November 10, 2015
By Alex Keown, BioSpace.com Breaking News Staff
BOSTON – Massachusetts is without a doubt the driving hub of innovation in the orphan disease market and a recent survey conducted for the Boston Business Journal reveals how the Bay State is leading the way in marketing and developing treatments for orphan diseases.
The data collected by Evaluate Pharma for the Journal surveyed the makers of drugs used to treat less than 200,000 patients in the United States and determined that 12 of the 25 biggest approved and late-stage development drugs for rare and orphan diseases are made by Massachusetts-based companies, such as Vertex Pharmaceuticals and Biogen, Inc. . The Journal includes a drug from Shire Pharmaceuticals and Genzyme Corporation in the Massachusetts’ total because the bulk of the work done by those two companies are conducted in Massachusetts, even though their headquarters are elsewhere. The 12 drugs made in Massachusetts, which includes those in late-stage development that have yet to receive regulatory approval, are estimated to bring in combined revenue of $8.5 billion by 2020.
The top 10 orphan disease drugs in Massachusetts below include makers and estimated revenue by 2020:
1. Vertex Pharmaceuticals
Orkambi for cystic fibrosis, which was approved earlier this year, is expected to generate $2.8 billion by 2020. Analysts predict Orkambi will push Vertex to profitability, in large part because of the reach the drug is expected to reach a broader number of cystic fibrosis patients than its other blockbuster drug Kalydeco, which is number five on the list. Orkambi is the combination of Kalydeco and lumacaftor, which will be used to treat patients with the F508del mutation of cystic fibrosis, a mutation the lead drug cannot treat on its own. The F508del mutation is the most common genetic mutation in cystic fibrosis.
2. Biogen
Avonex for multiple sclerosis is expected to generate $1 billion in revenue.
3. Biogen
Eloctate for hemophilia A is expected to generate $800 million.
4. Shire Pharmaceuticals
Cinryze for hereditary angioedema is expected to generate $765 million. In October, Cinryze received Fast Track Designation from the U.S. Food and Drug Administration (FDA) in subjects with Antibody Mediated Rejection in renal transplant recipients.
5. Vertex
Kalydeco for cystic fibrosis is expected to generate $551 million. Earlier this year, Kalydeco, the first drug designed to counter the genetic cause of cystic fibrosis, was touted by the White House during the State of the Union Address. During his talk to the nation, President Barack Obama pointed out a guest of the first lady who is taking Kalydeco. Kalydeco was approved in 2012 and is aimed at a “select few” of the genetic mutations that can cause cystic fibrosis.
6. Infinity Pharmaceuticals
Duvelisib for blood cancers, which is currently in Phase III development, is expected to generate $462 million in revenue.
7. Genzyme
Fabrazyme for Fabry Disease is expected to generate $449 million in revenue.
Makena for pre-term birth, which was approved in 2011, is expected to generate $430 million.
9. Alnylam Pharmaceuticals
Patisiran for TTR amyloidosis, which is currently in Phase III development, is expected to generate $391 million.
10. Tesaro
Nirparib for ovarian and breast cancer, which is currently in Phase III development, is expected to generate $325 million.Following Massachusetts, New Jersey is home to eight orphan disease drugs, including Merck & Co. ’s blockbuster Keytruda and Celgene Corporation ’s Revlimid. California is home to seven orphan disease drugs.
