Rare Diseases

Shire, Salix buy way to expansion

The companies are plunking down substantial sums to increase their reach.

Analyst says smart money is on drugs for certain orphan diseases

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Price, market potential and emotional pull make this subcategory of orphan diseases one to watch, an analyst writes. Hint: it's not oncology.

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.

Company news: smartphones, Janssen, FDA

Smartphones will influence 19% of in-store sales in four years, Janssen acquires an experimental heart treatment, and the FDA can now fast-track rare disease medications.

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.

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MM&M EBOOK: PATIENT ACCESS

Patient access to pharmaceuticals is a tale of two worlds—affordability has improved for the majority, while the minority is hampered by cost, distribution and red tape. To provide marketers with a well-rounded perspective, MM&M presents this e-book chock full of key insights. Click here to access it.