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.

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: 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).

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