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.
FORBES: To Cure Rare Diseases, Unleash Orphan Drug Innovations
New medicines for cancer and rare diseases are still being reimbursed at very high prices.
FORBES: A Note to The Pharmaceutical Industry Upon The Re-Election Of Barack Obama
Studies on dog genetics are also useful for studying rare diseases in humans, the researchers wrote.
MSN: Cause of rare and fatal dog skeletal disease pinpointed
In 1983 the U.S. enacted a law giving special protection from competition for drugs for rare diseases.
FORBES: How A $440,000 Drug Is Turning Alexion Into Biotech's New Innovation Powerhouse
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.
FORBES: A Dying Cancer Patient's Plea For Personalized Medicine
Importantly, early success in relatively rare diseases seems to be translating into much broader potential for these medicines.
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.
Of course, many patients with rare diseases actually receive their medicines for free if insurers or governments cannot pay for them.
By airing two episodes on the need for treatments for people with rare diseases, he addressed a very important public health challenge.
FORBES: How The Odd Couple's Jack Klugman Helped Create The Biotech Industry
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).
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.
FORBES: The Lifesaving Promise Of The Pharmaceutical Drug Pipeline
So the overall market for treatments for rare diseases is large.
FORBES: To Cure Rare Diseases, Unleash Orphan Drug Innovations
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.
应用推荐