On a quest to deliver its first profits this year, BioMarin is starting to reap the rewards of its latest rare disease launch, executives said on an investor call Wednesday.
“So we begin 2022 poised for significant growth and the transition to sustainable … profitability,” BioMarin CEO Jean-Jacques Bienaime said at the top of the call. Interest in the company’s new med Voxzogo—which won an accelerated approval in November to treat a form of dwarfism known as achondroplasia—has been “very positive,” the CEO said.
Thanks to the launch’s strong start, BioMarin has upped its full-year Voxzogo sales forecast to between $100 million and $125 million. The company previously expected the med to pull in between $90 million and $115 million for the year.
Overall, the company said it tallied record quarterly revenues of $519 million in the first three months of 2022, with Voxzogo chipping in $20 million.
Of the $20 million for Voxzogo, $13 million came from outside the U.S., which demonstrates “the breadth of our global footprint and commercial capabilities and the importance of the ex-U.S. markets,” Bienaime said.
Meanwhile, BioMarin scored $89 million from the sale of a rare pediatric disease priority review voucher it landed in connection with its U.S. Voxzogo approval.
“We will continue to build on this financial, commercial and regulatory momentum in 2022 and beyond as we make the transition to an earnings growth story,” the CEO added.
Digging more into Voxzogo’s launch figures, 284 children were being treated with the med as of March 31, Jeff Ajer, BioMarin’s chief commercial officer, said on the call.
“This includes 201 children in countries outside of the United States and 83 children within the United States,” he said, adding that “[a]n estimated additional 53 children were in process in the United States as of April 15.”
Looking ahead, the company hopes to nab Voxzogo approvals in Australia and Japan later this year.
“The opportunity in Japan is expected to be significant and we expect revenue contributions to begin there later this year,” Ajer said.
Stateside, BioMarin has seen prescription demand “pick up quickly,” the COO explained. The company has been able to “rapidly” convert patient referrals to patient starts and has received prescriptions from geneticists and pediatric endocrinologists “as expected.”
“We also see more payer coverage policies published, which are largely consistent with our label or our clinical trials criteria and are aligned with our expectations,” Ajer said.
Aside from its Voxzogo launch, execs are looking forward to a potential approval for hemophilia gene therapy Roctavian after a prior delay. The company is expecting an approval decision in Europe this year and plans to resubmit its FDA application in June.
BioMarin expects total revenues between $2.05 billion and $2.15 billion this year.