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.
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.
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.
FDA's afatinib approval enables the drug maker set up shop in a new therapy space.
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.
Why executives evaluating biotech acquisitions shouldn't overlook the cultural dimension: A new paper finds a link to revenue performance.
Aegerion's Juxtapid is first to market for treating the ultrarare inherited cholesterol condition HoFH, or homozygous familial hypercholesterolemia.
Notes to the advisory committee indicate that recycled data has perceived weak spots.
Corcept Therapeutics said its Cushing's syndrome drug will be launched three weeks earlier than expected.
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.
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).