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.
Swiss drugmaker Novartis received an FDA nod for rare disease treatment Signifor LAR Tuesday, as orphan drug approvals reach new heights.
Bayer consolidates OTC creative portfolio; Genentech R&D head steps down; Novartis receives approval for orphan drug.
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.
The $310,000 price for the Gaucher disease medication is typical of the rare-disease category.
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.
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.
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
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.
Kynamro's slow start, coupled with competition, has prompted Sanofi to add sales reps.
Regulatory Focus discusses how the expanding US population may make it harder for a condition to qualify as rare.
The drugmaker announced Friday that the DOJ wants documents regarding sales and marketing of Juxtapid, its drug for an ultra-rare lipid disorder.
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.
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.
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).
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