After last quarter presenting three factors necessary for a more successful launch of hemophilia A gene therapy Roctavian, BioMarin has now added a divestiture to the list of potential paths for the struggling medicine.
Following a European approval in 2022 and a U.S. nod last year, the gene therapy has only treated four hemophilia A patients to date.
The most recent quarterly sales of just $800,000 came from the first patient to be dosed in Italy, while three Roctavian patients last year added up to $3.5 million in 2023 sales.
BioMarin pointed out reimbursement and market access challenges that prevented “interested patients” from receiving the therapy. While this year the company will focus on uptake in the U.S., Italy and Germany—or the three countries with preexisting reimbursement approvals—it is simultaneously keeping an eye on the uncertain future of the medicine in general.
All will be revealed during the rare disease drugmaker’s September investor day, where BioMarin will divulge the outcome of an assessment weighing Roctavian's future. New CEO Alexander Hardy laid out three possible paths the drug can take on the company's earnings call last week.
If uptake does increase in a “meaningful way,” Roctavian will continue as is. Or, if a “lower potential” for the asset plus a way to secure a reasonable return on investment becomes clear, the company will look to even out the current level of investment “across the organization,” Hardy explained on the Wednesday, April 24, call.
The third option would be to scrap the gene therapy completely through a divestiture, for which the evaluation criteria and related valuation timing would be announced at the investor day. While that option is “not our focus right now,” a divestiture would be “absolutely” considered if Roctavian is removed from the portfolio, Hardy said.
Still, as of now, “we are not engaging with people around the divestment of Roctavian,” Hardy added. “Our focus is really on establishing the opportunity right now.”
To that end, the company is trying to drive uptake by zeroing in on “patient pull-through” in the three main commercial markets of the U.S, Italy and Germany.
“Establishing proof of concept in pulling those patients through that last mile and getting them treated is what we need to do on a going-forward basis,” outgoing chief commercial officer Jeffrey Ajer said on the call.
Meanwhile, over in R&D, the company is turning to solely the most productive assets. On the commercial side, the dwarfism drug Voxzogo has become a top priority given its rapid growth. Over the first quarter alone, more than 500 new patients began treatment. Voxzogo pulled sales of $152 million over the period, spiking 70% year-over-year and largely contributing to the company’s total quarterly haul of $648 million.
Editor's note: The story was updated to correctly reflect Roctavian's first-quarter sales of $800,000.