BioMarin, improving lives of rare disease patients

BioMarin, improving lives of rare disease patients
Dan Maher, Former Senior Vice President of Product Development, BioMarin; Dr Robert Baffi, Executive VP Technical Operations, BioMarin, and Michael O’Donnell, Site Leader, BioMarin.

BioMarin is investing €38 million at its plant in Shanbally, Co Cork. It has been seven years since the US pharmaceutical rare disease leader BioMarin first established a presence in Ireland.

Since then, the company has gone from strength to strength. In 2019, it will put its latest expansion plans into action to meet rising demand for its innovative therapies by increasing operational capacity at its Irish manufacturing facility.

The €38 million upgrade at the plant in Shanbally, Co Cork, will boost BioMarin’s workforce in Ireland by around 50 people, to over 450 employees.

BioMarin is an international biopharmaceutical company headquartered in San Rafael, California, that focuses on developing first-in-class and best-in-class treatments that provide meaningful advances to patients who live with serious and life-threatening rare genetic diseases.

These conditions mostly affect children and they can be so uncommon that the entire patient population numbers as few as 1,000 people worldwide.

Expanding in Ireland

The BioMarin development at Shanbally is being overseen by Site Leader and the Vice President of Operations, Michael O’Donnell.

“Creating an end-to-end manufacturing site will ultimately benefit our patients by providing us with much more local control over our entire supply chain,” says O’Donnell.

Construction enabling works are underway, with an anticipated completion date in 2020. The facility will eventually fill three BioMarin product lines, with capability to transfer in more as required.

As well as an additional 100 staff working on site during the development, BioMarin is recruiting a wide range of permanent roles to support the site’s expanded function.

Mr O’Donnell says: “We are recruiting for multiple disciplines from quality, validation, engineering and operations functions.

“BioMarin also has a Dublin office, led by Jim Lennertz, Senior Vice President of Commercial Operations for EUMEA, employing a team of more than 80 people in Global Commercial Operations — which includes marketing, sales, customer service, legal and compliance functions. This is another area of the business that is also continuing to grow.”

Biomarin, Cork, where new plant works will be complete in 2020.
Biomarin, Cork, where new plant works will be complete in 2020.

Supporting rare patient diseases

Founded in 1997, BioMarin is a pioneer in the treatment of rare diseases, with a recognised track record of developing innovative medicines quickly, including seven approved therapies for rare inherited diseases.

The company’s mission is to bring new treatments to market that will make a big impact on small patient populations.

“We like to think of ourselves as pioneers, committed to delivering new therapies from the lab bench to the patient’s bedside as quickly as possible, often when none existed previously,” says O’Donnell.

“Rare diseases like the ones we treat are often inherited, difficult to diagnose and progressively debilitating.

Many of the patients we support are children, whose lives are profoundly affected by genetic conditions they’ve been born with.

BioMarin has developed treatments for rare diseases including phenylketonuria and several types of mucopolysaccharide.

“One of our most recent advances is for CLN2, a form of Batten disease. It’s a devastating condition affecting children, with rapid progression that means patients eventually lose the ability to walk and talk, as well as experiencing chronic seizures, dementia, blindness and severe pain. BioMarin has developed the only treatment for CLN2 proven to stabilise or slow the natural progression of the disease, allowing children to maintain cognitive and motor function for a longer period.”

According to O’Donnell, BioMarin’s focus on life-changing therapies like this stands out in an industry where addressing unmet medical needs is the norm.

“There are around 7,000 known rare diseases, and despite some breakthroughs, the overwhelming majority of them still have no treatment.

"At BioMarin we invest nearly 50 per cent of our global annual revenue/turnover in R&D for these challenging conditions, that’s more than double the industry average.

"We’re committed to making a real difference, so we only focus on treatments that are first-to-market or offer a significant benefit over existing options.”

Innovating for the future

BioMarin is consistently recognised for innovation and in 2017 was ranked among the world’s top 15 most innovative companies by Forbes magazine for the fourth consecutive year.

“The scaling up of our operations in Shanbally is just one part of BioMarin’s amazing success story — and that success stems from the company’s focus on science and the hope our therapies can bring to patients who often have few other options,” said Michael O’Donnell.

“BioMarin is an exciting company to be part of. We already market first-ever therapies for a number of conditions, and our pipeline has the potential to deliver transformative innovation in other rare diseases.”

That pipeline includes one of the world’s first investigational gene therapies which could present a new way to treat haemophilia.

It’s not just Shanbally that’s expanding.

For BioMarin’s team in Ireland, and the company’s 2,700 employees worldwide, 2019 will see new horizons opening within the care of patients with rare diseases.

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