Orphan Drug Developers Dominate the Top 25 Biotech Companies of 2013

In Clinical Development, Partnering & Investment by Matt Turner

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So the annual list of Top 25 Biotech Companies, as published by GEN, has been released. The list is ranked on their market capitalization for the latest quarter that they furnished figures on their numbers of outstanding shares.

See the full list and article here.

Interestingly, of the top 25 biotech companies that made this year’s list, there was a strong representation of companies dedicated to developing innovative treatments for rare diseases.

Below are a selection from the top 25 who are pioneering the development of orphan drugs to the rare disease patient market:

#25 – Isis Pharmaceuticals

2013 Market Cap: $2.324 billion (5/2: 103,780,421 shares * price $22.39)

Isis Pharmaceuticals’ orphan drug Kynamro (in collaboration with Genzyme) was approved on January 29, 2013, for the treatment of Homozygous Familial Hypercholesterolemia (HoFH). Kynamro is Isis Pharmaceuticals first drug to make it to the market.

#17 – Onyx Pharmaceuticals

2013 Market Cap: $6.580 billion (5/3: 72,738,263 shares * price $90.46)

Based in South San Francisco, California, Onyx Pharmaceuticals, Inc. is a global biopharmaceutical company engaged in the development and commercialization of innovative therapies for improving the lives of people with cancer.

#14 – BioMarin Pharmaceuticals

2013 Market Cap: $9.097 billion (4/19: 139,010,175 shares * price $65.44)

With four products on the market and a fully-integrated multinational organization in place, BioMarin is providing innovative therapeutics to patients with serious unmet medical needs.
#12 – Vertex Pharmaceuticals

2013 Market Cap: $17.495 billion (4/26: 221,400,864 shares * price $79.02)

In 2012, Vertex Pharmaceuticals (VRTX) received approval from The U.S. Food and Drug Administration for their drug Kalydeco, the first therapeutic drug to treat an underlying cause of cystic fibrosis. Kaydeco targets a genetic mutation affecting a small minority of patients suffering from the life-threatening breathing disorder.

#11 – Shire

2013 Market Cap: $17.252 billion (4/29: 562,826,237 shares * price £20.17)

Shire pursue opportunities on behalf of patients and families facing such rare diseases as Fabry disease, Hunter syndrome, Gaucher disease, hereditary angioedema (HAE), Duchenne Muscular Dystrophy and Sanfillipo A + B — patients whose lives often hinge on the discovery and delivery of treatments.

#10 – Alexion Pharmaceuticals

2013 Market Cap: $17.893 billion (4/23: 195,146,634 shares * price $91.69)

Soliris® (eculizumab), is a first-in-class terminal complement inhibitor discovered, developed and commercialized by Alexion. Soliris is approved for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH)1, a progressive and life-threatening disease characterized by the excessive destruction of red blood cells (hemolysis).1,2,3 Soliris is the first and only therapy approved for the treatment of PNH to reduce hemolysis.

#4 – Celgene

2013 Market Cap: $52.557 billion (4/23: 417,122,477 shares * price $126.00)

In February 2013, Celgene Corporation was been awarded the prestigious EURORDIS Company Award for excellence in the field of rare diseases.

See the full article and Top 25 list here.

The orphan drug sector continues to grow with opportunities aplenty. These opportunities are being taken and business is being done at the World Orphan Drug Congress, Geneva. It is a privilege to have Alexion Pharmaceuticals, Shire & Celgene sponsoring the congress in 2013 and we look forward hearing the leading biotech companies present best practice for orphan drug developers across an interactive two days.

Download the brochure for the World Orphan Drug Congress Europe 2013.