In a move to control some of the world’s most expensive medicine, nearly a dozen drugmakers have allocated in aggregated $2 billion to invest in the manufacturing of gene therapy.
Drugmakers Pfizer and Novartis are leading the pack as both are working toward building their own gene therapy manufacturing plants. Novartis has already allocated $500 million for its Swiss facility, while Pfizer has $600 million put aside for its gene manufacturing base. Additionally, PTC Therapeutics, Krystal Biotech, and bluebird bio are also investing in gene therapy facilities.
So why the big interest, and investment, in gene therapy? These treatments work as a replacement for missing or mutated genes in a person’s cells and help treat certain diseases. Yet, these treatments are expensive to produce, and can cost patients upwards $1 million.
Drugmakers want to build their own manufacturing plants devoted entirely to cultivating these treatments for several reasons, including the rising costs of third party manufacturers and the need for space and tools necessary to undertake proper cultivating and testing of the complex treatments. Moreover, drugmakers require close supervision of safe manufacturing standards that meet U.S. Food and Drug Administration (FDA) guidelines. (The FDA is in the process finalizing new guidelines for gene therapy manufacturing, which are expected by the end of the year.)
Time and money are lost for drugmakers without the proper manufacturing space to produce these treatments. When wait times can go up to 18 months for a production slot to open up, millions of dollars are lost.
“There’s so little capacity and capability at contract manufacturers for the novel gene therapy processes being developed by companies,” said David Lennon, president of AveXis, Novartis’s gene therapy division in a Reuters interview. “We need internal manufacturing capabilities in the long term.”
The USDA is currently working towards getting more gene therapy treatments to market faster and has already approved Novartis’ treatment for a rare form of muscular disorder. It is expecting the U.S. market to see 40 new therapies within the next two years.
Currently, there are approximately several hundred gene therapies for everything from muscular dystrophy to sickle cell anemia that are in development from nearly 30 drug producers. BioMarin Pharmaceutical, which developed a treatment for hemophilia, was recently recognized for its Novato, CA-based gene manufacturing facility, which is currently the largest, built in 2017.
“Between the trade secrets, the cost schedules and the time lag, it makes a whole lot of sense,” said Krish Krishnan, chief executive, Krystal Biotech, in a recent report. “If you can do it, build out your own facilities—and more and more gene therapy companies have started to do that.”