Eleven drug manufacturers, led by Pfizer and Novartis, have pledged nearly $ 2 billion in investment in the gene therapy industry since 2018 to better control the world’s most expensive drugs, according to a Reuters analysis..
In a recent interview with the head of the company’s gene therapy business, Reuters was told the full $ 500 million investment plan for Novartis. It is second only to Pfizer, which has pledged $ 600 million to build its own gene therapy factories, according to public filings and interviews with others in the industry..
Gene therapy aims to treat certain diseases by replacing a missing or mutated version of a gene found in a patient’s cells with healthy copies. Drug makers claim they can cure devastating diseases in a single procedure and justify prices in excess of US $ 1 million per patient.
But treatments are also extremely complex, including growing live material, and yet pose the risk of serious side effects..
Pharmaceutical companies say building their own manufacturing facilities is a response to rising costs and delays associated with reliance on third-party contractors that are also expanding to capitalize on demand..
Pharmaceutical companies say owning their own manufacturing facilities helps protect proprietary manufacturing methods and more effectively address any concerns raised by the US Food and Drug Administration (FDA), which closely monitors manufacturing standards..
«Contract manufacturers have very little capacity and capacity for new gene therapy processes being developed by companies», – said David Lennon, President of AveXis, Novartis Gene Therapy Division. «We need internal manufacturing capabilities in the long term».
Bob Smith, senior vice president of global gene therapy at Pfizer, admitted that drug makers are doing «leap of faith», when they make large capital investments in new treatments before they are approved or, in some cases, even provide data demonstrating benefits.
Gene therapy is one of the most "hot" areas of drug research, and with life-changing opportunities, the FDA is helping to accelerate the time to market for a new drug or treatment.
To date, the FDA has approved two of them, including the drug Novartis Zolgensma for a $ 2 Million Rare Muscle Disorder and Expects 40 New Gene Therapy Drugs to Hit the U.S. Market by 2022.
Currently, about 30 drug manufacturers are developing several hundred drugs from hemophilia to Duchenne muscular dystrophy and sickle cell anemia..
The proliferation of these treatments is pushing the boundaries of existing manufacturing capacity in the industry. Gene therapy developers who need to outsource production have to wait about 18 months, company executives told Reuters.
They are also charged millions of dollars in seat reservation fees, more than double what they received a few years ago, according to gene therapy developer RegenxBio..
As a result, companies including Bluebird Bio, PTC Therapeutics and Krystal Biotech are also investing in gene therapy production, according to Reuters..
They follow Biomarin Pharmaceutical Inc, the developer of the hemophilia gene therapy, which built one of the largest manufacturing facilities in 2017..
The FDA closely monitors behind compliance with production and research standards in the gene therapy industry.
This comes amid the agency’s disclosure in August that it was investigating alleged data manipulation by former Novartis executives – AveXis.
AveXis has changed its method of measuring the activity of Zolgensma in animal studies. When the results with the new method fell short of expectations, the FDA and Novartis say, executives tweaked the data to hide it..
Scandal highlighted the importance of a consistent manufacturing process in gene therapy, industry executives say.
According to some of them, the FDA in recent meetings emphasized the need for the continuity of production processes all the way from drug development to its commercialization..
Drugmakers can avoid pitfalls such as having to move to a larger facility if the options for contract manufacturers are limited, executives say..
FDA to Finalize New Gene Therapy Manufacturing Guidelines Expected by Year End.
«The stability of the production process is always a major concern for the agency.», – FDA spokeswoman Stephanie Kakkomo told Reuters.
Highlighting the pressure on the industry, Sarepta Therapeutics, which mainly attracts third-party drug manufacturers, postponed clinical trials of its drug for the treatment of Duchenne muscular dystrophy in August, telling investors that it wants to avoid any questions from regulators about the sequencing of its therapy on a commercial scale..
Krystal, which develops treatments for rare skin conditions, has built one manufacturing facility and plans to invest over $ 50 million in a new one, which it will start building in December..
MeiraGTx, which specializes in gene therapy for eye diseases, currently spends an estimated $ 25 million a year on manufacturing, including the development process.
However, despite such moves, contract manufacturers such as Lonza and Thermo Fisher are confident that their businesses will continue to grow due to strong demand..
Thermo Fisher told investors that its Brammer gene therapy business, acquired in May, could soon generate $ 500 million in revenue per year, double its 2019 projected revenue..
Lonza CEO Mark Funk is also optimistic.
«Gene therapy demand has increased», – he said in an interview with Reuters. «We believe this will continue in the coming years.».