Orphan diseases companies

By Olga Oksman

Trying to find the next hot space in pharmaceutical M&A is like shooting at a moving target. Or perhaps more aptly, a bobbing and weaving target.

One area getting a lot of attention is the orphan drug space, industry bankers tell us. It’s a simple value proposition for big pharmaceutical companies: firms with one or two products that can be easily plugged into your existing offerings. Just in 2011, Sanofi acquired one of the largest orphan drug makers, Genzyme for an over $20 billion sale price.

Orphan drugs, which treat rare conditions, don’t require a large sales force. By contrast, cholesterol or diabetes drugs, for example, need an untold number of sales reps to onboard every Tom, Dick and Harriet providing primary care and endocrinology services.

Instead, orphan drugs often have patients seeking providers, looking for options and raising awareness of companies’ drugs through rare disease foundations.

The rates of success for drug development are about the same as a regular drug for common disorders. But because of the rarity of the conditions they treat, companies making orphan drugs get certain perks from the government to make it worthwhile to develop them, like seven years of market exclusivity in the US and ten years in Europe.

And then there is the pricing. Oh the pricing.

The most cited example is probably Alexion, a $19 billion company built on a treatment called Soliris for two rare blood disorders. That’s right, $19 billion. Which is what happens when you market a drug that costs about $409, 500 per patient, per year.

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Popular Q&A

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What does the FDA do? a topic, disease, or research study that the FDA is involved with and describe it?

what does the FDA do? a topic, disease, or research study that the FDA is involved with and describe it’s involvement?

The FDA takes possible cures to diseases and slows down the process of making it legal to buy and use to fix your ailments. They do this because of all the donated money to help finding a cure (for example cancer, or aids) If someone were to find a cure for a disease then they would stop getting the donation money. Theres more money to be made in finding cures rather then creating a cure. FACT

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