BridgeBio has faced plenty of questions about its “hub-and-spoke" model over the years, but now its strategy has yielded the company's first FDA approval. Though the new drug isn't expected to be a big moneymaker itself, the nod brings the biotech "one step closer to becoming a fully integrated company," one analyst figures.
The FDA approved BridgeBio’s Nulibry to treat the ultra-rare, life-threatening disease molybdenum cofactor deficiency (MoCD) Type A. Nulibry is now the first FDA-approved medicine to treat the disease.
MoCD symptoms, including severe encephalopathy and intractable seizures, typically arise just after birth. The condition progresses quickly, and median survival is only about four years.
Only around 150 patients worldwide are believed to have the disease, and though the drug debuts with a list price of $500,000 per year, it's only expected to bring in some $10 million at its peak, Mizuho analysts said after the approval. But the approval could help fuel other BridgeBio meds, thanks to a priority review voucher and the company build-out required to get Nulibry on the market.
BridgeBio's Origin BioSciences subsidiary picked up Nulibry when Alexion cleaned house in 2017, selling off programs in a companywide restructuring. A cyclic pyranopterin monophosphate replacement therapy, Nulibry treats the disease by replacing cPMP and allowing molybdenum cofactor synthesis steps to proceed as normal and prevent devastating complications, BridgeBio says.
Regulators approved the drug based on data from three studies showing the medicine or the recombinant form of cPMP reduced the risk of death by 82% compared with patients in a natural history study. After three years, 84% of patients who received treatment had survived, compared with 55% of those in a control group.
Before Origin snapped up Nulibry, the drug had scored a breakthrough therapy designation from the FDA. Now, with the approval, Origin—and thus BridgeBio—scored a rare pediatric disease priority review voucher from the agency. Such vouchers can be used to speed FDA review time and can be lucrative if sold.
The companies didn’t disclose specific terms in the Alexion deal, but BridgeBio said at the time it “committed sufficient resources” to its Origin subsidiary to proceed through development and regulatory efforts, plus support commercialization. Under the deal, Alexion was set to receive payments based on development and sales milestones.
On its own, Nulibry's approval isn't a "needle-mover" for BridgeBio's market value, Mizuho analyst Salim Syed wrote in a note to clients, but it's significant for other reasons.
First, the company can either sell or use the priority review voucher on a pipeline med—for instance acoramidis, a candidate in transthyretin amyloid cardiomyopathy that analysts see as a big sales prospect. Plus, the approval forces BridgeBio to "build out certain functions, which now are in place or getting in place for future products," the analyst wrote.
Origin Biosciences is the MoCD-focused subsidiary of BridgeBio. The parent company has numerous subsidiaries focusing on therapies targeting rare diseases and cancer, and also has several gene therapies in development.
Speaking with Fierce Biotech last year, BridgeBio CEO Neil Kumar said he's heard from skeptics over the years about his decentralized, “hub-and-spoke" model for biotech. But five years, an IPO and many research programs later, the company has hit its stride, he said.
Aside from Nulibry, BridgeBio’s affiliate companies are working on drugs targeting congenital adrenal hyperplasia, autosomal dominant hypocalcemia type 1 and more.