialists treating patients with the orphan condition hereditary angioedema (HAE).


Does a health psychology approach hold the key to Rx adherence? In MM&M's latest Leadership Exchange Uncut eBook, industry stakeholders from the payer, provider, academic and pharma realms explore the "why" behind medicine taking. Access here.

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