It has become one of the biggest biotech firms thanks to a series of rare diseases.
The decision is a setback, of course, given that Shire has emphasized treating rare diseases.
Cancer was the leading therapeutic area with 12 approvals, followed by rare diseases with 6.
This state of affairs often leaves those suffering from rare diseases with few treatment options.
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New medicines for cancer and rare diseases are still being reimbursed at very high prices.
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Studies on dog genetics are also useful for studying rare diseases in humans, the researchers wrote.
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In 1983 the U.S. enacted a law giving special protection from competition for drugs for rare diseases.
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Glaxo will also work with the Italian researchers to develop the gene therapy for six other rare diseases.
The Orphan Drug Act was passed only because of the support of the National Organization of Rare Diseases.
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Importantly, early success in relatively rare diseases seems to be translating into much broader potential for these medicines.
Patients' families with other rare diseases also are seeking to press researchers to share data from natural-history studies.
Big drug companies take little interest in rare diseases (those that affect fewer than one in 2, 000 people).
And as Genzyme's Mr Tambuyzer notes, rare diseases become less rare once a drug is on the market.
Having said all that, we do need to find ways to stimulate the development of treatments for rare diseases.
Without this drug, the costs of caring for people with rare diseases like this can run into the millions.
The drug industry is currently trying out 300-plus drugs for rare diseases, more than double the number in 1989.
Moreover, repositioning such drugs to treat other rare diseases can ring more registers.
Legislation is expected to double the number of drugs now available to treat 5, 000 rare diseases affecting 10m Europeans.
But while the Orphan Drug Act increased the amount of research into rare diseases, the field is still vastly underfunded.
Of course, many patients with rare diseases actually receive their medicines for free if insurers or governments cannot pay for them.
And when new or rare diseases are found, doctors worldwide turn to the CDC to figure out which microorganisms they are battling.
By airing two episodes on the need for treatments for people with rare diseases, he addressed a very important public health challenge.
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The Orphan Drug Act, enacted in 1982, provides incentives--including tax credits and exemption from user fees--for the development of drugs for rare diseases.
Such super-high-priced drugs for ultra-rare diseases are the biggest success story in pharma right now (see BioMarin and even Genzyme for other examples).
But because the number of people with rare diseases, like Sanfilippo, is so small, patients may be needed to participate in multiple studies.
There was a time not long ago when patients suffering from rare diseases had little hope of finding a medicine that could cure their ills.
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So the overall market for treatments for rare diseases is large.
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Shire is a pharmaceutical company that develops health care in the areas of behavior health and gastrointestinal conditions, rare diseases and regenerative medicine and other conditions.
More than 6, 000 of these disorders are rare diseases, most of them caused by a single gene, and fewer than 200 have a drug treatment available.
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