In his nine months as CEO, BioMarin’s new chief Alexander Hardy has so far overseen shake-ups in the company’s leadership team, a slim-down of its R&D program and decisive action on the future of hemophilia A gene therapy Roctavian. But the company still has work to do to achieve what Hardy says could be “one of the most exciting growth stories in biotech.”
At an investor event Wednesday, the former Genentech veteran divulged an ambitious plan to quadruple the California-based drugmaker’s patient reach over the next decade and land $4 billion in revenue by 2027.
The $4 billion goal would double 2023’s total revenue of $2.4 billion and put BioMarin in the top quartile of biopharma revenue growth, Hardy said.
To get there, BioMarin will depend on three separate but connected pillars. The growth aspect largely centers on the company’s Voxzogo, which is already its top growth driver with its sole indication for achondroplasia, or dwarfism.
With five more indications planned through 2031, Voxzogo could prove a $5 billion opportunity with a total addressable patient population of around 420,000 across its global markets, according to BioMarin.
The next expansion for Voxzogo would be to hypochondroplasia in 2027, which would make it the first approved medicine for the genetic disease, followed by what could be a lucrative idiopathic short stature nod in 2030 and then indications for Noonan syndrome, Turner syndrome and SHOX deficiency on the docket for 2031.
The patient pool for the drug’s current dwarfism label is 24,000, but it’s still “only the beginning” for Voxzogo in that indication with more than 80% of the market yet to be penetrated, Hardy pointed out. Even so, the company’s confidence is bolstered by the drug’s standard-of-care status, high compliance rates and a broad age range that can treat patients from infancy, Chief Commercial Officer Cristin Hubbard explained.
“Voxzogo is clearly the most exciting multi-indication product in our portfolio and one of the most exciting in the entire industry,” Hardy added.
Before Voxzogo came about, much of the company’s revenue was drawn from bread-and-butter enzyme therapies, starting with its very first commercial launch in 2005 of Naglazyme. These days, BioMarin’s five enzyme therapies still bring in more than $1 billion annually and remain the standards of care for some otherwise untreated conditions the drugs target in approximately 80 countries, Hubbard explained.
Still, BioMarin looks to further expand the franchise’s reach with efforts to identify more patients, maintain an over 90% patient adherence and invest in emerging markets such as the Middle East and Latin America, Hubbard said.
Meanwhile, in business structure, the company has divided itself into three business units: skeletal conditions, enzyme therapies and Roctavian.
Gene therapy Roctavian had already been a separate beast from the rest of BioMarin’s portfolio and presented more challenges as well. With a slower-than-expected launch, the company recently decided to cut costs around the drug and focus its efforts in only three markets with no further plans to expand. As a result, Roctavian support staff was separated from the rest of the company’s portfolio, Hardy said during BioMarin’s second-quarter earnings call in August.
The wider business structure shuffle, coupled with the Roctavian changes, is translating into 225 layoffs representing 7% of BioMarin’s global workforce, the company announced a few days ago. Another 170 positions were on the chopping block as part of a layoff round disclosed in May.
Outside of its approved medicines, BioMarin looks to launch two products by 2027 or 11 by 2034. The company’s pipeline targets diseases such as Duchenne muscular dystrophy and von Willebrand disease, to name a few.
As for the shorter-term outlook, BioMarin expects its 2024 revenues to total between $2.75 billion and $2.82 billion.